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Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies

Primary Purpose

Sickle Cell Anemia, Hemoglobinopathy, Thalassemia

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
FLUDARABINE
CAMPATH-IH
Total Body Irradiation
FK506
G-SCF (Granulocyte-colony stimulating factor)
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 - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

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
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1. Study Identification

Unique Protocol Identification Number
NCT00040417
Brief Title
Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies
Official Title
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 Type
Interventional

2. Study Status

Record Verification Date
January 2020
Overall Recruitment Status
Terminated
Why Stopped
unable to accrue patients
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

4. Oversight

5. Study Description

Brief Summary
The major goal of this study is to determine the risks and benefits of stem cell transplants in combination with a newer, less toxic conditioning chemotherapy treatment in patients with severe sickle cell disease (SCD) or sickle hemoglobin variants (hemoglobin SC or hemoglobin SB0/+), or homozygous b0/+ thalassemia or severe B0/+ thalassemia variants. Participation in this project will be for one year, with follow up evaluations done every 6 months thereafter for 10 years or until participants are 18 years old.
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.

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)
FLUDARABINE
Intervention Type
Drug
Intervention Name(s)
CAMPATH-IH
Intervention Type
Procedure
Intervention Name(s)
Total Body Irradiation
Intervention Type
Drug
Intervention Name(s)
FK506
Intervention Type
Drug
Intervention Name(s)
G-SCF (Granulocyte-colony stimulating factor)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Day
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Malcolm K. Brenner, MD, FRCP
Organizational Affiliation
Baylor College of Medicine
Official's Role
Principal Investigator
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 Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies

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