Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO (THALLO)
Patients have severe beta-thalassemia or one of the thalassemia variants. Thalassemia is a hereditary disease in which the bone marrow produces abnormal red blood cells that have a shorter life span than normal red blood cells. Because of that, the patient has chronically low red blood cell numbers (anemia) and need regular blood transfusions to help the patient feel better and to help prevent damage to important organs such as the heart. The following treatments are currently available to patients: lifelong blood transfusions and drugs that help remove iron from the body, and long-term antibiotics to prevent infections. These treatments are difficult for patients to take, and do not stop the effects of the disease.
Currently, the only treatment that may cure thalassemia is bone marrow or blood stem cell transplantation. Special blood or bone marrow cells from a healthy person might allow the bone marrow to create healthy cells, which will replace the abnormal red blood cells of thalassemia. There is a lot of experience using special blood or bone marrow cells from a healthy brother or sister who is the same HLA (immune) type. For patients who do not have such a donor in the family, an unrelated volunteer donor can be used. It is important for the patient to realize that this kind of transplant can have more problems than a transplant from a brother or sister.
Because we do not know the long-term effects of this treatment and because this type of transplant has not been used often for people with thalassemia, this is a research study. We hope, but cannot promise, that the transplanted marrow/stem cells will produce healthy cells and the patient will no longer have severe thalassemia.
Drug: Campath 1H
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Pilot Study of Allogeneic Stem Cell Transplantation From Unrelated Donors for Patients With Severe Homozygous Beta 0/+ Thalassemia or Severe Variants of Beta 0/+ Thalassemia|
- Evaluate engraftment. [ Time Frame: 30 days post-transplant ] [ Designated as safety issue: Yes ]
- To evaluate the occurrence of transient and stable mixed hematopoietic chimerism (HC) after unrelated donor SCT, and its effect on the recurrence of clinically measurable thalassemia. [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
- To measure hematopoietic and immune reconstitution, and assess the effects on infectious complications. [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
- Toxicities. [ Time Frame: +100 days post-transplant ] [ Designated as safety issue: Yes ]
- Loss of chimerism. [ Time Frame: 2 years post-transplant ] [ Designated as safety issue: Yes ]
- GVHD [ Time Frame: 2 years post-transplant ] [ Designated as safety issue: Yes ]
- Morbidity and mortality post-transplant. [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
|Study Start Date:||February 2004|
|Study Completion Date:||May 2016|
|Primary Completion Date:||May 2016 (Final data collection date for primary outcome measure)|
Experimental: Bone Marrow or Stem Cell Infusion
Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H
Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0.
Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor.
4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg
Days -9 through -6
Other Name: MyleranDrug: Fludarabine
30mg/m2 Day -5 through Day -2
Other Name: FludaraDrug: Campath 1H
Per institutional guidelines Days -5 through -2
Other Name: AlemtuzumabDrug: Cyclophosphamide
50 mg/kg Days -5 through -2
Other Name: CytoxanDrug: MESNA
10 mg/kg x 5 Days -5 through -2
Other Name: Mesnex
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00578292
|United States, Texas|
|Texas Children's Hospital|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Kathryn Suet Wa Leung, MD||Baylor College of Medicine/Texas Children's Hospital|