Nonmyeloablative Conditioning and Transplantation for Patients With Refractory Systemic Lupus Erythematosus (SLE)

Study Results

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-09-13

Study Completion Date

2017-03-29

Brief Summary

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The main goal of the study is to determine if bone marrow transplant (BMT) from a less specific pool of donors in combination with high dose cyclophosphamide can induce remission of refractory systemic lupus erythematosus.

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Detailed Description

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Systemic lupus erythematosus (SLE) is a devastating systemic autoimmune disease that predominantly affects young women, is more common in African-Americans than in whites, and results in poor quality of life. Lupus has no cure, and up to 90% of patients require corticosteroids for disease control. More than half of patients with lupus have permanent organ damage, much of which is either directly due to or increased by corticosteroids. To satisfactorily manage moderate-to-severe SLE, the investigators need effective treatments that will allow corticosteroid-sparing.

High-dose chemotherapy followed by autologous BMT or peripheral blood progenitor transplantation (PBSCT) has been proposed as a novel approach to treat severe autoimmune diseases. Allogeneic BMT is not currently utilized for the routine treatment of SLE because of the significant morbidity (GVHD) and mortality associated with the procedure.

The investigators have recently developed an approach to BMT using post-transplant cyclophosphamide that allows us to safely perform allogeneic BMT from matched, mismatched, unrelated or haploidentical donors. Transplant-related mortality, graft-failure and risk of GVHD have been very low with this approach. Furthermore, this approach allows us to greatly expand the donor pool since any patient shares exactly one HLA haplotype with each biological parent or child and half of siblings, an eligible haploidentical donor can be identified rapidly in nearly all cases.

This trial will employ a fludarabine + cyclophosphamide conditioning along with posttransplantation cyclophosphamide on for patients with refractory SLE. The purpose of this trial is to improve the salvage rate for patients with refractory SLE through a reformatting of the immune system.

Conditions

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Lupus Erythematosus Graft-versus-host Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Nonmyeloablative Conditioning and BMT

Nonmyeloablative conditioning with rabbit antithymocyte globulin, cyclophosphamide, fludarabine, and total body irradiation. Allogeneic bone marrow transplant on Day 0. Graft versus host disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and tacrolimus.

Group Type EXPERIMENTAL

Cyclophosphamide

Intervention Type DRUG

14.5 mg/kg/day on Days -6 and -5. 50 mg/kg/day on Days 3 and 4.

Fludarabine

Intervention Type DRUG

30 mg/m\^2/day on Days -6 through -2.

Tacrolimus

Intervention Type DRUG

Starting on Day 5. Dose will be adjusted according to blood levels.

Mycophenolate Mofetil

Intervention Type DRUG

15 mg/kg three times per day from Day 5 to Day 35.

Rabbit antithymocyte globulin

Intervention Type DRUG

0.5 mg/kg on Day -9. 2 mg/kg/day on Days -8 and -7.

Total body irradiation

Intervention Type RADIATION

200 centigray on Day -1.

Allogeneic bone marrow transplant

Intervention Type BIOLOGICAL

Infusion on Day 0.

Interventions

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Cyclophosphamide

14.5 mg/kg/day on Days -6 and -5. 50 mg/kg/day on Days 3 and 4.

Intervention Type DRUG

Fludarabine

30 mg/m\^2/day on Days -6 through -2.

Intervention Type DRUG

Tacrolimus

Starting on Day 5. Dose will be adjusted according to blood levels.

Intervention Type DRUG

Mycophenolate Mofetil

15 mg/kg three times per day from Day 5 to Day 35.

Intervention Type DRUG

Rabbit antithymocyte globulin

0.5 mg/kg on Day -9. 2 mg/kg/day on Days -8 and -7.

Intervention Type DRUG

Total body irradiation

200 centigray on Day -1.

Intervention Type RADIATION

Allogeneic bone marrow transplant

Infusion on Day 0.

Intervention Type BIOLOGICAL

Other Intervention Names

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Cytoxan Cy Fludara FK-506 Prograf Cellcept MMF Thymoglobulin BMT

Eligibility Criteria

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Inclusion Criteria

* Four or more American College of Rheumatology (ACR) criteria for the classification of SLE or 4 or more of the SLICE criteria
* Involvement of one or more of the following organ systems: renal, neurologic, hematologic, cardiac, pulmonary, gastrointestinal
* A lack of response to corticosteroids in moderate-to-high doses, and to either an equivalent degree of immunosuppression with azathioprine, methotrexate, cyclosporin, tacrolimus, belimumab, rituximab, mycophenolate mofetil, and/or appropriate other treatment
* Patients should be eligible for transplantation according to the BMT Policy Manual