NCT02080195

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

A Phase I/II Study of Nonmyeloablative Conditioning and Transplantation of Human Leukocyte Antigen (HLA)-Matched, Partially HLA-mismatched, HLA-haploidentical or Matched Unrelated Bone Marrow for Patients With Refractory SLE

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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

• Lupus Erythematosus
• Graft-versus-host Disease

Interventions

Type	Name	Description
DRUG	Cyclophosphamide	14.5 mg/kg/day on Days -6 and -5. 50 mg/kg/day on Days 3 and 4.
DRUG	Fludarabine	30 mg/m\^2/day on Days -6 through -2.
DRUG	Tacrolimus	Starting on Day 5. Dose will be adjusted according to blood levels.
DRUG	Mycophenolate Mofetil	15 mg/kg three times per day from Day 5 to Day 35.
DRUG	Rabbit antithymocyte globulin	0.5 mg/kg on Day -9. 2 mg/kg/day on Days -8 and -7.
RADIATION	Total body irradiation	200 centigray on Day -1.
BIOLOGICAL	Allogeneic bone marrow transplant	Infusion on Day 0.

Timeline

• Start date: 2016-09-13
• Primary completion: 2017-03-28
• Completion: 2017-03-29
• First posted: 2014-03-06
• Last updated: 2019-10-02
• Results posted: 2018-10-02

Locations

1 site across 1 country: United States