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Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO

NCT00578292 · Status: TERMINATED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2020-05-01

Study results available
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Summary

Patients have severe beta-thalassemia or one of the thalassemia variants. Thalassemia is a hereditary disease in which the bone marrow produces abnormal red blood cells that have a shorter life span than normal red blood cells. Because of that, the patient has chronically low red blood cell numbers (anemia) and need regular blood transfusions to help the patient feel better and to help prevent damage to important organs such as the heart. The following treatments are currently available to patients: lifelong blood transfusions and drugs that help remove iron from the body, and long-term antibiotics to prevent infections. These treatments are difficult for patients to take, and do not stop the effects of the disease.

Currently, the only treatment that may cure thalassemia is bone marrow or blood stem cell transplantation. Special blood or bone marrow cells from a healthy person might allow the bone marrow to create healthy cells, which will replace the abnormal red blood cells of thalassemia. There is a lot of experience using special blood or bone marrow cells from a healthy brother or sister who is the same HLA (immune) type. For patients who do not have such a donor in the family, an unrelated volunteer donor can be used. It is important for the patient to realize that this kind of transplant can have more problems than a transplant from a brother or sister.

Because we do not know the long-term effects of this treatment and because this type of transplant has not been used often for people with thalassemia, this is a research study. We hope, but cannot promise, that the transplanted marrow/stem cells will produce healthy cells and the patient will no longer have severe thalassemia.

Conditions

Interventions

DRUG

Busulfan

4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6

DRUG

Fludarabine

30mg/m2 Day -5 through Day -2

DRUG

Campath 1H

Per institutional guidelines Days -5 through -2

DRUG

Cyclophosphamide

50 mg/kg Days -5 through -2

DRUG

MESNA

10 mg/kg x 5 Days -5 through -2

Sponsors & Collaborators

  • Baylor College of Medicine

    lead OTHER

Principal Investigators

  • Tami John, MD · Baylor College of Medicine - Texas Children's Hospital

  • Tami John, MD · Baylor College of Medicine - Texas Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
64 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-02-29
Primary Completion
2016-05-31
Completion
2016-05-31

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00578292 on ClinicalTrials.gov