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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Steven Pavletic, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:
NCT00076752
First received: February 2, 2004
Last updated: April 9, 2017
Last verified: April 2017
Results First Received: March 25, 2014  
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

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Autologous HSCT in SLE

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.


Participant Flow:   Overall Study
    Autologous HSCT in SLE
STARTED   9 
COMPLETED   6 
NOT COMPLETED   3 
Death                2 
Withdrawal per PI, PI put study on hold                1 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Autologous HSCT in SLE

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.


Baseline Measures
   Autologous HSCT in SLE 
Overall Participants Analyzed 
[Units: Participants]
 9 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      1  11.1% 
Between 18 and 65 years      8  88.9% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 25.85  (7.67) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      7  77.8% 
Male      2  22.2% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Hispanic or Latino      1  11.1% 
Not Hispanic or Latino      8  88.9% 
Unknown or Not Reported      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of 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 
[Units: Participants]
 
United States   9 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Relapse-free Complete Clinical Response   [ Time Frame: 60 months ]

2.  Secondary:   Number of Participants With Adverse Events   [ Time Frame: 18 months ]

3.  Secondary:   Anti-Nuclear Antibody   [ Time Frame: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years. ]

4.  Secondary:   Extractable Nuclear Antigen (ENA)   [ Time Frame: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years. ]

5.  Secondary:   Anti-Double Stranded Deoxyribonucleic Acid (DNA) Antibody   [ Time Frame: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years. ]

6.  Secondary:   Anti-Smith-Ribonuclear Protein Antibody   [ Time Frame: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months and 2 years. ]

7.  Secondary:   White Blood Cells   [ Time Frame: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years. ]

8.  Secondary:   Absolute Neutrophil Count   [ Time Frame: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years. ]

9.  Secondary:   Absolute Lymphocyte Count   [ Time Frame: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years. ]

10.  Secondary:   Platelet Count   [ Time Frame: Day -7, day 0, 1 3, and 6 months, 1 year, 18 months, 2 years and 3 years. ]

11.  Secondary:   Cluster of Differentiation 3 (CD3) + Cells   [ Time Frame: Day 0, 1 month, 3 months, 6 months, 1 year and 2 years. ]

12.  Secondary:   Cluster of Differentiation 4 (CD4) + Cells   [ Time Frame: Day 0, 1 month, 3 months, 6 months, 1 year and 2 years. ]

13.  Secondary:   Cluster of Differentiation 8 (CD8) + Cells   [ Time Frame: Day 0, 1 month, 3 months, 6 months, 1 year and 2 years. ]

14.  Secondary:   Cluster of Differentiation 19 (CD19) + Cells   [ Time Frame: Day 0, 1 month, 3 months, 6 months, 1 year and 2 years. ]

15.  Secondary:   Natural Killer Cells   [ Time Frame: Day 0, 1 month, 3 months, 6 months, 1 year and 2 years. ]

16.  Secondary:   Systemic Lupus Erythematosus Disease Activity Index (SLEDAI)   [ Time Frame: Day -7, day 0, 1 month, 3 months, 6 months, 1 year, 18 months, 2 years and 3 years. ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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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
e-mail: pavletis@mail.nih.gov


Publications of Results:
Other Publications:

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
Study First Received: February 2, 2004
Results First Received: March 25, 2014
Last Updated: April 9, 2017