NCT00040417

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

Allo SCT From HLA Haploidentical Related Donors Using Sub-Myeloablative Conditioning For Patients With High Risk Hemoglobinopathies: Hemo SS, Hemo SC, Hemo SB0/+ Thalassemia, Homozygous B0/+ Thalassemia or Severe B0/+ Thalassemia Variants

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (planned)
• Sponsor: Baylor College of Medicine · Academic / Other
• Sex: All
• Age: 1 Day – 65 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Sickle Cell Anemia
• Hemoglobinopathy
• Thalassemia

Interventions

Type	Name	Description
DRUG	FLUDARABINE
DRUG	CAMPATH-IH
PROCEDURE	Total Body Irradiation
DRUG	FK506
DRUG	G-SCF (Granulocyte-colony stimulating factor)

Timeline

• Start date: 2000-08-01
• Primary completion: 2003-11-21
• Completion: 2003-11-21
• First posted: 2002-06-27
• Last updated: 2020-01-18

Locations

2 sites across 1 country: United States