NCT03263559

Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)

Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients With Symptomatic Sickle Cell Disease. (BMTCTN1507)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 95 (actual)
• Sponsor: Medical College of Wisconsin · Academic / Other
• Sex: All
• Age: 5 Years – 45 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD). Based on their age and entry criteria patients are stratified into two groups: (1) children with severe SCD; and (2) adults with severe SCD.

Detailed description

This study is designed as a Phase II multi-center trial to determine the feasibility of achieving a high rate of event-free survival (EFS) at 2 years post transplant using pre-conditioning hydroxyurea (HU) with a conditioning regimen that consists of a combination of Thymoglobulin/Cyclophosphamide/Fludarabine/Thiotepa with post-grafting high-dose cyclophosphamide in patients with severe SCD who have HLA-haploidentical donors. EFS is defined as survival without a qualifying event. This is a single arm study in which participants will be enrolled into one of two strata. The first stratum will be restricted to children who have stroke and 40 children will be enrolled in this stratum. The second stratum will consist of adult patients with severe sickle cell disease and 40 participants will be enrolled in this stratum.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
PROCEDURE	Haploidentical Bone Marrow Transplantation	Eligible patients with a first degree Human Leukocyte Antigen (HLA)- haploidentical donor will undergo Haploidentical bone marrow transplantation at Day 0 with non T-cell depleted bone marrow. For Graft-vs-Host Disease (GVHD) prophylaxis, patients will be given sirolimus and mycophenolate mofetil beginning on Day +5.
DRUG	Hydroxyurea	HU will be given daily at 30mg/kg from Day -70 through Day -10.
DRUG	Rabbit-ATG	Rabbit-ATG (rATG) will be given at 0.5mg/kg on Day -9, and at 2.0mg/kg on Day -8 and Day -7.
DRUG	Thiotepa	Thiotepa will be given at 10mg/kg on Day -7
DRUG	Fludarabine	Fludarabine will be given at 30mg/m2 from Day -6 to Day -2
DRUG	Cyclophosphamide	Cyclophosphamide will be given at 14.5mg/kg on Day -6 and Day -5, and at 50 mg/kg on Days +3 and +4.
RADIATION	Total Body Irradiation	Total Body Irradiation will be given at 200cGy on Day -1
DRUG	Mesna	Mesna will be given at 40mg/kg on Days +3 and +4

Timeline

• Start date: 2017-10-03
• Primary completion: 2025-01-29
• Completion: 2025-01-29
• First posted: 2017-08-28
• Last updated: 2025-04-27

Locations

32 sites across 1 country: United States

Regulatory

• FDA-regulated drug study