NCT00040469

Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies

Allogeneic Bone Marrow Transplant From HLA Identical Related Donors for Patients With High Risk Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, Hemoglobin SB0/+ Thalassemia, or Homozygous B0/+ Thalassemia or Severe Variants of B0/+ Thalassemia

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (planned)
• Sponsor: Baylor College of Medicine · Academic / Other
• Sex: All
• Age: 1 Day – 64 Years
• Healthy volunteers: Not accepted

Summary

The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years.

Detailed description

To do the bone marrow transplant, we must first kill the cells in the bone marrow that make the abnormal red blood cells that are found in patients with severe thalassemia or sickle cell disease. We will do this by using three drugs: busulfan, cyclophosphamide, and CAMPATH-1H. CAMPATH-IH is an investigational drug. CAMPATH-1H is used to prevent participants from rejecting or refusing to let the donor blood cells grow in the body. After the drug treatment, participants will be given bone marrow from a brother or sister who has healthy bone marrow that matches.

Conditions

• Sickle Cell Anemia
• Hemoglobinopathy
• Thalassemia

Interventions

Type	Name	Description
DRUG	Campath -1H
DRUG	Dilantin
DRUG	Busulfan
DRUG	Cyclophosphamide

Timeline

• Start date: 2000-08-01
• Primary completion: 2003-11-21
• Completion: 2003-11-21
• First posted: 2002-06-28
• Last updated: 2020-01-18

Locations

2 sites across 1 country: United States