Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP
Patients are being asked to participate in this study because they have severe sickle cell anemia (SCD) with or without the beta thalassemia trait. Sickle cell anemia is an illness where the red blood cells change shape and can clog up blood vessels. This keeps the body from getting the oxygen it needs. Thalassemia is when the body does not make enough hemoglobin, something that helps the oxygen get to the places it needs to go in the body. The patient may or may not need to get regular blood transfusions (getting more blood) to improve their quality of life (feel better) and prevent organ damage (problems with the brain, heart, lung, kidney, and gonad, for example.). The transfusions can also cause problems, including iron overload (too much iron in the blood), which can be fatal (patients can die) without regular deferoxamine shots. Even with the best usual treatments, people with thalassemia or SCD die sooner. There is no proven cure.
We would like to treat patients using bone marrow transplantation, a treatment that has been used for people with SCD. The transplant uses healthy "matched" bone marrow. This comes from a brother or sister who does not have sickle cell disease or severe thalassemia. If the treatment works, the sickle cell disease or thalassemia may be cured. This treatment has been used to treat patients with sickle cell disease or thalassemia. It has worked in most cases. We hope, but cannot promise, that the transplanted marrow will make healthy cells, and patients will not have sickle cell disease or severe thalassemia anymore.
We do not know what effect this treatment will have on the damage that has already been done by the disease. Finding that out is the main reason for this study. Currently, very little has been reported about organ function after bone marrow transplants in patients with sickle cell anemia.
Sickle Cell Disease
Biological: Campath 1H
Drug: Cyclophosphamide and MESNA
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Allogeneic Bone Marrow Transplantation From HLA Identical Related Donors for Patients With Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, or Hemoglobin SB0/+ Thalassemia|
- Evaluate recovery of organ function in patients with sickle cell disease (SCD) or sickle hemoglobin variants after undergoing allogeneic SCT/BMT from HLA genotype identical donors and if they can be improved or reversed. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
- Evaluate the use of PET scan examination in assessing metabolic function of organs in patients with SCD, hemoglobin SC, or hemoglobin Sb0/+ after undergoing allogeneic SCT/BMT from HLA genotype identical donors. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
- Evaluate response to immunization after BMT in patients with SCD, hemoglobin SC, or hemoglobin Sb0/+. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
|Study Start Date:||July 2005|
|Study Completion Date:||July 2012|
|Primary Completion Date:||July 2012 (Final data collection date for primary outcome measure)|
Experimental: Allogeneic BMT/SCT Transplant
Busulfan, Campath 1H, Cyclophosphamide and MESNA:
Bone marrow infusion with pre-meds as per SOPs to take place on Day 0.
Bone marrow dose: To ensure the probability for bone marrow engraftment, 4 x 10^8 nucleated cells/kg patient weight will be the target at donor bone marrow harvest.
Starting Day -9 / Busulfan 4.0 mg/kg/day IV divided into four doses daily for four days; total dose = 16 mg/kg.
Other Name: MyleranBiological: Campath 1H
Day -5 through Day -2; Campath-1H dosed as per institutional guidelines.Drug: Cyclophosphamide and MESNA
Day -5 through Day -2; Cyclophosphamide 50 mg/kg + MESNA.
Other Name: CTX
Show Detailed Description
Please refer to this study by its ClinicalTrials.gov identifier: NCT00578344
|United States, Texas|
|Houston, Texas, United States, 77030|
|Texas Children's Hospital|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Kathryn Suet Wa Leung, MD||Baylor College of Medicine/Texas Children's Hospital|