Clinical trial NCT00040469: Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies
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 type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Sickle Cell Anemia
Hemoglobinopathy
Thalassemia
Lead sponsor: Baylor College of Medicine
Sponsor class: OTHER
Collaborators: name: The Methodist Hospital Research Institute
name: Center for Cell and Gene Therapy, Baylor College of Medicine
First posted: 2002-06-28
Enrollment: 15
Sex: ALL
Minimum age (years): 1
Maximum age (years): 64
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: Inclusion:

* 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.

Exclusion:

* 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Houston
country: United States
facility: The Methodist Hospital
state: Texas
Study officials:
affiliation: Baylor College of Medicine
name: Malcolm K. Brenner, MD
role: STUDY_CHAIR
References: