NCT02828592

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

A Study of T-Cell Replete, HLA-Mismatched Haploidentical Bone Marrow Transplantation With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Lacking HLA-Matched Related Donor

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Northside Hospital, Inc. · Academic / Other
• Sex: All
• Age: 1 Year – 75 Years
• Healthy volunteers: Not accepted

Summary

Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding. Patients with matched sibling donors for transplantation have a 80-90% chance of survival; however, a response rate with just immunosuppression for those patients lacking suitable HLA-matched related siblings is only 60%. With immunosuppression, only 1/3 of patients are cured, 1/3 are dependent on long term immunosuppression, and the other 1/3 relapse or develop a clonal disorder. Recent studies have shown that using a haploidentical donor for transplantation has good response rates and significantly lower rates of acute and chronic GVHD.

Detailed description

Mismatched haploidentical donors will be identified for patients with severe aplastic anemia. These patients will undergo a preparative regimen of Fludarabine/Cyclophosphamide/TBI followed by haploidentical bone marrow transplantation. Post-transplant Cyclophosphamide will be administered on Days 3 \& 4. Immunosuppression with Tacrolimus and MMF will begin on Day +5; MMF will be discontinued on Day +35 while Tacrolimus continues until Day +180. Investigators hypothesize that haploidentical transplantation with the above-mentioned preparative regimen will have a \<30% graft failure rate. The one-sided exact Binomial test at 5% significance level will be used to test this hypothesis. The size of 20 patients provides the power of 92.5% for confirming the 30-day graft failure rate \<30%.

Conditions

• Severe Aplastic Anemia

Interventions

Type	Name	Description
DRUG	Fludarabine	30 mg/m2 IV QD x 5 days (Days -6 to -2)
DRUG	Cyclophosphamide	14.5 mg/kg/day IV x 2 doses (Days -6 \& -5)
RADIATION	Total Body Irradiation	300 cGy x1 dose (Day -1)
DRUG	Rabbit ATG	1.5 mg/kg/day x 3 days (Days -3 to -1)
DRUG	Cyclophosphamide	Post-transplant: 50 mg/kg IV QD (Day +3 to +4)

Timeline

• Start date: 2016-09-09
• Primary completion: 2026-08-31
• Completion: 2027-08-31
• First posted: 2016-07-11
• Last updated: 2025-10-30

Locations

2 sites across 1 country: United States