We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Try the New Site
We're building a modernized ClinicalTrials.gov! Visit Beta.ClinicalTrials.gov to try the new functionality.
ClinicalTrials.gov Menu

Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient 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: NCT00040417
Recruitment Status : Terminated (unable to accrue patients)
First Posted : June 27, 2002
Last Update Posted : January 18, 2020
The Methodist Hospital Research Institute
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 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.

Condition or disease Intervention/treatment Phase
Sickle Cell Anemia Hemoglobinopathy Thalassemia Drug: FLUDARABINE Drug: CAMPATH-IH Procedure: Total Body Irradiation Drug: FK506 Drug: G-SCF (Granulocyte-colony stimulating factor) Phase 2

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.

Layout table for study information
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: 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
Study Start Date : August 2000
Actual Primary Completion Date : November 21, 2003
Actual Study Completion Date : November 21, 2003

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   1 Day to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Patients with a haploidentical related HLA donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb0/+ thalassemia and at least one of the following conditions:

    1. previous central nervous system vaso-occlusive episode with or without residual neurologic findings;
    2. frequent painful vaso-occlusive episodes which significantly interfere with normal life activities and which necessitate chronic transfusion therapy;
    3. recurrent SCD chest syndrome events, which necessitate chronic transfusion therapy;
    4. severe anemia, which prevents acceptable quality of life and necessitates chronic transfusion therapy.
  • Patients with a haploidentical related HLA donor and homozygous b0/+ thalassemia or severe variants of b0/+ thalassemia and require chronic transfusion therapy.
  • Women of childbearing potential must have a negative pregnancy test.
  • Between the ages of birth and 65 years.


  • HLA identical or 5/6 HLA matched sibling donor
  • Biopsy proven chronic active hepatitis or portal fibrosis.
  • 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.
  • Unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate Stem Cell Transplant.
  • Patient or guardian(s) unable to understand the nature and risks inherent in the stem cell transplant process.
  • Pregnant or lactating females and those unwilling to use acceptable contraception.

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): NCT00040417

Layout table for location information
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
Layout table for investigator information
Principal Investigator: Malcolm K. Brenner, MD, FRCP Baylor College of Medicine
Layout table for additonal information
Responsible Party: Robert Krance, Professor, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT00040417    
Other Study ID Numbers: H8750
First Posted: June 27, 2002    Key Record Dates
Last Update Posted: January 18, 2020
Last Verified: January 2020
Additional relevant MeSH terms:
Layout table for MeSH terms
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Antineoplastic Agents
Immunologic Factors
Physiological Effects of Drugs
Adjuvants, Immunologic
Antineoplastic Agents, Immunological