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

Primary Purpose

Sickle Cell Anemia, Hemoglobinopathy, Thalassemia

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Campath -1H
Dilantin
Busulfan
Cyclophosphamide
Sponsored by
Baylor College of Medicine
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sickle Cell Anemia

Eligibility Criteria

1 Day - 64 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Texas Children's Hospital
  • The Methodist Hospital

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
June 26, 2002
Last Updated
January 15, 2020
Sponsor
Baylor College of Medicine
Collaborators
The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine
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1. Study Identification

Unique Protocol Identification Number
NCT00040469
Brief Title
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

2. Study Status

Record Verification Date
January 2020
Overall Recruitment Status
Terminated
Why Stopped
accrual was slow and sporadic so the study was closed
Study Start Date
August 2000 (undefined)
Primary Completion Date
November 21, 2003 (Actual)
Study Completion Date
November 21, 2003 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Baylor College of Medicine
Collaborators
The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Anemia, Hemoglobinopathy, Thalassemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
15 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Campath -1H
Intervention Type
Drug
Intervention Name(s)
Dilantin
Intervention Type
Drug
Intervention Name(s)
Busulfan
Intervention Type
Drug
Intervention Name(s)
Cyclophosphamide

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Day
Maximum Age & Unit of Time
64 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Malcolm K. Brenner, MD
Organizational Affiliation
Baylor College of Medicine
Official's Role
Study Chair
Facility Information:
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
The Methodist Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States

12. IPD Sharing Statement

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

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