Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies - Full Text View

Purpose

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.

Condition	Intervention	Phase
Sickle Cell Anemia Hemoglobinopathy Thalassemia	Drug: FLUDARABINE Drug: CAMPATH-IH Procedure: Total Body Irradiation Drug: FK506 Drug: G-SCF (Granulocyte-colony stimulating factor)	Phase II

Genetics Home Reference related topics:

beta thalassemia sickle cell disease

MedlinePlus related topics:

Anemia Sickle Cell Anemia Thalassemia

ChemIDplus related topics:

Fludarabine Fludarabine monophosphate Tacrolimus Sargramostim Granulocyte-macrophage colony-stimulating factor Granulocyte colony-stimulating factor Alemtuzumab Campath Tacrolimus anhydrous

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

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

Further study details as provided by Baylor College of Medicine:

Estimated Enrollment:	15
Study Start Date:	August 2000

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.

Eligibility

Ages Eligible for Study:	up to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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:
1. previous central nervous system vaso-occlusive episode with or without residual neurologic findings;
2. frequent painful vaso-occlusive episodes which significantly interfere with normal life activities and which necessitate chronic transfusion therapy;
3. recurrent SCD chest syndrome events, which necessitate chronic transfusion therapy;
4. 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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00040417

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

Texas Children's Hospital

The Methodist Hospital System

Investigators


Principal Investigator:	Malcolm K. Brenner, MD, FRCP	Baylor College of Medicine

More Information

Study ID Numbers:	H8750, Smallo
First Received:	June 26, 2002
Last Updated:	April 9, 2007
ClinicalTrials.gov Identifier:	NCT00040417
Health Authority:	United States: Food and Drug Administration

Study placed in the following topic categories:

Hematologic Diseases

Anemia

Anemia, Hemolytic

Tacrolimus

Fludarabine monophosphate

Thalassemia

Sickle cell anemia

Anemia, Hemolytic, Congenital

Genetic Diseases, Inborn

Hemoglobinopathies

Alemtuzumab

Fludarabine

Hemoglobinopathy

Anemia, Sickle Cell

Additional relevant MeSH terms:

Antineoplastic Agents

Therapeutic Uses

Pharmacologic Actions

ClinicalTrials.gov processed this record on September 05, 2008

Sponsors and Collaborators:	Baylor College of Medicine Texas Children's Hospital The Methodist Hospital System
Information provided by:	Baylor College of Medicine
ClinicalTrials.gov Identifier:	NCT00040417