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Acthar for Treatment of Proteinuria in Diabetic Nephropathy Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01601236
Recruitment Status : Completed
First Posted : May 17, 2012
Results First Posted : July 24, 2017
Last Update Posted : August 21, 2017
Sponsor:
Information provided by (Responsible Party):
Mallinckrodt

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Diabetic Nephropathy
Interventions Drug: Repository Corticotropin Injection
Drug: Placebo
Enrollment 34
Recruitment Details Initial recruitment target was 40 subjects.
Pre-assignment Details Adaptive design mandated closure of groups 5 & 6 with re-assignment of subjects to groups 3 & 4 if pre-defined tolerability criteria were met in 2 of first 6 patients.
Arm/Group Title Group 1: Acthar 8 U (0.1 mL) Daily Group 2: Placebo (0.1 mL) Daily Group 3: Acthar 16 U (0.2 mL) Daily Group 4: Placebo (0.2 mL) Daily Group 5: Acthar 32 U (0.4 mL) Daily Group 6: Placebo (0.4 mL) Daily
Hide Arm/Group Description H.P. Acthar Gel (repository corticotropin injection) administered via daily subcutaneous (SC) injection for 36 weeks Placebo: contains the same inactive ingredients as H.P. Acthar Gel without the active pharmaceutical ingredient (API)administered via daily SC injection for 36 weeks H.P. Acthar Gel (repository corticotropin injection) administered via daily SC injection for 36 weeks Placebo: contains the same inactive ingredients as H.P. Acthar Gel without the API administered via daily SC injection for 36 weeks H.P. Acthar Gel (repository corticotropin injection) administered via daily SC injection for 36 weeks Placebo: contains the same inactive ingredients as H.P. Acthar Gel without the API administered via daily SC injection for 36 weeks
Period Title: Overall Study
Started 7 3 14 6 3 1
Completed 2 2 8 4 0 0
Not Completed 5 1 6 2 3 1
Arm/Group Title Group 1: Acthar 8 U (0.1 mL) Daily Group 2: Placebo (0.1 mL) Daily Group 3: Acthar 16 U (0.2 mL) Daily Group 4: Placebo (0.2 mL) Daily Group 5: Acthar 32 U (0.4 mL) Daily Group 6: Placebo (0.4 mL) Daily Total
Hide Arm/Group Description

Repository Corticotropin Injection

Repository Corticotropin Injection: H.P. Acthar Gel (repository corticotropin injection) is administered via daily SC injection for 36 weeks in the three dose groups [8 U (0.1 mL), 16 U (0.2 mL), or 32 U (0.4 mL)].

Placebo

Placebo: Placebo contains the same inactive ingredients as H.P. Acthar Gel without the active pharmaceutical ingredient (API). Placebo is administered via daily SC injection for 36 weeks in equal volumes as the Acthar comparator volumes.

Repository Corticotropin Injection

Repository Corticotropin Injection: H.P. Acthar Gel (repository corticotropin injection) is administered via daily SC injection for 36 weeks in the three dose groups [8 U (0.1 mL), 16 U (0.2 mL), or 32 U (0.4 mL)].

Placebo

Placebo: Placebo contains the same inactive ingredients as H.P. Acthar Gel without the active pharmaceutical ingredient (API). Placebo is administered via daily SC injection for 36 weeks in equal volumes as the Acthar comparator volumes.

Repository Corticotropin Injection

Repository Corticotropin Injection: H.P. Acthar Gel (repository corticotropin injection) is administered via daily SC injection for 36 weeks in the three dose groups [8 U (0.1 mL), 16 U (0.2 mL), or 32 U (0.4 mL)].

Placebo

Placebo: Placebo contains the same inactive ingredients as H.P. Acthar Gel without the active pharmaceutical ingredient (API). Placebo is administered via daily SC injection for 36 weeks in equal volumes as the Acthar comparator volumes.

Total of all reporting groups
Overall Number of Baseline Participants 7 3 14 6 3 1 34
Hide Baseline Analysis Population Description
All randomized subjects
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 3 participants 14 participants 6 participants 3 participants 1 participants 34 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
6
  85.7%
2
  66.7%
9
  64.3%
5
  83.3%
2
  66.7%
0
   0.0%
24
  70.6%
>=65 years
1
  14.3%
1
  33.3%
5
  35.7%
1
  16.7%
1
  33.3%
1
 100.0%
10
  29.4%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 3 participants 14 participants 6 participants 3 participants 1 participants 34 participants
Female
4
  57.1%
0
   0.0%
9
  64.3%
1
  16.7%
1
  33.3%
0
   0.0%
15
  44.1%
Male
3
  42.9%
3
 100.0%
5
  35.7%
5
  83.3%
2
  66.7%
1
 100.0%
19
  55.9%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 7 participants 3 participants 14 participants 6 participants 3 participants 1 participants 34 participants
7 3 14 6 3 1 34
1.Primary Outcome
Title Percent Change in Estimated Glomerular Filtration Rate (eGFR) at Visit 12
Hide Description Percent change in eGFR at Visit 12 (Week 36) compared to average baseline eGFR obtained during screening
Time Frame Visit 12 (Week 36)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
This trial had an adaptive design that pre-specified the closure of the 32 U (units) (Group 5) in the event Acthar was not well tolerated at that dose. Based on tolerability, all patients initially included in the 32 U arm were combined with the 16 U arm (Group 3).
Arm/Group Title Placebo Acthar 8 Units Acthar 16 Units
Hide Arm/Group Description:
Groups 2, 4, 6
Group 1
Groups 3, 5
Overall Number of Participants Analyzed 10 7 16
Least Squares Mean (Standard Error)
Unit of Measure: percent change
-24.927  (5.4198) -20.563  (6.8126) -17.447  (4.3473)
2.Secondary Outcome
Title Percent Change in eGFR at Visit 17
Hide Description Percent change in eGFR at Visit 17 (Week 52) compared to baseline eGFR obtained during screening
Time Frame Visit 17 (Week 52)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
This trial had an adaptive design that pre-specified the closure of the 32 U arm (Group 5) in the event Acthar was not well tolerated at that dose. Based on tolerability, all patients initially included in the 32 U arm were combined with the 16 U arm (Group 3).
Arm/Group Title Placebo Acthar 8 Units Acthar 16 Units
Hide Arm/Group Description:
Groups 2, 4, 6
Group 1
Groups 3, 5
Overall Number of Participants Analyzed 8 6 10
Least Squares Mean (Standard Error)
Unit of Measure: percent change
-20.407  (6.0183) -37.456  (7.8387) -22.097  (5.4619)
3.Secondary Outcome
Title Frequency of Patients With a Doubling of Serum Creatinine, Progression to End-stage Renal Disease (ESRD), or Death
Hide Description [Not Specified]
Time Frame Visit 12 (Week 36) and Visit 17 (Week 52)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
This trial had an adaptive design that pre-specified the closure of the 32 U arm (Group 5) in the event Acthar was not well tolerated at that dose. Based on tolerability, all patients initially included in the 32 U arm were combined with the 16 U arm (Group 3).
Arm/Group Title Placebo Acthar 8 Units Acthar 16 Units
Hide Arm/Group Description:
Groups 2, 4, 6
Group 1
Groups 3, 5
Overall Number of Participants Analyzed 10 7 16
Measure Type: Count of Participants
Unit of Measure: Participants
Visit 12 (Week 36) Yes
0
   0.0%
1
  14.3%
1
   6.3%
No
10
 100.0%
6
  85.7%
15
  93.8%
Visit 17 (Week 52) Yes
0
   0.0%
1
  14.3%
2
  12.5%
No
10
 100.0%
6
  85.7%
14
  87.5%
4.Secondary Outcome
Title Time to Doubling of Serum Creatinine or ESRD or Death
Hide Description The frequency doubling of serum creatinine or progression to ESRD or death was small therefore this parameter could not be analyzed.
Time Frame Visit 12 (Week 36) or Visit 17 (Week 52)
Outcome Measure Data Not Reported
5.Secondary Outcome
Title Complete or Partial Remission of Proteinuria
Hide Description Proportion of patients with complete remission (PCR 0.5 g/g) or partial remission (reduction in PCR of >50% from baseline, plus PCR≤2.5 g/g but >0.5 g/g) of proteinuria at Visit 12 (Week 36) and/or at Visit 17 (Week 52)
Time Frame Visit 12 (Week 36) and Visit 17 (Week 52)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
This trial had an adaptive design that pre-specified the closure of the 32 U arm (Group 5) in the event Acthar was not well tolerated at that dose. Based on tolerability, all patients initially included in the 32 U arm were combined with the 16 U arm (Group 3).
Arm/Group Title Placebo Acthar 8 Units Acthar 16 Units
Hide Arm/Group Description:
Groups 2, 4, 6
Group 1
Groups 3, 5
Overall Number of Participants Analyzed 9 3 8
Measure Type: Count of Participants
Unit of Measure: Participants
Visit 12 (Week 36) Number Analyzed 9 participants 3 participants 8 participants
Complete remission
0
   0.0%
0
   0.0%
1
  12.5%
Partial remission
1
  11.1%
0
   0.0%
0
   0.0%
No remission
8
  88.9%
3
 100.0%
7
  87.5%
Visit 17 (Week 52) Number Analyzed 6 participants 2 participants 8 participants
Complete remission
0
   0.0%
0
   0.0%
1
  12.5%
Partial remission
0
   0.0%
0
   0.0%
0
   0.0%
No remission
6
 100.0%
2
 100.0%
7
  87.5%
6.Secondary Outcome
Title Percent Change in eGFR Calculated Using Cystatin C
Hide Description Percent change in eGFR calculated using cystatin C compared to baseline obtained at Visit 2.
Time Frame Visit 12 (Week 36) and Visit 17 (Week 52)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
This trial had an adaptive design that pre-specified the closure of the 32 U arm (Group 5) in the event Acthar was not well tolerated at that dose. Based on tolerability, all patients initially included in the 32 U arm were combined with the 16 U arm (Group 3).
Arm/Group Title Placebo Acthar 8 Units Acthar 16 Units
Hide Arm/Group Description:
Groups 2, 4, 6
Group 1
Groups 3, 5
Overall Number of Participants Analyzed 9 3 8
Mean (Standard Deviation)
Unit of Measure: percent change
Visit 12 (Week 36) Number Analyzed 9 participants 3 participants 8 participants
-14.25  (17.155) -11.49  (10.334) -10.24  (22.066)
Visit 17 (Week 52) Number Analyzed 6 participants 2 participants 8 participants
1.14  (21.155) -35.90  (1.509) -12.43  (20.522)
7.Secondary Outcome
Title Percent Change From Baseline in eGFR by Visit
Hide Description Percent change from baseline in eGFR by visit
Time Frame Visit 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16 and 17
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
This trial had an adaptive design that pre-specified the closure of the 32 U arm (Group 5) in the event Acthar was not well tolerated at that dose. Based on tolerability, all patients initially included in the 32 U arm were combined with the 16 U arm (Group 3).
Arm/Group Title Placebo Acthar 8 Units Acthar 16 Units
Hide Arm/Group Description:
Groups 2, 4, 6
Group 1
Groups 3, 5
Overall Number of Participants Analyzed 10 7 16
Mean (Standard Deviation)
Unit of Measure: percent change
Baseline to Visit 3 Number Analyzed 9 participants 7 participants 14 participants
-6.19  (16.494) 1.82  (11.347) 0.86  (11.659)
Baseline to Visit 4 Number Analyzed 10 participants 7 participants 14 participants
-11.76  (14.131) 4.39  (9.133) -2.69  (11.252)
Baseline to Visit 5 Number Analyzed 10 participants 5 participants 12 participants
-8.42  (14.942) -10.11  (9.614) -12.01  (14.395)
Baseline to Visit 6 Number Analyzed 9 participants 5 participants 11 participants
-12.73  (8.881) -9.36  (14.723) -14.20  (16.300)
Baseline to Visit 7 Number Analyzed 9 participants 5 participants 11 participants
-12.30  (6.888) -25.42  (10.079) -17.33  (21.629)
Baseline to Visit 8 Number Analyzed 8 participants 3 participants 10 participants
-11.64  (6.539) -27.46  (11.435) -17.58  (23.071)
Baseline to Visit 9 Number Analyzed 9 participants 3 participants 10 participants
-14.46  (8.535) -21.04  (8.414) -23.25  (18.344)
Baseline to Visit 10 Number Analyzed 8 participants 3 participants 8 participants
-15.75  (10.914) -28.73  (6.789) -20.57  (23.747)
Baseline to Visit 11 Number Analyzed 9 participants 3 participants 8 participants
-14.71  (12.052) -26.13  (3.041) -19.05  (20.996)
Baseline to Visit 12 Number Analyzed 9 participants 3 participants 8 participants
-26.09  (11.687) -23.54  (7.853) -18.32  (22.929)
Baseline to Week 36 Endpoint Number Analyzed 10 participants 7 participants 16 participants
-24.96  (11.581) -20.27  (15.818) -17.55  (19.655)
Baseline to Visit 13 Number Analyzed 8 participants 3 participants 7 participants
-17.94  (14.297) -29.37  (10.193) -23.46  (25.490)
Baseline to Visit 14 Number Analyzed 8 participants 2 participants 8 participants
-21.88  (11.695) -33.99  (20.993) -29.96  (18.970)
Baseline to Visit 15 Number Analyzed 7 participants 2 participants 7 participants
-17.40  (14.334) -38.05  (8.677) -19.95  (18.899)
Baseline to Visit 16 Number Analyzed 5 participants 2 participants 8 participants
-19.16  (18.686) -40.18  (5.668) -27.33  (16.709)
Baseline to Visit 17 Number Analyzed 6 participants 2 participants 8 participants
-18.43  (12.893) -42.70  (15.255) -24.55  (22.322)
Baseline to Week 52 Endpoint Number Analyzed 8 participants 6 participants 10 participants
-20.69  (12.433) -36.50  (11.573) -22.44  (20.669)
8.Secondary Outcome
Title Percent Change From Baseline in Protein to Creatinine Ratio (PCR)
Hide Description Percent change from baseline in PCR
Time Frame Visits 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16 and 17
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
This trial had an adaptive design that pre-specified the closure of the 32 U arm (Group 5) in the event Acthar was not well tolerated at that dose. Based on tolerability, all patients initially included in the 32 U arm were combined with the 16 U arm (Group 3).
Arm/Group Title Placebo Acthar 8 Units Acthar 16 Units
Hide Arm/Group Description:
Groups 2, 4, 6
Group 1
Groups 3, 5
Overall Number of Participants Analyzed 10 7 16
Mean (Standard Error)
Unit of Measure: percent change
Baseline to Visit 4 Number Analyzed 9 participants 6 participants 14 participants
0.3837  (31.26202) 44.7508  (78.75623) 32.5701  (71.34201)
Baseline to Visit 5 Number Analyzed 10 participants 5 participants 10 participants
-13.6630  (25.57771) 47.4429  (54.03221) 2.0594  (59.12062)
Baseline to Visit 6 Number Analyzed 9 participants 5 participants 8 participants
-8.3326  (36.44142) 72.1740  (85.20350) -1.6086  (47.69994)
Baseline to Visit 7 Number Analyzed 9 participants 5 participants 9 participants
2.4621  (43.42213) 55.9950  (42.92855) -10.6879  (50.13402)
Baseline to Visit 8 Number Analyzed 8 participants 3 participants 10 participants
12.6601  (51.03925) 53.2134  (43.68087) -8.5381  (65.06657)
Baseline to Visit 9 Number Analyzed 9 participants 3 participants 9 participants
27.1483  (60.72960) 82.7319  (67.83831) 3.6409  (54.51979)
Baseline to Visit 10 Number Analyzed 8 participants 1 participants 7 participants
8.2594  (45.36335) 18.9898 -6.7500  (64.47765)
Baseline to Visit 11 Number Analyzed 8 participants 3 participants 7 participants
15.1205  (92.21718) 69.8646  (63.88789) 0.4566  (71.78576)
Baseline to Visit 12 Number Analyzed 9 participants 3 participants 8 participants
21.3540  (98.45289) 59.8357  (55.64981) 14.4619  (66.58000)
Baseline to Week 36 Endpoint Number Analyzed 10 participants 7 participants 16 participants
19.1847  (93.07542) 34.8848  (25.06961) 50.2732  (85.87435)
Baseline to Visit 14 Number Analyzed 8 participants 2 participants 8 participants
1.1894  (44.40008) 11.7663  (5.63288) -16.4956  (52.71778)
Baseline to Visit 15 Number Analyzed 7 participants 2 participants 7 participants
0.6294  (34.89553) 4.3807  (6.70067) 0.0091  (81.37129)
Baseline to Visit 16 Number Analyzed 6 participants 1 participants 8 participants
0.0025  (47.25812) 21.7536 3.6555  (74.40718)
Baseline to Visit 17 Number Analyzed 6 participants 2 participants 8 participants
20.8644  (88.18113) 19.1625  (40.46023) -6.2356  (72.81798)
Baseline to Week 52 Endpoint Number Analyzed 8 participants 6 participants 9 participants
32.9735  (78.62621) 66.7698  (84.63637) 12.1049  (87.56153)
9.Secondary Outcome
Title Proportion of Subjects Whose Best Response Was Complete or Partial Remission of Proteinuria
Hide Description Proportion of subjects whose best response was complete remission (PCR 0.5 g/g) or partial remission (reduction in PCR of >50% from baseline, plus PCR≤2.5 g/g but >0.5 g/g) of proteinuria
Time Frame Visit 12 (Week 36) and Visit 17 (Week 52)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
This trial had an adaptive design that pre-specified the closure of the 32 U arm (Group 5) in the event Acthar was not well tolerated at that dose. Based on tolerability, all patients initially included in the 32 U arm were combined with the 16 U arm (Group 3).
Arm/Group Title Placebo Acthar 8 Units Acthar 16 Units
Hide Arm/Group Description:
Groups 2, 4, 6
Group 1
Groups 3, 5
Overall Number of Participants Analyzed 10 7 16
Measure Type: Count of Participants
Unit of Measure: Participants
Week 36 Number Analyzed 10 participants 7 participants 16 participants
Complete remission
0
   0.0%
0
   0.0%
1
   6.3%
Partial remission
1
  10.0%
0
   0.0%
3
  18.8%
No remission
9
  90.0%
7
 100.0%
12
  75.0%
Between Week 36 and Week 52 Number Analyzed 8 participants 2 participants 8 participants
Complete remission
0
   0.0%
0
   0.0%
1
  12.5%
Partial remission
0
   0.0%
0
   0.0%
2
  25.0%
No remission
8
 100.0%
2
 100.0%
5
  62.5%
10.Secondary Outcome
Title Percent Change From Baseline of Serum Total Cholesterol, Triglycerides, LDL, HDL, Lp(a), Albumin, and Cortisol
Hide Description Percent change from baseline of serum total cholesterol, triglycerides, LDL, HDL, Lp(a), albumin, and cortisol
Time Frame Visit 6, 9, 12, and 17
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
This trial had an adaptive design that pre-specified the closure of the 32 U arm (Group 5) in the event Acthar was not well tolerated at that dose. Based on tolerability, all patients initially included in the 32 U arm were combined with the 16 U arm (Group 3).
Arm/Group Title Placebo Acthar 8 Units Acthar 16 Units
Hide Arm/Group Description:
Groups 2, 4, 6
Group 1
Groups 3, 5
Overall Number of Participants Analyzed 10 7 16
Mean (Standard Deviation)
Unit of Measure: percent change
Total cholesterol - Baseline to Visit 6 Number Analyzed 9 participants 5 participants 11 participants
-7.2  (8.15) 34.1  (23.94) 10.1  (29.82)
Total cholesterol - Baseline to Visit 9 Number Analyzed 9 participants 3 participants 10 participants
13.9  (22.90) 20.3  (27.82) 14.2  (25.95)
Total cholesterol - Baseline to Visit 12 Number Analyzed 9 participants 3 participants 8 participants
3.4  (22.97) 11.1  (24.57) 11.1  (32.58)
Total cholesterol - Baseline to Week 36 Endpoint Number Analyzed 10 participants 5 participants 16 participants
3.9  (21.74) 26.4  (28.05) 12.9  (24.22)
Total cholesterol - Baseline to Visit 17 Number Analyzed 6 participants 2 participants 8 participants
6.3  (20.82) -12.1  (1.57) 9.6  (33.08)
Total cholesterol - Baseline to Week 52 Endpoint Number Analyzed 6 participants 6 participants 10 participants
6.3  (20.82) 26.8  (45.79) 9.4  (30.28)
Triglycerides - Baseline to Visit 6 Number Analyzed 9 participants 5 participants 11 participants
1.6  (32.24) 30.0  (34.97) 20.5  (58.06)
Triglycerides - Baseline to Visit 9 Number Analyzed 9 participants 3 participants 10 participants
18.4  (34.74) 7.9  (36.59) 31.0  (65.70)
Triglycerides - Baseline to Visit 12 Number Analyzed 9 participants 3 participants 8 participants
8.3  (40.39) -7.1  (25.12) 29.2  (61.09)
Triglycerides - Baseline to Week 36 Endpoint Number Analyzed 10 participants 5 participants 16 participants
8.9  (38.12) 48.8  (141.71) 19.9  (38.54)
Triglycerides - Baseline to Visit 17 Number Analyzed 6 participants 2 participants 8 participants
22.6  (62.47) -37.4  (18.78) 27.7  (63.00)
Triglycerides - Baseline to Week 52 Endpoint Number Analyzed 6 participants 6 participants 10 participants
22.6  (62.47) 33.5  (76.59) 18.2  (59.15)
LDL-C - Baseline to Visit 6 Number Analyzed 9 participants 5 participants 11 participants
-4.8  (28.04) 47.4  (35.40) 8.1  (42.39)
LDL-C - Baseline to Visit 9 Number Analyzed 9 participants 3 participants 10 participants
16.9  (33.30) 36.1  (50.37) 24.5  (48.65)
LDL-C - Baseline to Visit 12 Number Analyzed 9 participants 3 participants 8 participants
16.0  (45.78) 23.2  (45.40) 26.4  (74.60)
LDL-C - Baseline to Week 36 Endpoint Number Analyzed 10 participants 5 participants 16 participants
15.9  (43.16) 42.0  (48.66) 12.4  (54.63)
LDL-C - Baseline to Visit 17 Number Analyzed 6 participants 2 participants 8 participants
7.4  (23.12) -9.9  (12.73) 25.9  (86.13)
LDL-C - Baseline to Week 52 Endpoint Number Analyzed 6 participants 6 participants 10 participants
7.4  (23.12) 43.2  (66.57) 24.1  (76.69)
HDL-C - Baseline to Visit 6 Number Analyzed 9 participants 5 participants 11 participants
-3.2  (14.03) 11.8  (8.94) 18.1  (35.97)
HDL-C - Baseline to Visit 9 Number Analyzed 9 participants 3 participants 10 participants
0.0  (11.52) 9.8  (4.12) 3.4  (11.34)
HDL-C - Baseline to Visit 12 Number Analyzed 9 participants 3 participants 8 participants
3.7  (26.98) 11.4  (2.70) -6.9  (13.21)
HDL-C - Baseline to Week 36 Endpoint Number Analyzed 10 participants 5 participants 16 participants
2.8  (25.59) 6.7  (7.83) 19.0  (58.40)
HDL-C - Baseline to Visit 17 Number Analyzed 6 participants 2 participants 8 participants
-5.0  (7.15) 3.0  (12.26) -9.2  (15.91)
HDL-C - Baseline to Week 52 Endpoint Number Analyzed 6 participants 6 participants 10 participants
-5.0  (7.15) -0.2  (17.50) -3.2  (21.37)
Lp(a) - Baseline to Visit 6 Number Analyzed 9 participants 5 participants 11 participants
-7.2  (14.30) 35.9  (95.42) 22.7  (41.97)
Lp(a) - Baseline to Visit 9 Number Analyzed 9 participants 3 participants 10 participants
1.9  (20.24) 1.2  (9.32) 28.0  (67.60)
Lp(a) - Baseline to Visit 12 Number Analyzed 9 participants 3 participants 8 participants
6.1  (31.56) 23.3  (20.74) 10.7  (41.39)
Lp(a) - Baseline to Week 36 Endpoint Number Analyzed 10 participants 5 participants 16 participants
7.5  (30.07) 49.2  (85.05) 16.9  (48.51)
Lp(a) - Baseline to Visit 17 Number Analyzed 6 participants 2 participants 8 participants
28.5  (57.71) 19.9  (24.25) 21.7  (65.09)
Lp(a) - Baseline to Week 52 Endpoint Number Analyzed 6 participants 6 participants 10 participants
28.5  (57.71) 55.6  (85.65) 21.2  (58.11)
Serum albumin - Baseline to Visit 6 Number Analyzed 9 participants 5 participants 11 participants
-1.92  (4.848) -9.79  (7.256) -3.78  (9.721)
Serum albumin - Baseline to Visit 9 Number Analyzed 9 participants 3 participants 10 participants
-2.49  (6.685) -7.77  (8.638) -3.75  (10.749)
Serum albumin - Baseline to Visit 12 Number Analyzed 9 participants 3 participants 8 participants
-0.89  (6.798) -2.67  (14.597) -4.09  (4.507)
Serum albumin - Baseline to Week 36 Endpoint Number Analyzed 10 participants 7 participants 16 participants
-1.42  (6.629) -5.96  (10.357) -7.23  (6.083)
Serum albumin - Baseline to Visit 17 Number Analyzed 6 participants 2 participants 8 participants
-0.22  (9.002) 8.06  (15.119) -2.33  (5.898)
Serum albumin - Baseline to Week 52 Endpoint Number Analyzed 8 participants 6 participants 10 participants
-1.77  (8.822) -6.15  (14.379) -2.75  (6.207)
Cortisol - Baseline to Visit 6 Number Analyzed 9 participants 5 participants 11 participants
-5.86  (28.676) 45.08  (151.882) 180.70  (237.206)
Cortisol - Baseline to Visit 9 Number Analyzed 9 participants 3 participants 10 participants
-11.23  (22.791) 14.44  (26.399) 50.08  (135.687)
Cortisol - Baseline to Visit 12 Number Analyzed 9 participants 3 participants 8 participants
-9.44  (39.280) 17.72  (35.704) 52.77  (97.269)
Cortisol - Baseline to Week 36 Endpoint Number Analyzed 10 participants 7 participants 16 participants
-8.36  (37.189) 63.46  (125.208) 64.32  (187.125)
Cortisol - Baseline to Visit 17 Number Analyzed 6 participants 2 participants 8 participants
-13.11  (31.825) 84.26  (88.861) 7.09  (24.739)
Cortisol - Baseline to Week 52 Endpoint Number Analyzed 8 participants 6 participants 10 participants
-8.53  (28.727) 51.82  (91.328) 30.21  (89.880)
11.Other Pre-specified Outcome
Title Change in Mean HbA1c
Hide Description Change from baseline mean HbA1c (%) to Week 36
Time Frame Week 36
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
This trial had an adaptive design that pre-specified the closure of the 32 U arm (Group 5) in the event Acthar was not well tolerated at that dose. Based on tolerability, all patients initially included in the 32 U arm were combined with the 16 U arm (Group 3).
Arm/Group Title Placebo Acthar 8 Units Acthar 16 Units
Hide Arm/Group Description:
Groups 2, 4, 6
Group 1
Groups 3, 5
Overall Number of Participants Analyzed 9 3 8
Mean (Standard Deviation)
Unit of Measure: %HbA1c
-0.22  (0.694) -0.33  (1.429) -0.25  (0.729)
Time Frame Adverse event data displayed was collected during the 36 week treatment period.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo Acthar 8 Units Acthar 16 Units Overall
Hide Arm/Group Description Groups 2, 4, 6 Group 1 Groups 3, 5; This trial had an adaptive design that pre-specified the closure of the 32 U arm (Group 5) in the event Acthar was not well tolerated at that dose. Based on tolerability, all patients initially included in the 32 U arm were combined with the 16 U arm (Group 3). Thus, Adverse Events were not collected separately for these Arms. Groups 1, 2, 3, 4, 5, 6
All-Cause Mortality
Placebo Acthar 8 Units Acthar 16 Units Overall
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/10 (0.00%)   1/7 (14.29%)   0/17 (0.00%)   1/34 (2.94%) 
Show Serious Adverse Events Hide Serious Adverse Events
Placebo Acthar 8 Units Acthar 16 Units Overall
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/10 (20.00%)   1/7 (14.29%)   2/17 (11.76%)   5/34 (14.71%) 
Cardiac disorders         
Acute myocardial infarction  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Cardiac failure congestive  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Gastrointestinal disorders         
Gastric antral vascular ectasia  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Upper gastrointestinal haemorrhage  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Duodenitis  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Metabolism and nutrition disorders         
Hypoglycaemia  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Renal and urinary disorders         
Acute kidney injury  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Respiratory, thoracic and mediastinal disorders         
Dyspnoea  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Vascular disorders         
Deep vein thrombosis  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo Acthar 8 Units Acthar 16 Units Overall
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   10/10 (100.00%)   6/7 (85.71%)   14/17 (82.35%)   30/34 (88.24%) 
Blood and lymphatic system disorders         
Anaemia  1/10 (10.00%)  1/7 (14.29%)  1/17 (5.88%)  3/34 (8.82%) 
Iron deficiency anaemia  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Cardiac disorders         
Arrhythmia  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Atrial fibrillation  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Cardiogenic shock  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Eye disorders         
Retinopathy  1/10 (10.00%)  0/7 (0.00%)  1/17 (5.88%)  2/34 (5.88%) 
Cataract  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Eyelid oedema  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Gastrointestinal disorders         
Nausea  1/10 (10.00%)  0/7 (0.00%)  2/17 (11.76%)  3/34 (8.82%) 
Diarrhoea  1/10 (10.00%)  0/7 (0.00%)  1/17 (5.88%)  2/34 (5.88%) 
Gastritis erosive  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Gastrointestinal angiodysplasia  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Melaena  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Abdominal pain lower  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Abdominal tenderness  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Constipation  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Gastroesophageal reflux disease  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Impaired gastric emptying  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Vomitting  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
General disorders         
Oedema peripheral  2/10 (20.00%)  3/7 (42.86%)  8/17 (47.06%)  13/34 (38.24%) 
Fatigue  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Injection site bruising  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Influenza like illness  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Injection site pain  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Oedema  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Pain  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Pyrexia  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Immune system disorders         
Drug hypersensitivity  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Seasonal allergy  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Infections and infestations         
Upper respiratory tract infection  0/10 (0.00%)  1/7 (14.29%)  2/17 (11.76%)  3/34 (8.82%) 
Herpes zoster  2/10 (20.00%)  0/7 (0.00%)  0/17 (0.00%)  2/34 (5.88%) 
Bronchitis  1/10 (10.00%)  0/7 (0.00%)  1/17 (5.88%)  2/34 (5.88%) 
Fungal skin infection  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Influenza  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Mastitis  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Sinusitis  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Localised infection  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Cellulitis  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Impetigo  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Urinary tract infection  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Viral diarrhoea  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Injury, poisoning and procedural complications         
Contusion  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Laceration  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Meniscus injury  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Procedural pain  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Rib fracture  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Tibia fracture  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Wrist fracture  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Thermal burn  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Investigations         
Blood creatinine phosphokinase increased  1/10 (10.00%)  0/7 (0.00%)  2/17 (11.76%)  3/34 (8.82%) 
Blood pressure increased  0/10 (0.00%)  1/7 (14.29%)  1/17 (5.88%)  2/34 (5.88%) 
Weight increased  0/10 (0.00%)  1/7 (14.29%)  1/17 (5.88%)  2/34 (5.88%) 
Blood uric acid increased  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Blood triglycerides increased  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Albumin urine present  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Blood glucose increased  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Blood ketone body increased  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Protein urine present  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Urine protein/creatinine ratio increased  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Metabolism and nutrition disorders         
Hyperkalaemia  2/10 (20.00%)  0/7 (0.00%)  1/17 (5.88%)  3/34 (8.82%) 
Diabetes mellitus  1/10 (10.00%)  1/7 (14.29%)  1/17 (5.88%)  3/34 (8.82%) 
Hyperphosphataemia  1/10 (10.00%)  1/7 (14.29%)  0/17 (0.00%)  2/34 (5.88%) 
Metabolic acidosis  1/10 (10.00%)  1/7 (14.29%)  0/17 (0.00%)  2/34 (5.88%) 
Gout  1/10 (10.00%)  0/7 (0.00%)  1/17 (5.88%)  2/34 (5.88%) 
Hyperglycaemia  0/10 (0.00%)  1/7 (14.29%)  1/17 (5.88%)  2/34 (5.88%) 
Hypokalaemia  0/10 (0.00%)  0/7 (0.00%)  2/17 (11.76%)  2/34 (5.88%) 
Decreased appetite  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Musculoskeletal and connective tissue disorders         
Muscle spasms  2/10 (20.00%)  2/7 (28.57%)  1/17 (5.88%)  5/34 (14.71%) 
Back pain  1/10 (10.00%)  0/7 (0.00%)  2/17 (11.76%)  3/34 (8.82%) 
Pain in extremity  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Plantar fasciitis  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Flank pain  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Neck pain  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Osteoarthritis  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Nervous system disorders         
Headache  0/10 (0.00%)  0/7 (0.00%)  2/17 (11.76%)  2/34 (5.88%) 
Dysgeusia  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Migraine  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Sinus headache  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Lethargy  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Post-traumatic headache  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Psychiatric disorders         
Insomnia  0/10 (0.00%)  1/7 (14.29%)  1/17 (5.88%)  2/34 (5.88%) 
Irritability  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Stress  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Anxiety  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Depression  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Renal and urinary disorders         
Chronic kidney disease  0/10 (0.00%)  2/7 (28.57%)  1/17 (5.88%)  3/34 (8.82%) 
Proteinuria  1/10 (10.00%)  0/7 (0.00%)  1/17 (5.88%)  2/34 (5.88%) 
Dysuria  0/10 (0.00%)  0/7 (0.00%)  2/17 (11.76%)  2/34 (5.88%) 
Renal impairment  0/10 (0.00%)  0/7 (0.00%)  2/17 (11.76%)  2/34 (5.88%) 
Hydronephrosis  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Nephrolithiasis  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Nephropathy  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Renal failure  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Reproductive system and breast disorders         
Amenorrhoea  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Respiratory, thoracic and mediastinal disorders         
Cough  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Pleural effusion  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Respiratory tract congestion  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Skin and subcutaneous tissue disorders         
Dermatitis  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Erythema  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Hyperkeratosis  0/10 (0.00%)  1/7 (14.29%)  0/17 (0.00%)  1/34 (2.94%) 
Acne  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Hair growth abnormal  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Pruritus  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Rash generalised  0/10 (0.00%)  0/7 (0.00%)  1/17 (5.88%)  1/34 (2.94%) 
Vascular disorders         
Hypertension  1/10 (10.00%)  0/7 (0.00%)  2/17 (11.76%)  3/34 (8.82%) 
Aortic stenosis  1/10 (10.00%)  0/7 (0.00%)  0/17 (0.00%)  1/34 (2.94%) 
Groups 5 & 6 were closed when 2 of the 1st 4 subjects met predefined tolerability criteria. Enrollment terminated early at 34/40 subjects due to slow recruitment.16 of 34 subjects discontinued prior to collecting primary endpoint data at week 36.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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.
A sole participant institution shall not, without Sponsor’s prior written consent, independently publish or otherwise disclose any results of this multicenter study prior to a “multicenter” publication, or 12 months after completion of the study, whichever occurs first. Institution and Principal Investigator shall have the right to publish and present the results of Institution’s and Principal Investigator’s activities solely in accordance with the Sponsor’s written provisions.
Results Point of Contact
Name/Title: Lawrence Hill
Organization: Mallinckrodt
Phone: (908) 238-6370
Responsible Party: Mallinckrodt
ClinicalTrials.gov Identifier: NCT01601236     History of Changes
Other Study ID Numbers: QSC01-DN-01
First Submitted: May 15, 2012
First Posted: May 17, 2012
Results First Submitted: May 18, 2017
Results First Posted: July 24, 2017
Last Update Posted: August 21, 2017