- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00040417
Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies
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 Overview
Status
Conditions
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
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 a haploidentical related HLA donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb0/+ thalassemia and at least one of the following conditions:
- 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.
- 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.
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
- Principal Investigator: Malcolm K. Brenner, MD, FRCP, 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
- Antineoplastic Agents
- Immunologic Factors
- Antineoplastic Agents, Immunological
- Adjuvants, Immunologic
- Lenograstim
- Fludarabine
- Alemtuzumab
Other Study ID Numbers
- H8750
- Smallo
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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