- ICH GCP
- US Clinical Trials Registry
- Clinical Trial 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
Study Overview
Status
Conditions
Intervention / Treatment
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.
Study Type
Enrollment
Phase
- Phase 2
Contacts and Locations
Study Locations
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Texas
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Houston, Texas, United States, 77030
- The Methodist Hospital
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Houston, Texas, United States, 77030
- Texas Children's Hospital
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion:
- Patients with homozygous B0/+ thalassemia or severe variants of B0/+ thalassemia with an HLA genotypically identical donor.
- Patients with an HLA genotype identical donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb 0/+ and at least one of the following:
Previous central nervous system vaso-occlusive episode with or without residual neurologic findings; Frequent painful vaso-occlusive episodes which significantly interfere with normal life activities and which necessitate chronic transfusion therapy; Recurrent SCD chest syndrome events which necessitate chronic transfusion therapy.
- Severe anemia which prevents acceptable quality of life and necessitates chronic transfusion therapy.
- The patient must have an HLA genotype identical donor.
- Between the ages of birth and 65 years.
- Women of childbearing potential must have a negative pregnancy test.
Exclusion:
- Biopsy proven chronic active hepatitis or fibrosis with portal bridging.
- SCD chronic lung disease >/= stage 3.
- Severe renal dysfunction defined as creatinine clearance <40 ml/min/1.73 M2
- Severe cardiac dysfunction defined as shortening fraction <25%.
- HIV infection.
- Severe but unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate bone marrow transplant (BMT).
- Inadequate intellectual capacity to understand the nature and risk inherent in the BMT process and give informed consent (in the case of minors, this criteria must be fulfilled by the legal guardian).
- Pregnant, lactating or unwilling to use appropriate birth control.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Study Chair: Malcolm K. Brenner, MD, Baylor College of Medicine
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Hematologic Diseases
- Genetic Diseases, Inborn
- Anemia
- Anemia, Hemolytic, Congenital
- Anemia, Hemolytic
- Anemia, Sickle Cell
- Thalassemia
- Hemoglobinopathies
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Antirheumatic Agents
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Antineoplastic Agents, Alkylating
- Alkylating Agents
- Myeloablative Agonists
- Antineoplastic Agents, Immunological
- Membrane Transport Modulators
- Anticonvulsants
- Voltage-Gated Sodium Channel Blockers
- Sodium Channel Blockers
- Cytochrome P-450 CYP1A2 Inducers
- Cytochrome P-450 Enzyme Inducers
- Cyclophosphamide
- Busulfan
- Alemtuzumab
- Phenytoin
Other Study ID Numbers
- H6847
- Scallo2
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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