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

NCT00040469 | Terminated Phase 2

• 2 other identifiers: H6847Scallo2
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 2

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.

Conditions

Sickle Cell Anemia; Hemoglobinopathy; Thalassemia

Interventions

Campath -1H DRUG

Dilantin DRUG

Busulfan DRUG

Cyclophosphamide DRUG

Eligibility Criteria

• Age: 1 Day - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Baylor College of Medicine: lead
• The Methodist Hospital Research Institute: collaborator
• Center for Cell and Gene Therapy, Baylor College of Medicine: collaborator

Study Sites (2)

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

The Methodist Hospital

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Hemoglobinopathies; Thalassemia

Interventions

Alemtuzumab; Phenytoin; Busulfan; Cyclophosphamide

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Hydantoins; Imidazolidines; Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds

Study Officials

• Malcolm K. Brenner, MD

  Baylor College of Medicine

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: June 26, 2002
• First Posted: June 28, 2002
• Study Start: August 1, 2000
• Primary Completion: November 21, 2003
• Study Completion: November 21, 2003
• Last Updated: January 18, 2020

Record last verified: 2020-01

Locations

US (2)