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Trial record 1 of 1 for:    ALE06
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Randomized MMF Withdrawal in Systemic Lupus Erythematosus (SLE) (ALE06)

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ClinicalTrials.gov Identifier: NCT01946880
Recruitment Status : Terminated (Slow enrollment.)
First Posted : September 20, 2013
Results First Posted : August 17, 2020
Last Update Posted : August 17, 2020
Sponsor:
Collaborators:
Autoimmunity Centers of Excellence
Rho Federal Systems Division, Inc.
Information provided by (Responsible Party):
National Institute of Allergy and Infectious Diseases (NIAID)

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Systemic Lupus Erythematosus
SLE
Interventions Drug: Mycophenolate Mofetil
Drug: Hydroxychloroquine or Chloroquine
Drug: Prednisone
Enrollment 102
Recruitment Details Nineteen study sites in the US were activated. A total of 123 participants were screened and 102 were randomized from November 2013 to May 2018.
Pre-assignment Details  
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60). Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Period Title: Overall Study
Started 50 52
Completed 43 49
Not Completed 7 3
Reason Not Completed
Lost to Follow-up             1             0
Physician Decision             1             1
Withdrawal by Subject             3             2
Met Primary Endpoint             1             0
Personal and Family Issues             1             0
Arm/Group Title Maintenance Withdrawal Total
Hide Arm/Group Description Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60). Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first). Total of all reporting groups
Overall Number of Baseline Participants 50 52 102
Hide Baseline Analysis Population Description
All randomized participants.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 50 participants 52 participants 102 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
48
  96.0%
50
  96.2%
98
  96.1%
>=65 years
2
   4.0%
2
   3.8%
4
   3.9%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 50 participants 52 participants 102 participants
42.4  (12.9) 41.6  (12.5) 42.0  (12.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 50 participants 52 participants 102 participants
Female
39
  78.0%
47
  90.4%
86
  84.3%
Male
11
  22.0%
5
   9.6%
16
  15.7%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 50 participants 52 participants 102 participants
Hispanic or Latino
10
  20.0%
12
  23.1%
22
  21.6%
Not Hispanic or Latino
40
  80.0%
40
  76.9%
80
  78.4%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 50 participants 52 participants 102 participants
American Indian or Alaska Native
2
   4.0%
0
   0.0%
2
   2.0%
Asian
2
   4.0%
8
  15.4%
10
   9.8%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
19
  38.0%
22
  42.3%
41
  40.2%
White
25
  50.0%
17
  32.7%
42
  41.2%
More than one race
0
   0.0%
2
   3.8%
2
   2.0%
Unknown or Not Reported
2
   4.0%
3
   5.8%
5
   4.9%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 50 participants 52 participants 102 participants
50
 100.0%
52
 100.0%
102
 100.0%
Disease Manifestation: Renal vs. Non-Renal Disease   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 50 participants 52 participants 102 participants
Renal Disease
40
  80.0%
38
  73.1%
78
  76.5%
Non-Renal Disease
10
  20.0%
14
  26.9%
24
  23.5%
[1]
Measure Description: Renal disease, which included participants who had renal disease at screening as well as those who had renal manifestations in the past, versus non-renal disease, which included participants who never had renal disease.
Baseline MMF Dose   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 50 participants 52 participants 102 participants
< 2000 mg/day
26
  52.0%
30
  57.7%
56
  54.9%
≥ 2000 mg/day
24
  48.0%
22
  42.3%
46
  45.1%
[1]
Measure Description: Baseline dose of Mycophenolate Mofetil (MMF), defined as < 2000 mg per day versus ≥ 2000 mg per day.
1.Primary Outcome
Title Number of Participants Experiencing Clinically Significant Disease Reactivation by Week 60
Hide Description Disease reactivation requires:1) SELENA-SLEDAI mild/moderate or severe flare,and 2) Increased immunosuppressive therapy on a sustained basis,defined by one of the following criteria:a) Sustained activity:Significant prolonged SLE flare requiring steroid increase/burst to ≥15 mg/day prednisone (or equivalent) for >4 weeks.b) Frequent relapsing/remitting:Participant flares requiring an increase/burst of steroids and is successfully tapered to <15 mg/day within 4 weeks, but this occurs on >2 occasions, or IA, IM or IV steroids on more than1 occasion.c)Clinical activity of sufficient severity to warrant resumption of/increased dose of MMF or addition of other major immunosuppressive including AZA or MTX.Regardless of steroid use, if the investigator observes disease activity of sufficient severity to warrant resumption, addition or increase in dosage of major immunosuppressant in the setting of a SELENA-SLEDAI flare, participant has met the primary endpoint.Risk difference also included
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups to which they were randomized.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 49 51
Measure Type: Count of Participants
Unit of Measure: Participants
5
  10.2%
9
  17.6%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments [Not Specified]
Type of Statistical Test Other
Comments The estimated risk difference (i.e. risk(MMF Withdrawal) - risk(MMF Maintenance)) and 95% confidence intervals around the risk difference at Week 60 were reported using the Kaplan-Meier product-limit estimator.
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value 0.07
Confidence Interval (2-Sided) 95%
-0.068 to 0.214
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Time to Clinically Significant Disease Reactivation
Hide Description The time to clinically significant disease reactivation was defined as the time from Baseline/Day 0 to the date of the first Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) assessment that met (or went on to meet) the criteria for clinically significant disease reactivation. Time to clinically significant disease reactivation was defined in study weeks as: date of SELENA-SLEDAI assessment that met reactivation criteria minus (-) baseline date.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups they were randomized. Participants who met disease reactivation were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 5 9
Mean (Standard Deviation)
Unit of Measure: Weeks
38.0  (18.7) 38.5  (19.6)
3.Secondary Outcome
Title Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60
Hide Description The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes mild/moderate or severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups to which they were randomized.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 49 51
Measure Type: Count of Participants
Unit of Measure: Participants
20
  40.8%
25
  49.0%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value 0.08
Confidence Interval (2-Sided) 95%
-0.116 to 0.279
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Lupus Nephritis Subgroup
Hide Description The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the group they were randomized. Participants in the renal subgroup were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 39 37
Measure Type: Count of Participants
Unit of Measure: Participants
15
  38.5%
19
  51.4%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments [Not Specified]
Type of Statistical Test Other
Comments The estimated risk difference (i.e. risk(MMF Withdrawal) - risk(MMF Maintenance)) and 95% confidence intervals around the risk difference at Week 60 were reported using the Kaplan-Meier product-limit estimator.
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value 0.13
Confidence Interval (2-Sided) 95%
-0.091 to 0.360
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Non-Lupus Nephritis Subgroup
Hide Description The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the group they were randomized. Participants in the non-renal subgroup were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 10 14
Measure Type: Count of Participants
Unit of Measure: Participants
5
  50.0%
6
  42.9%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments [Not Specified]
Type of Statistical Test Other
Comments The estimated risk difference (i.e. risk(MMF Withdrawal) - risk(MMF Maintenance)) and 95% confidence intervals around the risk difference at Week 60 were reported using the Kaplan-Meier product-limit estimator.
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value -0.07
Confidence Interval (2-Sided) 95%
-0.475 to 0.333
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF<2000 mg/Day Subgroup
Hide Description

The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.

MMF: mycophenolate mofetil

Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the group they were randomized. Baseline MMF <2000 mg/day participants were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 26 30
Measure Type: Count of Participants
Unit of Measure: Participants
11
  42.3%
13
  43.3%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments The estimated risk difference (i.e. risk(MMF Withdrawal) - risk(MMF Maintenance)) and 95% confidence intervals around the risk difference at Week 60 were reported using the Kaplan-Meier product-limit estimator.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value -0.02
Confidence Interval (2-Sided) 95%
-0.286 to 0.249
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF ≥ 2000 mg/Day Subgroup
Hide Description

The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.

MMF: mycophenolate mofetil

Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the group they were randomized. Baseline MMF >=2000 mg/day participants were analyzed
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 23 21
Measure Type: Count of Participants
Unit of Measure: Participants
9
  39.1%
12
  57.1%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments [Not Specified]
Type of Statistical Test Other
Comments The estimated risk difference (i.e. risk(MMF Withdrawal) - risk(MMF Maintenance)) and 95% confidence intervals around the risk difference at Week 60 were reported using the Kaplan-Meier product-limit estimator.
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value 0.20
Confidence Interval (2-Sided) 95%
-0.090 to 0.497
Estimation Comments [Not Specified]
8.Secondary Outcome
Title Number of Participants Experiencing a Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60
Hide Description The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups to which they were randomized.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 49 51
Measure Type: Count of Participants
Unit of Measure: Participants
4
   8.2%
8
  15.7%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments [Not Specified]
Type of Statistical Test Other
Comments The estimated risk difference (i.e. risk(MMF Withdrawal) - risk(MMF Maintenance)) and 95% confidence intervals around the risk difference at Week 60 were reported using the Kaplan-Meier product-limit estimator.
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value 0.08
Confidence Interval (2-Sided) 95%
-0.056 to 0.211
Estimation Comments [Not Specified]
9.Secondary Outcome
Title Number of Participants Experiencing a Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Lupus Nephritis Subgroup
Hide Description The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the group they were randomized. Participants in the renal subgroup were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 39 37
Measure Type: Count of Participants
Unit of Measure: Participants
3
   7.7%
7
  18.9%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments [Not Specified]
Type of Statistical Test Other
Comments The estimated risk difference (i.e. risk(MMF Withdrawal) - risk(MMF Maintenance)) and 95% confidence intervals around the risk difference at Week 60 were reported using the Kaplan-Meier product-limit estimator.
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value 0.12
Confidence Interval (2-Sided) 95%
-0.041 to 0.284
Estimation Comments [Not Specified]
10.Secondary Outcome
Title Number of Participants Experiencing a Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Non-Lupus Nephritis Subgroup
Hide Description The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the group they were randomized. Participants in the non-renal subgroup were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 10 14
Measure Type: Count of Participants
Unit of Measure: Participants
1
  10.0%
1
   7.1%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments [Not Specified]
Type of Statistical Test Other
Comments The estimated risk difference (i.e. risk(MMF Withdrawal) - risk(MMF Maintenance)) and 95% confidence intervals around the risk difference at Week 60 were reported using the Kaplan-Meier product-limit estimator.
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value -0.04
Confidence Interval (2-Sided) 95%
-0.285 to 0.206
Estimation Comments [Not Specified]
11.Secondary Outcome
Title Number of Participants Experiencing Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF <2000 mg/Day Subgroup
Hide Description

The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.

MMF: mycophenolate mofetil

Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the group they were randomized. Baseline MMF <2000 mg/day participants were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 26 30
Measure Type: Count of Participants
Unit of Measure: Participants
3
  11.5%
6
  20.0%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments [Not Specified]
Type of Statistical Test Other
Comments The estimated risk difference (i.e. risk(MMF Withdrawal) - risk(MMF Maintenance)) and 95% confidence intervals around the risk difference at Week 60 were reported using the Kaplan-Meier product-limit estimator.
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value 0.09
Confidence Interval (2-Sided) 95%
-0.118 to 0.289
Estimation Comments [Not Specified]
12.Secondary Outcome
Title Number of Participants Experiencing Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF ≥ 2000 mg/Day Subgroup
Hide Description

The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.

MMF: mycophenolate mofetil

Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the group they were randomized. Baseline MMF >=2000 mg/day participants were analyzed
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 23 21
Measure Type: Count of Participants
Unit of Measure: Participants
1
   4.3%
2
   9.5%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments [Not Specified]
Type of Statistical Test Other
Comments The estimated risk difference (i.e. risk(MMF Withdrawal) - risk(MMF Maintenance)) and 95% confidence intervals around the risk difference at Week 60 were reported using the Kaplan-Meier product-limit estimator.
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value 0.05
Confidence Interval (2-Sided) 95%
-0.103 to 0.212
Estimation Comments [Not Specified]
13.Secondary Outcome
Title Time to First Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare
Hide Description The time to first mild/moderate or severe SELENA-SLEDAI flare was defined as the time from Baseline/Day 0 to the date of the first mild/moderate or severe SELENA-SLEDAI flare. Time to SELENA-SLEDAI flare was defined in study weeks as: date of SELENA-SLEDAI flare minus (-) baseline date. The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes mild/moderate or severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups they were randomized. Participants who had SELENA-SLEDAI flares were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 20 25
Mean (Standard Deviation)
Unit of Measure: Weeks
20.5  (19.5) 27.5  (16.7)
14.Secondary Outcome
Title Time to First Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare
Hide Description The time to first severe SELENA-SLEDAI flare was defined as the time from Baseline/Day 0 to the date of the first severe SELENA-SLEDAI flare. Time to severe SELENA-SLEDAI flare was defined in study weeks as: date of SELENA-SLEDAI flare minus (-) baseline date. The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes mild/moderate or severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups they were randomized. Participants who had SELENA-SLEDAI flares were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 4 8
Mean (Standard Deviation)
Unit of Measure: Weeks
43.5  (9.9) 41.5  (17.5)
15.Secondary Outcome
Title Number of Participants Experiencing Any British Isles Lupus Assessment Group (BILAG) A Flare by Week 60
Hide Description The BILAG assesses participants on a variety of disease activity criteria in nine body system categories (constitutional, mucocutaneous, neuropsychiatric, musculoskeletal, cardiorespiratory, gastrointestinal, ophthalmic, renal, and hematological). Each category is scored as an A, B, C, or D/E, where A indicates most severe disease activity and D/E indicates inactive/no disease activity. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups to which they were randomized.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 49 51
Measure Type: Count of Participants
Unit of Measure: Participants
1
   2.0%
4
   7.8%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments [Not Specified]
Type of Statistical Test Other
Comments The estimated risk difference (i.e. risk(MMF Withdrawal) - risk(MMF Maintenance)) and 95% confidence intervals around the risk difference at Week 60 were reported using the Kaplan-Meier product-limit estimator.
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value 0.06
Confidence Interval (2-Sided) 95%
-0.028 to 0.149
Estimation Comments [Not Specified]
16.Secondary Outcome
Title Number of Participants in the Lupus Nephritis Subgroup Experiencing a British Isles Lupus Assessment Group (BILAG) A Renal Flare by Week 60
Hide Description The BILAG assesses participants on a variety of disease activity criteria in nine body system categories (constitutional, mucocutaneous, neuropsychiatric, musculoskeletal, cardiorespiratory, gastrointestinal, ophthalmic, renal, and hematological). Each category is scored as an A, B, C, or D/E, where A indicates most severe disease activity and D/E indicates inactive/no disease activity. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the group they were randomized. Participants in the renal subgroup were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 39 37
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
2
   5.4%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments [Not Specified]
Type of Statistical Test Other
Comments The estimated risk difference (i.e. risk(MMF Withdrawal) - risk(MMF Maintenance)) and 95% confidence intervals around the risk difference at Week 60 were reported using the Kaplan-Meier product-limit estimator.
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value 0.06
Confidence Interval (2-Sided) 95%
-0.020 to 0.134
Estimation Comments [Not Specified]
17.Secondary Outcome
Title Change From Baseline in the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Disease Damage Index for Systemic Lupus Erythematosus (SLICC/DI): Total Score
Hide Description The SLICC/DI measures accumulated damage that has occurred since the onset of systemic lupus erythematosus (SLE), regardless of cause, in 12 organ systems. SLE damage is defined as an irreversible change in an organ or system that has been present for at least 6 months. The SLICC/DI includes 39 areas of damage in 12 domains, where each item is rated as present or absent; if evidence of damage is present for a particular item, it is given a score of 1. Some items are scored with 2 or 3 points in the case of recurring events or end stage renal disease. The SLICC/DI total score will be computed as the sum of all scores for items indicated as present; scores can range from 0 to 45. Higher scores indicate more damage.
Time Frame Baseline (Treatment Randomization) to Week 24, Week 48, and Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups to which they were randomized.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 49 51
Mean (Standard Deviation)
Unit of Measure: Scores on a Scale
Week 24 0.0  (0.15) 0.0  (0.15)
Week 48 0.0  (0.22) 0.0  (0.15)
Week 60 0.0  (0.37) 0.0  (0.21)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments Descriptive statistics (mean and 95% confidence interval) are reported for the difference in the change from baseline in the SLICC/DI score between the treatment groups at Week 24.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.0
Confidence Interval (2-Sided) 95%
-0.1 to 0.1
Estimation Comments Estimate is for the difference in the change from baseline for MMF Withdrawal - MMF Maintenance at Week 24.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments Descriptive statistics (mean and 95% confidence interval) are reported for the difference in the change from baseline in the SLICC/DI score between the treatment groups at Week 48.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.0
Confidence Interval (2-Sided) 95%
-0.1 to 0.1
Estimation Comments Estimate is for the difference in the change from baseline for MMF Withdrawal - MMF Maintenance at Week 48.
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments Descriptive statistics (mean and 95% confidence interval) are reported for the difference in the change from baseline in the SLICC/DI score between the treatment groups at Week 60.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.0
Confidence Interval (2-Sided) 95%
-0.2 to 0.1
Estimation Comments Estimate is for the difference in the change from baseline for MMF Withdrawal - MMF Maintenance at Week 60.
18.Secondary Outcome
Title The Addition of Aggressive Adjunctive Therapy to Mycophenolate Mofetil (MMF) or Change in MMF Therapy to Cytotoxic Drug Due to Flare by Week 60
Hide Description The addition of aggressive adjunctive therapy could include intravenous (IV) immunoglobulin or rituximab at any point during the participant's study participation. A change in therapy to cytotoxic drug due to flare could include drugs such as cyclophosphamide, etc. A blinded list of study medications was reviewed to identify the addition of aggressive adjunctive therapy or cytotoxic drugs.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups to which they were randomized.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 49 51
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
1
   2.0%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments [Not Specified]
Type of Statistical Test Other
Comments The estimated risk difference (i.e. risk(MMF Withdrawal) - risk(MMF Maintenance)) and 95% confidence intervals around the risk difference at Week 60 were reported using the Kaplan-Meier product-limit estimator.
Method of Estimation Estimation Parameter Risk Difference (RD)
Estimated Value 0.02
Confidence Interval (2-Sided) 95%
-0.019 to 0.059
Estimation Comments [Not Specified]
19.Secondary Outcome
Title Cumulative Systemic Steroid Dose by Week 60
Hide Description Steroids include medications that code to a medication class which includes the terms "glucocorticoid" or "corticosteroid". Systemic steroids will include any of these steroids that are taken by mouth (PO), intravenous (IV), or intramuscular (IM). Total cumulative systemic steroid dose, in milligrams, was summarized over the 60 week study period, or until early study termination, for each participant.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups to which they were randomized.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 49 51
Mean (Standard Deviation)
Unit of Measure: steroid dose (mg)
851  (1148) 912  (1995)
20.Secondary Outcome
Title Change From Baseline in the Functional Assessment of Chronic Illness Therapy (FACIT-F) Fatigue Scale (FS): Total Score
Hide Description FACIT-Fatigue scale (FS) is a 13-item questionnaire completed by the patient (participant), that provides a measure of fatigue/quality of life, with a 7-day recall period. The participant scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). The larger the participant's response to the questions (with the exception of 2 negatively stated), the greater the fatigue. For all questions, except for the 2 negatively stated ones, the code was reversed and a new score was calculated as 4 minus the participant's response. The sum of all responses resulted in the FACIT-FS score for a total possible score of 0 (worse score) to 52 (better score). A higher score reflected an improvement in the participant's health status. A decrease in the FACIT-FS score reflects worse fatigue/quality of life.
Time Frame Baseline (Treatment Randomization) to Week 24, Week 48, and Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups to which they were randomized.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 49 51
Mean (Standard Deviation)
Unit of Measure: units on a scale
Week 24 -2.41  (8.13) -0.81  (10.62)
Week 48 -3.39  (7.53) -0.85  (9.75)
Week 60 -3.13  (8.82) 0.42  (9.05)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments Descriptive statistics (mean and 95% confidence interval) are reported for the difference in the change from baseline in the FACIT fatigue score between the treatment groups at Week 24.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 1.61
Confidence Interval (2-Sided) 95%
-2.24 to 5.45
Estimation Comments Estimate is for the difference in the change from baseline for MMF Withdrawal - MMF Maintenance at Week 24.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments Descriptive statistics (mean and 95% confidence interval) are reported for the difference in the change from baseline in the FACIT fatigue score between the treatment groups at Week 48.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 2.54
Confidence Interval (2-Sided) 95%
-1.13 to 6.21
Estimation Comments Estimate is for the difference in the change from baseline for MMF Withdrawal - MMF Maintenance at Week 48.
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments Descriptive statistics (mean and 95% confidence interval) are reported for the difference in the change from baseline in the FACIT fatigue score between the treatment groups at Week 60.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 3.55
Confidence Interval (2-Sided) 95%
-0.17 to 7.28
Estimation Comments Estimate is for the difference in the change from baseline for MMF Withdrawal - MMF Maintenance at Week 60.
21.Secondary Outcome
Title Change From Baseline in the Short Form Health Survey (SF-36) Physical Functioning (PF) Score
Hide Description The SF-36 is a 36-item, patient-reported survey of patient health. Higher scores indicate better outcomes while lower scores indicate more disability. The PF score is used to assess changes in physical functioning. It is scaled from 0 to 100 with a score of 0 equivalent to maximum disability and a score of 100 equivalent to no disability.
Time Frame Baseline (Treatment Randomization) to Week 24, Week 48, and Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups to which they were randomized.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 49 51
Mean (Standard Deviation)
Unit of Measure: Scores on a Scale
Week 24 -0.33  (4.05) 0.22  (7.20)
Week 48 -0.49  (6.21) 0.95  (6.13)
Week 60 -0.19  (5.63) 1.79  (7.13)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments Descriptive statistics (mean and 95% confidence interval) are reported for the difference in the change from baseline in the PF score between the treatment groups at Week 24.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.55
Confidence Interval (2-Sided) 95%
-1.82 to 2.92
Estimation Comments Estimate is for the difference in the change from baseline for MMF Withdrawal - MMF Maintenance at Week 24.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments Descriptive statistics (mean and 95% confidence interval) are reported for the difference in the change from baseline in the PF score between the treatment groups at Week 48.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 1.44
Confidence Interval (2-Sided) 95%
-1.14 to 4.03
Estimation Comments Estimate is for the difference in the change from baseline for MMF Withdrawal - MMF Maintenance at Week 48.
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments Descriptive statistics (mean and 95% confidence interval) are reported for the difference in the change from baseline in the PF score between the treatment groups at Week 60.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 1.98
Confidence Interval (2-Sided) 95%
-0.71 to 4.67
Estimation Comments Estimate is for the difference in the change from baseline for MMF Withdrawal - MMF Maintenance at Week 60.
22.Secondary Outcome
Title Change From Baseline in the Short Form Health Survey (SF-36) Physical Component Summary (PCS) Score
Hide Description The SF-36 is a 36-item, patient-reported survey of patient health. Higher scores indicate better outcomes while lower scores indicate more disability. The Physical Component Score is comprised of the Physical Functioning Scale, the Role-Physical Scale, the Bodily Pain Scale, and the General Health Scale. It is scaled from 0 to 100 with a score of 0 equivalent to maximum disability and a score of 100 equivalent to no disability.
Time Frame Baseline (Treatment Randomization) to Week 24, Week 48, and Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups to which they were randomized.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 49 51
Mean (Standard Deviation)
Unit of Measure: Scores on a Scale
Week 24 -0.75  (4.82) 0.90  (7.96)
Week 48 -1.92  (6.69) 0.25  (6.83)
Week 60 -1.51  (6.66) 1.51  (6.76)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments Descriptive statistics (mean and 95% confidence interval) are reported for the difference in the change from baseline in the PCS score between the treatment groups at Week 24.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 1.65
Confidence Interval (2-Sided) 95%
-1.03 to 4.32
Estimation Comments Estimate is for the difference in the change from baseline for MMF Withdrawal - MMF Maintenance at Week 24.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments Descriptive statistics (mean and 95% confidence interval) are reported for the difference in the change from baseline in the PCS score between the treatment groups at Week 48.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 2.17
Confidence Interval (2-Sided) 95%
-0.69 to 5.02
Estimation Comments Estimate is for the difference in the change from baseline for MMF Withdrawal - MMF Maintenance at Week 48.
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection MMF Maintenance, MMF Withdrawal
Comments Descriptive statistics (mean and 95% confidence interval) are reported for the difference in the change from baseline in the PCS score between the treatment groups at Week 60.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 3.02
Confidence Interval (2-Sided) 95%
0.22 to 5.82
Estimation Comments Estimate is for the difference in the change from baseline for MMF Withdrawal - MMF Maintenance at Week 60.
23.Secondary Outcome
Title Change From Baseline in the Lupus Quality of Life (QoL)Score
Hide Description The Lupus QoL assessment is a 34 item questionnaire across 8 domains that is designed to find out how systemic lupus erythematosus (SLE) affects a participant's life over the preceding 4 weeks. Scores range from 0 (worst QoL) to 100 (best QoL). Domains include physical health, pain, planning, intimate relationships, burden to others, emotional health, body image, and fatigue.
Time Frame Baseline (Treatment Randomization) to Week 24, Week 48, and Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups to which they were randomized.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 49 51
Mean (Standard Deviation)
Unit of Measure: Scores on a Scale
Change in Physical Health at Week 24 -1.11  (9.20) 1.04  (17.66)
Change in Physical Health at Week 48 -2.83  (11.57) 0.46  (14.06)
Change in Physical Health at Week 60 0.00  (9.02) 1.56  (13.18)
Change in Pain at Week 24 -0.17  (10.67) 1.77  (18.87)
Change in Pain at Week 48 -3.10  (12.73) 1.60  (16.72)
Change in Pain at Week 60 -0.19  (12.39) 1.42  (14.36)
Change in Planning at Week 24 0.35  (16.75) 3.90  (17.28)
Change in Planning at Week 48 -5.43  (16.66) -0.18  (18.59)
Change in Planning at Week 60 -3.03  (16.48) 0.18  (17.76)
Change in Intimate Relationships at Week 24 -1.47  (23.39) 4.29  (18.68)
Change in Intimate Relationships at Week 48 -0.81  (10.67) -1.10  (14.88)
Change in Intimate Relationships at Week 60 -2.08  (10.93) 2.86  (13.92)
Change in Burden to Others at Week 24 1.56  (21.99) 0.18  (20.30)
Change in Burden to Others at Week 48 -0.58  (16.20) -4.43  (16.56)
Change in Burden to Others at Week 60 -1.33  (17.14) -3.55  (18.78)
Change in Emotional Health at Week 24 -0.87  (18.57) 0.09  (13.02)
Change in Emotional Health at Week 48 -2.03  (15.71) -1.22  (15.13)
Change in Emotional Health at Week 60 -1.52  (17.10) -0.87  (13.06)
Change in Body Image at Week 24 2.94  (16.12) -1.09  (15.61)
Change in Body Image at Week 48 -0.17  (14.84) -0.56  (17.76)
Change in Body Image at Week 60 3.22  (15.29) -4.45  (20.15)
Change in Fatigue at Week 24 3.78  (16.04) -1.17  (20.74)
Change in Fatigue at Week 48 -2.18  (18.04) -1.56  (17.37)
Change in Fatigue at Week 60 -0.57  (15.36) -1.95  (17.69)
24.Secondary Outcome
Title Time From Clinically Significant Disease Reactivation to Improvement in British Isles Lupus Assessment Group (BILAG) From Maximum Level During Flare
Hide Description For each participant who experienced disease reactivation, time from clinically significant disease reactivation to improvement in BILAG from maximum level (at least an A or B) during the flare was calculated in study days as: date of clinically significant disease reactivation minus (-) date of BILAG improvement. If multiple body systems had a BILAG flare at the visit, then the body system with the most severe score was tracked for improvement; if multiple body systems had the same score (at least an A or B), then just one needed to show improvement.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups they were randomized. Participants who met primary endpoint were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 5 9
Mean (Standard Deviation)
Unit of Measure: Days
114.5  (123.7) 40.5  (31.8)
25.Secondary Outcome
Title Time From Clinically Significant Disease Reactivation to Recovery to Baseline British Isles Lupus Assessment Group (BILAG) Score or BILAG C
Hide Description For each participant who experienced disease reactivation, time from clinically significant disease reactivation to recovery to baseline BILAG scores or BILAG C, whichever is worse, was calculated in study days as: date of clinically significant disease reactivation minus (-) date of BILAG recovery.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups they were randomized. Participants who met primary endpoint were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 5 9
Mean (Standard Deviation)
Unit of Measure: Days
114.5  (123.7) 77.7  (63.4)
26.Secondary Outcome
Title Cumulative Excess Systemic Steroid Dose From Time of Clinically Significant Disease Reactivation to Return to Pre-Flare Dose or End of Trial Participation
Hide Description For each participant who experienced disease reactivation, excess systemic steroid dose was summed from the time of clinically significant disease reactivation until the dose returns to pre-flare levels or the end of study participation, whichever occurred first. Excess systemic steroid dose was defined as the total dose given for the flare minus (-) a participant's pre-flare steroid dose. Participants who do not have an increase in their steroid use due to the flare had their excess dose set to zero. Steroids include medications that code to a medication class which includes the terms "glucocorticoid" or "corticosteroid". Systemic steroids will include any of these steroids that are taken by mouth (PO), intravenous (IV), or intramuscular (IM).
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups they were randomized. Participants who met primary endpoint were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 5 9
Mean (Standard Deviation)
Unit of Measure: Steroid dose (mg)
812.8  (561.6) 1750.7  (3384.6)
27.Secondary Outcome
Title Time From Clinically Significant Disease Reactivation to Return to Pre-Flare Steroid Dose
Hide Description For each participant who experienced disease reactivation, time from clinically significant disease reactivation to recovery to pre-flare steroid dose was calculated in study days as: date of clinically significant disease reactivation minus (-) date of return to pre-flare steroid dose.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) population included all randomized participants who begin ALE06-provided MMF and meet study entry criteria. Participants who, for whatever reason, do not complete their assigned therapy will be included in the mITT population in the groups they were randomized. Participants who met primary endpoint were analyzed.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 5 9
Mean (Standard Deviation)
Unit of Measure: Days
NA [1]   (NA) 37 [1]   (NA)
[1]
Six of the 14 subjects who met disease reactivation were on baseline steroids. Only 1 recovered to their baseline steroid dose.
28.Secondary Outcome
Title Number of Grade 3, 4, or 5 Adverse Events (AEs) Related to Systemic Lupus Erythematosus (SLE)
Hide Description The number of Grade 3, 4, or 5 AEs classified as possibly, probably, or definitely related to SLE. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population (SP) includes all participants for whom study intervention was initiated.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 50 52
Measure Type: Number
Unit of Measure: Adverse Events
Grade 3 6 5
Grade 4 0 0
Grade 5 0 0
29.Secondary Outcome
Title Number of Grade 3, 4, or 5 Adverse Events (AEs) Related to Mycophenolate Mofetil (MMF)
Hide Description The number of Grade 3, 4, or 5 AEs classified as possibly, probably, or definitely related to MMF. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population (SP) includes all participants for whom study intervention was initiated.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 50 52
Measure Type: Number
Unit of Measure: Adverse Events
Grade 3 7 1
Grade 4 0 0
Grade 5 0 0
30.Secondary Outcome
Title Number of Grade 3, 4, or 5 Adverse Events (AEs)
Hide Description The number of Grade 3, 4, or 5 AEs. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population (SP) includes all participants for whom study intervention was initiated.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 50 52
Measure Type: Number
Unit of Measure: Adverse Events
Grade 3 18 15
Grade 4 2 0
Grade 5 0 0
31.Secondary Outcome
Title Number of Serious Adverse Events (SAEs).
Hide Description The number of SAEs. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population (SP) includes all participants for whom study intervention was initiated.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 50 52
Measure Type: Number
Unit of Measure: Serious Adverse Events
12 6
32.Secondary Outcome
Title Number of Infection-Related Adverse Events (AEs)
Hide Description The number of AEs classified as infections. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population (SP) includes all participants for whom study intervention was initiated.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 50 52
Measure Type: Number
Unit of Measure: Adverse Events
63 49
33.Secondary Outcome
Title Number of Malignancies Reported as Adverse Events (AEs).
Hide Description The number of malignancies reported as AEs. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population (SP) includes all participants for whom study intervention was initiated.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 50 52
Measure Type: Number
Unit of Measure: Adverse Events
2 3
34.Secondary Outcome
Title Number of Grade 3, 4, or 5 Hematological Adverse Events (AEs).
Hide Description The number of Grade 3, 4, or 5 hematological AEs. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population (SP) includes all participants for whom study intervention was initiated.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 50 52
Measure Type: Number
Unit of Measure: Adverse Events
Grade 3 3 0
Grade 4 0 0
Grade 5 0 0
35.Secondary Outcome
Title Mortality Related to Systemic Lupus Erythematosus (SLE)
Hide Description Mortality related to SLE is defined as any death possibly, probably, or definitely related to SLE. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population (SP) includes all participants for whom study intervention was initiated.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 50 52
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
36.Secondary Outcome
Title All-Cause Mortality
Hide Description All-cause mortality is defined as death from any cause occurring after randomization. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0.
Time Frame Baseline (Treatment Randomization) to Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population (SP) includes all participants for whom study intervention was initiated.
Arm/Group Title MMF Maintenance MMF Withdrawal
Hide Arm/Group Description:
Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60).
Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
Overall Number of Participants Analyzed 50 52
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
Time Frame 60 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Maintenance Withdrawal
Hide Arm/Group Description Participants received Mycophenolate Mofetil (MMF) treatment (1000-3000 mg/day) for the rest of their study participation (up to Week 60). Participants tapered off Mycophenolate Mofetil (MMF) per the protocol-specified schedule over 12 weeks and remained off MMF for the rest of their study participation (up to Week 60 or until the primary endpoint of disease reactivation was met, whichever came first).
All-Cause Mortality
Maintenance Withdrawal
Affected / at Risk (%) Affected / at Risk (%)
Total   0/50 (0.00%)      0/52 (0.00%)    
Hide Serious Adverse Events
Maintenance Withdrawal
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/50 (14.00%)      5/52 (9.62%)    
Blood and lymphatic system disorders     
Febrile neutropenia  1  1/50 (2.00%)  1 0/52 (0.00%)  0
Cardiac disorders     
Acute coronary syndrome  1  1/50 (2.00%)  1 0/52 (0.00%)  0
Myocardial ischaemia  1  2/50 (4.00%)  2 0/52 (0.00%)  0
Pericarditis  1  0/50 (0.00%)  0 1/52 (1.92%)  1
Gastrointestinal disorders     
Haematemesis  1  1/50 (2.00%)  1 0/52 (0.00%)  0
Pancreatitis  1  1/50 (2.00%)  1 0/52 (0.00%)  0
Infections and infestations     
Bronchitis  1  1/50 (2.00%)  1 0/52 (0.00%)  0
Mastoiditis  1  0/50 (0.00%)  0 1/52 (1.92%)  1
Pyelonephritis  1  1/50 (2.00%)  1 0/52 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Musculoskeletal pain  1  1/50 (2.00%)  1 0/52 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Breast cancer  1  0/50 (0.00%)  0 1/52 (1.92%)  1
Invasive ductal breast carcinoma  1  1/50 (2.00%)  1 0/52 (0.00%)  0
Psychiatric disorders     
Psychotic disorder  1  1/50 (2.00%)  1 0/52 (0.00%)  0
Renal and urinary disorders     
Lupus nephritis  1  0/50 (0.00%)  0 3/52 (5.77%)  3
Skin and subcutaneous tissue disorders     
Hidradenitis  1  1/50 (2.00%)  1 0/52 (0.00%)  0
1
Term from vocabulary, 16.1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Maintenance Withdrawal
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   38/50 (76.00%)      41/52 (78.85%)    
Blood and lymphatic system disorders     
Anaemia  1  2/50 (4.00%)  2 5/52 (9.62%)  6
Leukopenia  1  11/50 (22.00%)  17 13/52 (25.00%)  17
Lymphopenia  1  8/50 (16.00%)  10 8/52 (15.38%)  9
Neutropenia  1  5/50 (10.00%)  9 7/52 (13.46%)  9
Infections and infestations     
Bronchitis  1  1/50 (2.00%)  1 3/52 (5.77%)  3
Sinusitis  1  2/50 (4.00%)  3 3/52 (5.77%)  3
Tooth infection  1  1/50 (2.00%)  1 3/52 (5.77%)  3
Upper respiratory tract infection  1  14/50 (28.00%)  20 7/52 (13.46%)  11
Urinary tract infection  1  6/50 (12.00%)  9 10/52 (19.23%)  11
Investigations     
Blood creatinine increased  1  3/50 (6.00%)  4 3/52 (5.77%)  5
Glomerular filtration rate decreased  1  6/50 (12.00%)  8 2/52 (3.85%)  4
Metabolism and nutrition disorders     
Hypophosphataemia  1  5/50 (10.00%)  6 3/52 (5.77%)  3
Musculoskeletal and connective tissue disorders     
Arthralgia  1  4/50 (8.00%)  7 2/52 (3.85%)  2
Arthritis  1  1/50 (2.00%)  1 4/52 (7.69%)  4
Systemic lupus erythematosus  1  0/50 (0.00%)  0 4/52 (7.69%)  5
Nervous system disorders     
Headache  1  2/50 (4.00%)  3 5/52 (9.62%)  7
Renal and urinary disorders     
Acute prerenal failure  1  1/50 (2.00%)  1 3/52 (5.77%)  6
1
Term from vocabulary, 16.1
Indicates events were collected by systematic assessment
Enrollment ended early with 102 participants randomized instead of the planned 120.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Director, Clinical Research Operations Program
Organization: DAIT/NIAID
Phone: 301-594-7669
EMail: DAITClinicalTrialsGov@niaid.nih.gov
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: National Institute of Allergy and Infectious Diseases (NIAID)
ClinicalTrials.gov Identifier: NCT01946880    
Other Study ID Numbers: DAIT ALE06
NIAID CRMS ID#: 20154 ( Other Identifier: DAIT NIAID )
First Submitted: September 13, 2013
First Posted: September 20, 2013
Results First Submitted: July 3, 2020
Results First Posted: August 17, 2020
Last Update Posted: August 17, 2020