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

This study has been terminated.

First Received on June 26, 2002. Last Updated on April 9, 2007 History of Changes

Sponsor:	Baylor College of Medicine
Collaborators:	The Methodist Hospital System Texas Children's Hospital Center for Cell and Gene Therapy, Baylor College of Medicine
Information provided by:	Baylor College of Medicine
ClinicalTrials.gov Identifier:	NCT00040469

Purpose

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.

Condition	Intervention	Phase
Sickle Cell Anemia Hemoglobinopathy Thalassemia	Drug: Campath -1H Drug: Dilantin Drug: Busulfan Drug: Cyclophosphamide	Phase II

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
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

Resource links provided by NLM:

Genetics Home Reference related topics: sickle cell disease

MedlinePlus related topics: Anemia Bone Marrow Transplantation Sickle Cell Anemia Thalassemia

Drug Information available for: Cyclophosphamide Busulfan Phenytoin Campath Alemtuzumab Phenytoin sodium

U.S. FDA Resources

Further study details as provided by Baylor College of Medicine:

Estimated Enrollment:	15
Study Start Date:	August 2000

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

Ages Eligible for Study:	up to 64 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00040469

Locations

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 System

Texas Children's Hospital

Center for Cell and Gene Therapy, Baylor College of Medicine

Investigators

Study Chair:

Malcolm K. Brenner, MD

Baylor College of Medicine

More Information

No publications provided

ClinicalTrials.gov Identifier:	NCT00040469 History of Changes
Other Study ID Numbers:	H6847, Scallo2
Study First Received:	June 26, 2002
Last Updated:	April 9, 2007
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Anemia
Anemia, Sickle Cell
Hemoglobinopathies
Thalassemia
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn
Phenytoin
Busulfan
Cyclophosphamide
Campath 1G
Alemtuzumab

Anticonvulsants
Central Nervous System Agents
Therapeutic Uses
Pharmacologic Actions
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Antirheumatic Agents

ClinicalTrials.gov processed this record on February 09, 2012