Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)

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.

Conditions

• Sickle Cell Disease

Interventions

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

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI)

  collaborator NIH

• National Cancer Institute (NCI)

  collaborator NIH

• Blood and Marrow Transplant Clinical Trials Network

  collaborator NETWORK

• National Marrow Donor Program

  collaborator OTHER

• Medical College of Wisconsin

  lead OTHER

Principal Investigators

• Mary Horowitz, MD · Center for International Blood and Marrow Transplant Research

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

5 Years

Max Age

45 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2017-10-03

Primary Completion

2025-01-29

Completion

2025-01-29

FDA Drug

Yes

Countries

• United States

Study Locations