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Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00040469
Recruitment Status : Terminated (accrual was slow and sporadic so the study was closed)
First Posted : June 28, 2002
Last Update Posted : January 18, 2020
The Methodist Hospital Research Institute
Center for Cell and Gene Therapy, Baylor College of Medicine
Information provided by (Responsible Party):
Robert Krance, Baylor College of Medicine

Brief Summary:
The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years.

Condition or disease Intervention/treatment Phase
Sickle Cell Anemia Hemoglobinopathy Thalassemia Drug: Campath -1H Drug: Dilantin Drug: Busulfan Drug: Cyclophosphamide Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 15 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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 Start Date : August 2000
Actual Primary Completion Date : November 21, 2003
Actual Study Completion Date : November 21, 2003

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Day to 64 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • 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.


  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00040469

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United States, Texas
Texas Children's Hospital
Houston, Texas, United States, 77030
The Methodist Hospital
Houston, Texas, United States, 77030
Sponsors and Collaborators
Baylor College of Medicine
The Methodist Hospital Research Institute
Center for Cell and Gene Therapy, Baylor College of Medicine
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Study Chair: Malcolm K. Brenner, MD Baylor College of Medicine
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Responsible Party: Robert Krance, Professor, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT00040469    
Other Study ID Numbers: H6847
First Posted: June 28, 2002    Key Record Dates
Last Update Posted: January 18, 2020
Last Verified: January 2020
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Antineoplastic Agents, Immunological
Voltage-Gated Sodium Channel Blockers
Sodium Channel Blockers
Membrane Transport Modulators
Cytochrome P-450 CYP1A2 Inducers
Cytochrome P-450 Enzyme Inducers