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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
Sponsor:
Collaborator:
The Methodist Hospital Research Institute
Information provided by (Responsible Party):
Robert Krance, Baylor College of Medicine

Tracking Information
First Submitted Date  ICMJE June 26, 2002
First Posted Date  ICMJE June 27, 2002
Last Update Posted Date January 18, 2020
Study Start Date  ICMJE August 2000
Actual Primary Completion Date November 21, 2003   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE Not Provided
Original Primary Outcome Measures  ICMJE Not Provided
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies
Official Title  ICMJE 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
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.
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Sickle Cell Anemia
  • Hemoglobinopathy
  • Thalassemia
Intervention  ICMJE
  • Drug: FLUDARABINE
  • Drug: CAMPATH-IH
  • Procedure: Total Body Irradiation
  • Drug: FK506
  • Drug: G-SCF (Granulocyte-colony stimulating factor)
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Enrollment  ICMJE
 (submitted: June¬†23,¬†2005)
15
Original Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE November 21, 2003
Actual Primary Completion Date November 21, 2003   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion:

  • 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.

Exclusion:

  • 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Day to 65 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00040417
Other Study ID Numbers  ICMJE H8750
Smallo
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Robert Krance, Baylor College of Medicine
Original Responsible Party Not Provided
Current Study Sponsor  ICMJE Baylor College of Medicine
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE The Methodist Hospital Research Institute
Investigators  ICMJE
Principal Investigator: Malcolm K. Brenner, MD, FRCP Baylor College of Medicine
PRS Account Baylor College of Medicine
Verification Date January 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP