Allogeneic Stem Cell Transplantation in Systemic Lupus Erythematosus

NCT00278590 | Withdrawn Phase 1

• 1 other identifier: DI SLE.Allo2004
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

This trial is designed to evaluate the safety of treating systemic lupus erythematosus participants with cyclophosphamide and CAMPATH-1H followed by allogeneic stem cell transplant. There will be no randomization in this study. All subjects who are determined to be eligible for the study treatment will receive cyclophosphamide and CAMPATH-1H followed by allogeneic stem cell transplant. The purpose of the intense chemotherapy is to destroy the cells in the immune system which may be causing this disease. The purpose of the stem cell infusion is to produce a normal immune system that will no longer attack body. The study purpose is to examine whether this treatment will result in improvement in the lupus disease.

Conditions

Systemic Lupus Erythematosus

Outcome Measures

Primary Outcomes (1)

• Survival, disease free survival, number of relapses, organ function

  Evaluation of response will be performed for 5 years.

Study Arms (1)

allogeneic stem cell transplantation

EXPERIMENTAL

allogeneic stem cell transplantation will be performed

Biological: Allogeneic Stem Cell Transplantation

Interventions

Allogeneic Stem Cell Transplantation BIOLOGICAL

Allogeneic stem cell transplantation will be performed.

allogeneic stem cell transplantation

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Ages 18 to 50 years old.
• Meet at least 4 of 11 American College of Rheumatology (ACR) Classification criteria for SLE (see Appendix 1).
• Able to give informed consent.
• HLA matched sibling donor available.
• Meet one of following three:
• For lupus nephritis, participants must fail pulse cyclophosphamide (500 to 1000 mg/m2 monthly for a minimum of 6 months). Failure is defined as meeting criteria to be considered as BILAG (Appendix 4) renal category A. If indication for HSCT is nephritis, a renal biopsy must be obtained and document either class III or IV glomerulonephritis.
• For visceral organ involvement other than nephritis, participants must be BILAG cardiovascular/respiratory category A, vasculitis category A, or neurologic category A and must fail at least 3 months of oral or IV cyclophosphamide and be corticosteroid dependent. Steroid dependence being defined as at least 3 months of steroid therapy and inability to wean corticosteroid to less than 20 mg/day of prednisone or equivalent.
• For cytopenias that are immune mediated, participants must be BILAG hematologic category A. Participants must have an inability to maintain platelets \> 15,000, an inability to prevent active bleeding without transfusion, an inability to maintain hemoglobin \> 7.0, or an inability to prevent cardiovascular disease without transfusion. In addition, participants must fail corticosteroids (either oral prednisone \> 0.5 mg/kg/day for more than 6 months or pulse methylprednisolone for at least one cycle of three days), be refractory to IVIG, and at least one of the following: azathioprine at 2 mg/kg/day for at least 3 months, mycophenolate mofetil 2 grams daily for more than 3 months, cyclophosphamide intravenously or orally for at least 3 months, or cyclosporine at least 3 mg/kg/day for at least 3 months, danazol for at least 3 months, or splenectomy.

You may not qualify if:

• HIV positive.
• Ongoing malignancy except localized basal cell or squamous skin cancer. Other malignancies for which the participant is judged to be cured by local surgical therapy, such as head and neck cancer, or stage I or II breast cancer will be considered on an individual basis by the investigators doing the final screening for participant qualification.
• Positive pregnancy test, inability or unwillingness to pursue effective means of birth control, failure to willingly accept or comprehend irreversible sterility as a side effect of therapy.
• Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible.
• DLCO \< 45% of predicted unless attributed solely to active lupus.
• Resting LVEF \< 40% unless due to active lupus.
• Known hypersensitivity to E. Coli derived proteins.
• Transaminases greater than 2 times normal unless due to active lupus.
• Any illness that in the opinion of the investigator would jeopardize the ability of the patient to tolerate this treatment.
• Donor must be a HLA identical sibling or HLA matched cord blood donor.
• If donor is HLA matched sibling, donor must be \> 18 years of age and less than 50years old.
• If multiple HLA matched donors are available, preference will be given to samesex, same CMV status, and nulliparous donor, or in the case of cord blood higher nucleated cell count.
• If donor is HLA matched cord blood, cord blood stem cells will be obtained from the New York Blood Center Cord Blood Registry (Tel 212-570-3230) which is an internationally recognized registry or, if a match is not available, from Stemcyte (626-821-9860) which is a commercial registry that specializes in minority donors or from National Marrow Donor Program (NMDP). One unit of HLA matched cord blood unit will be infused on day zero.
• Physiologic age \> 50 years old or \<18 years old.
• HIV positive.

Sponsors & Collaborators

• Northwestern University: lead

Study Sites (1)

Northwestern University, Feinberg School of Medicine

Chicago, Illinois, 60611, United States

Location

MeSH Terms

Conditions

Lupus Erythematosus, Systemic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Richard Burt, MD

  Northwestern University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: January 16, 2006
• First Posted: January 18, 2006
• Study Start: July 1, 2004
• Primary Completion: July 1, 2015
• Study Completion: July 1, 2015
• Last Updated: January 13, 2017

Record last verified: 2017-01

Locations

US (1)