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Trial record 16 of 712 for:    lupus AND Lupus Erythematosus, Systemic

Lymphocyte Depletion and Stem Cell Transplantation to Treat Severe Systemic Lupus Erythematosus

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ClinicalTrials.gov Identifier: NCT00076752
Recruitment Status : Completed
First Posted : February 3, 2004
Results First Posted : June 10, 2014
Last Update Posted : May 2, 2017
Sponsor:
Information provided by (Responsible Party):
Steven Pavletic, M.D., National Institutes of Health Clinical Center (CC)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Lupus Erythematosus, Systemic
Interventions Drug: fludarabine phosphate
Drug: cyclophosphamide
Biological: Rituxan (rituximab)
Biological: filgrastim
Drug: methylprednisolone
Other: immunologic technique
Other: laboratory biomarker analysis
Procedure: autologous hematopoietic stem cell transplantation
Drug: Diphenhydramine
Drug: Mesna
Enrollment 9

Recruitment Details  
Pre-assignment Details  
Arm/Group Title Autologous HSCT in SLE
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Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, and diphenhydramine. Stem cell transplant infusion day 0, product will be infused rapidly intravenously after premedication with diphenhydramine 25-60 mg orally or intravenous.

Period Title: Overall Study
Started 9
Completed 6
Not Completed 3
Reason Not Completed
Death             2
Withdrawal per PI, PI put study on hold             1
Arm/Group Title Autologous HSCT in SLE
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Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Baseline Participants 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
<=18 years
1
  11.1%
Between 18 and 65 years
8
  88.9%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 9 participants
25.85  (7.67)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Female
7
  77.8%
Male
2
  22.2%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Hispanic or Latino
1
  11.1%
Not Hispanic or Latino
8
  88.9%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
American Indian or Alaska Native
0
   0.0%
Asian
2
  22.2%
Native Hawaiian or Other Pacific Islander
1
  11.1%
Black or African American
3
  33.3%
White
3
  33.3%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 9 participants
9
1.Primary Outcome
Title Relapse-free Complete Clinical Response
Hide Description Complete clinical response is defined as complete clinical response in the target organ and no clinical signs of active lupus as determined by a Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) score of ≤3; prednisone ≤10mg/day at 6 months and ≤5mg/day at 12 months or later.
Time Frame 60 months
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Hide Analysis Population Description
Ninth participant was taken off study before proceeding with transplant per principal investigator due to decision to put study on hold.
Arm/Group Title Autologous HSCT in SLE
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Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, and diphenhydramine. Stem cell transplant infusion day 0, product will be infused rapidly intravenously after premedication with diphenhydramine 25-60 mg orally or intravenous.

Overall Number of Participants Analyzed 8
Median (Full Range)
Unit of Measure: Months
54
(36 to 60)
2.Secondary Outcome
Title Number of Participants With Adverse Events
Hide Description Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module.
Time Frame 18 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Autologous HSCT in SLE
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Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, and diphenhydramine. Stem cell transplant infusion day 0, product will be infused rapidly intravenously after premedication with diphenhydramine 25-60 mg orally or intravenous.

Overall Number of Participants Analyzed 9
Measure Type: Number
Unit of Measure: participants
9
3.Secondary Outcome
Title Anti-Nuclear Antibody
Hide Description Anti-Nuclear antibody is a well accepted biological clinical laboratory marker of systemic lupus. Range of normal values is 0-0.9 EU.
Time Frame Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant was enrolled but the study was closed before the patient could be treated.
Arm/Group Title Autologous HSCT in SLE
Hide Arm/Group Description:

Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: EU
Day -7 (n=8) 5.4  (4.65)
Day 0 (n=8) 4.7  (4.73)
1 month (n=8) 3.7  (3.89)
3 months (n=8) 3.2  (3.91)
6 months (n=7) 2.7  (3.01)
1 year (n=5) 2.6  (2.77)
18 months (n=5) 2.8  (2.7)
2 years (n=5) 2.5  (2.59)
3 years 9 (n=4) 2.5  (2.96)
4.Secondary Outcome
Title Extractable Nuclear Antigen (ENA)
Hide Description Extractable nuclear antigen is a well accepted biological clinical laboratory marker of systemic lupus. Range of normal values is 0-19.
Time Frame Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant was enrolled but the study was closed before the patient could be treated.
Arm/Group Title Autologous HSCT in SLE
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Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: EU
Day -7 (n=8) 66.9  (74.48)
Day 0 (n=8) 64.8  (70.61)
1 month (n=8) 61.5  (73.72)
3 months (n=8) 58.5  (67.03)
6 months (n=7) 51.3  (57.22)
1 year (n=5) 57.2  (58.07)
18 months (n=5) 60.6  (60.76)
2 years (n=5) 50.6  (49.04)
3 years (n=3) 26.3  (41.12)
5.Secondary Outcome
Title Anti-Double Stranded Deoxyribonucleic Acid (DNA) Antibody
Hide Description Anti-Double stranded deoxyribonucleic acid antibody is a well accepted biological clinical laboratory marker especially specific for systemic lupus. Range of normal values is 0-24 IU.
Time Frame Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant was enrolled but the study was closed before the patient could be treated.
Arm/Group Title Autologous HSCT in SLE
Hide Arm/Group Description:

Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: IU
Day -7 (n=8) 17.3  (24.92)
Day 0 (n=8) 8.8  (16.8)
1 month (n=8) 0  (0)
3 months (n=8) 0  (0)
6 months (n=7) 0  (0)
1 year (n=5) 0  (0)
18 months (n=5) 0  (0)
2 years (n=5) 0  (0)
3 years (n=5) 0  (0)
6.Secondary Outcome
Title Anti-Smith-Ribonuclear Protein Antibody
Hide Description Anti-Smith-Ribonuclear protein antibody is a well accepted biological clinical laboratory marker of systemic lupus.Range of normal values is 0-19 EU.
Time Frame Day -7, day 0, 1 3, and 6 months, 1 year, 18 months and 2 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Autologous HSCT in SLE
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Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: EU
Day -7 (n=5) 49  (49.51)
Day 0 (n=5) 51.6  (53.72)
1 month (n=4) 31.5  (40.31)
3 months (n=5) 29.8  (41.48)
6 months (n=4) 28.5  (40.07)
1 year (n=3) 20  (34.64)
18 months (n=3) 16.67  (28.87)
2 years (n=3) 25.67  (44.46)
7.Secondary Outcome
Title White Blood Cells
Hide Description The white blood cell test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 3.4-9.6 K/uL.
Time Frame Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant was enrolled but the study was closed before the patient could be treated.
Arm/Group Title Autologous HSCT in SLE
Hide Arm/Group Description:

Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: K/uL
Day -7 (n=8) 6.89  (3.07)
Day 0 (n=8) 0.47  (0.31)
1 month (n=8) 10.32  (6.6)
3 months (n=8) 3.84  (1.4)
6 months (n=7) 4.21  (1.38)
1 year (n=5) 5.81  (1.01)
18 months (n=5) 5.24  (0.82)
2 years (n=5) 7.17  (3.93)
3 years (n=5) 6.34  (1.08)
8.Secondary Outcome
Title Absolute Neutrophil Count
Hide Description The absolute neutrophil count test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 1.29-7.5 K/uL.
Time Frame Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant was enrolled but the study was closed before the patient could be treated.
Arm/Group Title Autologous HSCT in SLE
Hide Arm/Group Description:

Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: K/uL
Day -7 (n=8) 5.66  (2.88)
Day 0 (n=8) 0.45  (0.3)
1 month (n=8) 9.11  (6.67)
3 months (n=8) 2.72  (0.93)
6 months (n=7) 2.78  (1.38)
1 year (n=5) 3.44  (0.97)
18 months (n=5) 2.72  (1.04)
2 years (n=5) 4.04  (1.7)
3 years (n=5) 3.77  (0.63)
9.Secondary Outcome
Title Absolute Lymphocyte Count
Hide Description The absolute lymphocyte count test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 0.45-4.9 K/uL.
Time Frame Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant was enrolled but the study was closed before the patient could be treated.
Arm/Group Title Autologous HSCT in SLE
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Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: K/uL
Day -7 (n=8) 0.54  (0.4)
Day 0 (n=8) 0.0065  (0.0064)
1 month (n=8) 0.42  (0.18)
3 months (n=8) 0.53  (0.46)
6 months (n=7) 0.82  (0.31)
1 year (n=5) 1.75  (0.64)
18 months (n=5) 1.8  (0.61)
2 years (n=5) 1.8  (0.84)
3 years (n=5) 1.75  (0.56)
10.Secondary Outcome
Title Platelet Count
Hide Description The platelet count test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 162-380 K/uL.
Time Frame Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant was enrolled but the study was closed before the patient could be treated.
Arm/Group Title Autologous HSCT in SLE
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Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: K/uL
Day -7 (n=8) 251  (62.9)
Day 0 (n=8) 113  (49)
1 month (n=8) 166  (61.6)
3 months (n=8) 187  (95.4)
6 months (n=7) 170  (64)
1 year (n=5) 226  (47.9)
18 months (n=5) 210  (43.5)
2 years (n=5) 308  (251.5)
3 years (n=5) 272  (125.4)
11.Secondary Outcome
Title Cluster of Differentiation 3 (CD3) + Cells
Hide Description The CD3+Cells test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 650-2108 uL.
Time Frame Day 0, 1 month, 3 months, 6 months, 1 year and 2 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant was enrolled but the study was closed before the patient could be treated.
Arm/Group Title Autologous HSCT in SLE
Hide Arm/Group Description:

Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: cells/mL^3
Day 0 (n=8) 2.99  (3.25)
1 month (n=8) 239.47  (210.74)
3 months (n=8) 435.97  (335.69)
6 months (n=7) 699.47  (256.67)
1 year (n=6) 1493.29  (605.26)
2 years (n=5) 1678.76  (888.41)
12.Secondary Outcome
Title Cluster of Differentiation 4 (CD4) + Cells
Hide Description The CD4 + Cells test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 358-1259 uL.
Time Frame Day 0, 1 month, 3 months, 6 months, 1 year and 2 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant was enrolled but the study was closed before the patient could be treated.
Arm/Group Title Autologous HSCT in SLE
Hide Arm/Group Description:

Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: cells/mL^3
Day 0 (n=8) 2.58  (2.98)
1 month (n=8) 103.37  (117.43)
3 months (n=8) 112.8  (101.94)
6 months (n=7) 316.25  (193.01)
1 year (n=6) 702.87  (362.75)
2 years (n=5) 958.03  (735.04)
13.Secondary Outcome
Title Cluster of Differentiation 8 (CD8) + Cells
Hide Description The CD8 + Cells test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 194-836 u/L.
Time Frame Day 0, 1 month, 3 months, 6 months, 1 year and 2 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant was enrolled but the study was closed before the patient could be treated.
Arm/Group Title Autologous HSCT in SLE
Hide Arm/Group Description:

Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: cells/mL^3
Day 0 (n=8) 0.34  (0.34)
1 month (n=8) 138.68  (112.61)
3 months (n=8) 318.47  (259.28)
6 months (n=7) 334.91  (139.55)
1 year (n=6) 736.67  (532.19)
2 years (n=5) 674.69  (378.6)
14.Secondary Outcome
Title Cluster of Differentiation 19 (CD19) + Cells
Hide Description The CD19 + Cells test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 47-409 u/L.
Time Frame Day 0, 1 month, 3 months, 6 months, 1 year and 2 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant was enrolled but the study was closed before the patient could be treated.
Arm/Group Title Autologous HSCT in SLE
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Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: cells/mL^3
Day 0 (n=8) 0.01  (0.02)
1 month (n=8) 0.23  (0.32)
3 months (n=8) 6.7  (17.22)
6 months (n=7) 45.32  (58.21)
1 year (n=6) 142.69  (116.24)
3 years (n=5) 246.83  (316.53)
15.Secondary Outcome
Title Natural Killer Cells
Hide Description The natural killer cells test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Range of normal values is 87-505 uL.
Time Frame Day 0, 1 month, 3 months, 6 months, 1 year and 2 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant was enrolled but the study was closed before the patient could be treated.
Arm/Group Title Autologous HSCT in SLE
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Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: cells/mL^3
Day 0 (n=8) 0.03  (0.035)
1 month (n=8) 117.06  (81.76)
3 months (n=8) 116.57  (93.57)
6 months (n=7) 123.18  (63.32)
1 year (n=6) 158.9  (136.42)
3 years (n=5) 115.18  (68.3)
16.Secondary Outcome
Title Systemic Lupus Erythematosus Disease Activity Index (SLEDAI)
Hide Description The SLEDAI activity index test was performed to investigate immunological efficacy and mechanisms of response after lymphodepleting auto-hematopoietic stem cell transplant for systemic lupus erythematosus. Complete clinical response is defined as complete clinical response in the target organ and no clinical signs of active lupus as determined by a SLEDAI score of ≤3; partial response is at least 50% improvement in general disease activity as measured by SLEDAI. Remission is a SLEDAI score <3 and prednisone <10mg/day. 6+ indicates active disease requiring therapy. A score of 0 indicates a better outcome and a score greater then 6+ indicates a worse outcome.
Time Frame Day -7, day 0, 1 month, 3 months, 6 months, 1 year, 18 months, 2 years and 3 years.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant was enrolled but the study was closed before the patient could be treated.
Arm/Group Title Autologous HSCT in SLE
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Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: scores on a scale.
Day -7 (n=8) 4.25  (3.28)
Day 0 (n=8) 4.13  (4.49)
1 month (n=8) 3.63  (2.83)
3 months (n=5) 1.6  (3.58)
6 months (n=4) 0  (0)
1 year (n=4) 0  (0)
18 months (n=2) 0  (0)
2 years (n=2) 0  (0)
3 years (n=2) 0  (0)
Time Frame 18 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Autologous HSCT in SLE
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Autologous hematopoietic stem cell transplantation (HSCT) in systemic lupus erythematosus (SLE).

SLE is a chronic, inflammatory disease of the immune system. Participants received a priming, conditioning and transplant regimen. Priming regimen consisted of treatment with rituxan, filgrastim, cyclophosphamide, mesna, fludarabine phosphate, and methylprednisolone. Conditioning and transplant regimen consisted of fludarabine, cyclophosphamide, rituxan, filgrastim, mesna, diphenhydramine and stem cell transplant infusion.

All-Cause Mortality
Autologous HSCT in SLE
Affected / at Risk (%)
Total   6/9 (66.67%)    
Show Serious Adverse Events Hide Serious Adverse Events
Autologous HSCT in SLE
Affected / at Risk (%) # Events
Total   8/9 (88.89%)    
Cardiac disorders   
conduction abnormality/atrioventricular heart block: asystole  1  1/9 (11.11%)  1
hypertension  1  1/9 (11.11%)  1
Gastrointestinal disorders   
gastrointestinal-Other (Specify)  1 [1]  1/9 (11.11%)  1
General disorders   
Death Not Associated with CTCAE term: Death NOS  1  6/9 (66.67%)  6
Immune system disorders   
allergic reaction/hypersensitivity (including drug fever)  1  1/9 (11.11%)  1
Infections and infestations   
infection  1 [2]  1/9 (11.11%)  1
infection - Other (Specify, CMV antigenemia without neutropenia)  1  1/9 (11.11%)  1
Metabolism and nutrition disorders   
ALT/SGPT (serum glutamic pyruvic transaminase)  1  1/9 (11.11%)  1
AST/SGOT (serum glutamic oxaloacetic transaminase)  1  1/9 (11.11%)  1
potassium, serum-high (hyperkalemia)  1  1/9 (11.11%)  1
sodium, serum-low (hyponatremia)  1  1/9 (11.11%)  1
Nervous system disorders   
encephalopathy  1  1/9 (11.11%)  2
hydrocephalus  1  1/9 (11.11%)  2
neuropathy: motor  1  1/9 (11.11%)  1
neuropathy: sensory  1  1/9 (11.11%)  1
Respiratory, thoracic and mediastinal disorders   
hypoxia  1  2/9 (22.22%)  3
1
Term from vocabulary, CTCv3.0
Indicates events were collected by systematic assessment
[1]
laparoscopic surgery w/appendectomy
[2]
(documented clinically or microbiologically) with grades 3 or 4 neutrophils (ANC <1.0x10e9/L): lung (pneumonia)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Autologous HSCT in SLE
Affected / at Risk (%) # Events
Total   8/9 (88.89%)    
Blood and lymphatic system disorders   
Blood/Bone Marrow - Other (Specify, __)  1 [1]  1/9 (11.11%)  2
Bone marrow cellularity  1  1/9 (11.11%)  1
Hemoglobin  1  8/9 (88.89%)  55
Leukocytes (total WBC)  1  8/9 (88.89%)  82
Lymphopenia  1  8/9 (88.89%)  79
Neutrophils/granulocytes (ANC/AGC)  1  8/9 (88.89%)  81
Platelets  1  8/9 (88.89%)  130
PTT (Partial Thromboplastin Time)  1  4/9 (44.44%)  8
Thrombotic microangiopathy  1 [2]  1/9 (11.11%)  1
Hemorrhage/Bleeding - Other (Specify,bruising-skin)  1  1/9 (11.11%)  1
Petechiae/purpura (hemorrhage/bleeding into skin or mucosa)  1  2/9 (22.22%)  3
Edema: limb  1  1/9 (11.11%)  1
Cardiac disorders   
Supraventricular and nodal arrhythmia:: Atrial fibrillation  1  1/9 (11.11%)  1
Supraventricular and nodal arrhythmia:: Supraventricular arrhythmia NOS  1  1/9 (11.11%)  3
Ventricular arrhythmia:: PVCs  1  1/9 (11.11%)  1
Ventricular arrhythmia:: Ventricular tachycardia  1  1/9 (11.11%)  1
Cardiac General - Other (Specify, __)  1 [3]  1/9 (11.11%)  7
Cardiac troponin I (cTnI)  1  1/9 (11.11%)  1
Hypertension  1  1/9 (11.11%)  1
Hypotension  1  4/9 (44.44%)  4
Pericardial effusion (non-malignant)  1  1/9 (11.11%)  1
Ear and labyrinth disorders   
Auditory/Ear - Other (Specify, __)  1 [4]  1/9 (11.11%)  1
Pain:: External ear  1  1/9 (11.11%)  1
Endocrine disorders   
Cushingoid appearance (e.g., moon face, buffalo hump, centripetal obesity, cutaneous striae)  1  1/9 (11.11%)  1
Masculinization of female  1  1/9 (11.11%)  1
Eye disorders   
Ocular/Visual - Other (Specify, __)  1 [5]  2/9 (22.22%)  2
Retinopathy  1  1/9 (11.11%)  2
Vision-blurred vision  1  4/9 (44.44%)  4
Vision-photophobia  1  1/9 (11.11%)  1
Pain:: Eye  1  2/9 (22.22%)  2
Gastrointestinal disorders   
Anorexia  1  1/9 (11.11%)  1
Constipation  1  2/9 (22.22%)  2
Diarrhea  1  4/9 (44.44%)  5
Hemorrhoids  1  1/9 (11.11%)  1
Mucositis/stomatitis (clinical exam):: Oral cavity  1  1/9 (11.11%)  1
Nausea  1  5/9 (55.56%)  9
Taste alteration (dysgeusia)  1  2/9 (22.22%)  2
Vomiting  1  4/9 (44.44%)  8
Pain:: Abdomen NOS  1  2/9 (22.22%)  2
General disorders   
Constitutional Symptoms - Other (Specify,decreased appetite )  1  1/9 (11.11%)  1
Fatigue (asthenia, lethargy, malaise)  1  2/9 (22.22%)  2
Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L)  1  3/9 (33.33%)  8
Rigors/chills  1  1/9 (11.11%)  1
Pain - Other (Specify, __)  1 [6]  1/9 (11.11%)  2
Hepatobiliary disorders   
Cholecystitis  1  1/9 (11.11%)  1
Immune system disorders   
Allergic reaction/hypersensitivity (including drug fever)  1  1/9 (11.11%)  1
Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip)  1  1/9 (11.11%)  1
Infections and infestations   
Colitis, infectious (e.g., Clostridium difficile)  1  1/9 (11.11%)  1
Febrile neutropenia  1 [7]  3/9 (33.33%)  4
Infection  1 [8]  1/9 (11.11%)  1
Infection  1 [9]  1/9 (11.11%)  1
Infection - Other (Specify, __)  1 [10]  2/9 (22.22%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils:: Abdomen NOS  1  1/9 (11.11%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils:: Blood  1  4/9 (44.44%)  8
Infection with normal ANC or Grade 1 or 2 neutrophils:: Bone (osteomyelitis)  1  1/9 (11.11%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils:: Catheter-related  1  2/9 (22.22%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils:: Colon  1  1/9 (11.11%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils:: Conjunctiva  1  2/9 (22.22%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils:: Eye NOS  1  1/9 (11.11%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils:: Lung (pneumonia)  1  2/9 (22.22%)  3
Infection with normal ANC or Grade 1 or 2 neutrophils:: Pharynx  1  1/9 (11.11%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils:: Sinus  1  1/9 (11.11%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils:: Small bowel NOS  1  1/9 (11.11%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils:: Upper airway NOS  1  1/9 (11.11%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils:: Urinary tract NOS  1  3/9 (33.33%)  4
Infection with unknown ANC:: Middle ear (otitis media)  1  1/9 (11.11%)  1
Infection with unknown ANC:: Urinary tract NOS  1  1/9 (11.11%)  1
Metabolism and nutrition disorders   
ALT, SGPT (serum glutamic pyruvic transaminase)  1  5/9 (55.56%)  36
AST, SGOT(serum glutamic oxaloacetic transaminase)  1  5/9 (55.56%)  41
Albumin, serum-low (hypoalbuminemia)  1  5/9 (55.56%)  31
Alkaline phosphatase  1  4/9 (44.44%)  25
Amylase  1  3/9 (33.33%)  6
Bicarbonate, serum-low  1  3/9 (33.33%)  8
Bilirubin (hyperbilirubinemia)  1  1/9 (11.11%)  6
Calcium, serum-high (hypercalcemia)  1  1/9 (11.11%)  2
Calcium, serum-low (hypocalcemia)  1  8/9 (88.89%)  67
Cholesterol, serum-high (hypercholesteremia)  1  1/9 (11.11%)  1
Creatinine  1  3/9 (33.33%)  9
Glucose, serum-high (hyperglycemia)  1  5/9 (55.56%)  24
Glucose, serum-low (hypoglycemia)  1  1/9 (11.11%)  5
Hemoglobinuria  1  3/9 (33.33%)  16
Lipase  1  2/9 (22.22%)  5
Magnesium, serum-high (hypermagnesemia)  1  3/9 (33.33%)  13
Magnesium, serum-low (hypomagnesemia)  1  4/9 (44.44%)  10
Phosphate, serum-low (hypophosphatemia)  1  7/9 (77.78%)  86
Potassium, serum-high (hyperkalemia)  1  5/9 (55.56%)  16
Potassium, serum-low (hypokalemia)  1  5/9 (55.56%)  14
Proteinuria  1  1/9 (11.11%)  1
Sodium, serum-high (hypernatremia)  1  1/9 (11.11%)  1
Sodium, serum-low (hyponatremia)  1  4/9 (44.44%)  8
Uric acid, serum-high (hyperuricemia)  1  2/9 (22.22%)  6
Musculoskeletal and connective tissue disorders   
Fracture  1  1/9 (11.11%)  1
Osteoporosis  1  2/9 (22.22%)  2
Pain:: Back  1  5/9 (55.56%)  7
Pain:: Bone  1  5/9 (55.56%)  7
Pain:: Chest wall  1  3/9 (33.33%)  4
Pain:: Chest/thorax NOS  1  4/9 (44.44%)  4
Pain:: Muscle  1  1/9 (11.11%)  2
Pain:: Neck  1  3/9 (33.33%)  6
Nervous system disorders   
Dizziness  1  2/9 (22.22%)  2
Extrapyramidal/involuntary movement/restlessness  1  1/9 (11.11%)  2
Memory impairment  1  1/9 (11.11%)  1
Mood alteration:: Agitation  1  1/9 (11.11%)  1
Mood alteration:: Anxiety  1  1/9 (11.11%)  1
Mood alteration:: Depression  1  1/9 (11.11%)  1
Neuropathy: sensory  1  2/9 (22.22%)  2
Pain:: Head/headache  1  5/9 (55.56%)  5
Renal and urinary disorders   
Cystitis  1  1/9 (11.11%)  1
Respiratory, thoracic and mediastinal disorders   
Throat/pharynx/larynx  1  2/9 (22.22%)  2
Carbon monoxide diffusion capacity (DL(co))  1  1/9 (11.11%)  2
Cough  1  4/9 (44.44%)  5
Dyspnea (shortness of breath)  1  5/9 (55.56%)  9
Hypoxia  1  4/9 (44.44%)  4
Pleural effusion (non-malignant)  1  4/9 (44.44%)  4
Pneumonitis/pulmonary infiltrates  1  2/9 (22.22%)  3
Pulmonary/Upper Respiratory - Other (Specify, __)  1 [11]  1/9 (11.11%)  2
Skin and subcutaneous tissue disorders   
Bruising (in absence of Grade 3 or 4 thrombocytopenia)  1  2/9 (22.22%)  2
Dermatology/Skin - Other (Specify, scalp dermatitis)  1  1/9 (11.11%)  1
Dry skin  1  1/9 (11.11%)  1
Hair loss/alopecia (scalp or body)  1  3/9 (33.33%)  3
Pruritus/itching  1  2/9 (22.22%)  3
Rash/desquamation  1  1/9 (11.11%)  3
Skin breakdown/decubitus ulcer  1  1/9 (11.11%)  1
Ulceration  1  1/9 (11.11%)  1
1
Term from vocabulary, CTCv3.0
Indicates events were collected by systematic assessment
[1]
low HGB (anemia); low HGB
[2]
(e.g., thrombotic thrombocytopenic purpura [TTP] or hemolytic uremic syndrome [HUS])
[3]
2+L leg edema; 2+ pitting R leg edema; 3+ pitting L leg edema; 3+ pitting edema R leg; intermittent 2+, 3+ pitting b/l LE edema
[4]
hearing diminished on the left side
[5]
residual itching post herpetic area; episcleritis
[6]
b/l flanks; intermittent pain in hips and knees
[7]
(fever of unknown origin without clinically or microbiologically documented infection)(ANC <1.0 x 10e9/L, fever >=38.5 degrees C)
[8]
(documented clinically or microbiologically) with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L):: Bladder (urinary)
[9]
(documented clinically or microbiologically) with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L):: Urinary tract NOS
[10]
R toe infection, was believed fungal; clostridium difficile in stool
[11]
nasal congestion; nasal drainage
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
Name/Title: Dr. Steven Pavletic
Organization: National Cancer Institute, National Institutes of Health
Phone: 301-402-4899
Responsible Party: Steven Pavletic, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT00076752     History of Changes
Obsolete Identifiers: NCT00726518
Other Study ID Numbers: 040095
04-C-0095
First Submitted: February 2, 2004
First Posted: February 3, 2004
Results First Submitted: March 25, 2014
Results First Posted: June 10, 2014
Last Update Posted: May 2, 2017