Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies

Phase 2

Terminated

• Conditions: Sickle Cell Anemia; Hemoglobinopathy; Thalassemia

• Registration Number: NCT00040417
• Lead Sponsor: Baylor College of Medicine

Brief Summary

The major goal of this study is to determine the risks and benefits of stem cell transplants in combination with a newer, less toxic conditioning chemotherapy treatment in patients with severe sickle cell disease (SCD) or sickle hemoglobin variants (hemoglobin SC or hemoglobin SB0/+), or homozygous b0/+ thalassemia or severe B0/+ thalassemia variants. Participation in this project will be for one year, with follow up evaluations done every 6 months thereafter for 10 years or until participants are 18 years old.

Detailed Description

To do the stem cell transplant, we must first kill most of the cells in the bone marrow that make the sickle hemoglobin or abnormal blood cells of severe beta thalassemia. We will do this by using a single dose of body irradiation and two drugs called Fludarabine and Campath-IH.

The treatment schedule is as follows:

Day - 6: Total body irradiation Day - 5: Fludarabine and Campath 1H Day - 4: Fludarabine and Campath 1H Day - 3: Fludarabine and Campath 1H Day - 2: Fludarabine and Campath 1H Day - 1: REST Day 0: Stem Cell Transplant (infusion)

After the drug treatment, participants will be given healthy stem cells from a related donor that partially matches their HLA (immune) type, most likely from a parent or sibling. This is known as the stem cell transplant.

The healthy stem cells will be put into a blood vein in the same way that transfusions are given. The cells then travel to the right places in the body, where they should grow and make new blood cells that do not sickle.

Study Timeline

• Study Start: 2000-08
• Study First Submitted: 2002-06-26
• Study First Submitted QC: 2002-06-26
• Study First Posted: 2002-06-27
• Primary Completion: 2003-11-21
• Study Completion: 2003-11-21
• Status Verified: 2020-01
• Last Update Submitted: 2020-01-15
• Last Update Posted: 2020-01-18

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Trial Locations

Locations (2)

Texas Children's Hospital; 🇺🇸 Houston, Texas, United States

The Methodist Hospital; 🇺🇸 Houston, Texas, United States