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Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP

NCT00578344 · Status: TERMINATED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2020-07-31

Study results available
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Summary

Patients are being asked to participate in this study because they have severe sickle cell anemia (SCD) with or without the beta thalassemia trait. Sickle cell anemia is an illness where the red blood cells change shape and can clog up blood vessels. This keeps the body from getting the oxygen it needs. Thalassemia is when the body does not make enough hemoglobin, something that helps the oxygen get to the places it needs to go in the body. The patient may or may not need to get regular blood transfusions (getting more blood) to improve their quality of life (feel better) and prevent organ damage (problems with the brain, heart, lung, kidney, and gonad, for example.). The transfusions can also cause problems, including iron overload (too much iron in the blood), which can be fatal (patients can die) without regular deferoxamine shots. Even with the best usual treatments, people with thalassemia or SCD die sooner. There is no proven cure.

We would like to treat patients using bone marrow transplantation, a treatment that has been used for people with SCD. The transplant uses healthy "matched" bone marrow. This comes from a brother or sister who does not have sickle cell disease or severe thalassemia. If the treatment works, the sickle cell disease or thalassemia may be cured. This treatment has been used to treat patients with sickle cell disease or thalassemia. It has worked in most cases. We hope, but cannot promise, that the transplanted marrow will make healthy cells, and patients will not have sickle cell disease or severe thalassemia anymore.

We do not know what effect this treatment will have on the damage that has already been done by the disease. Finding that out is the main reason for this study. Currently, very little has been reported about organ function after bone marrow transplants in patients with sickle cell anemia.

Conditions

Interventions

DRUG

Busulfan

Starting Day -9 / Busulfan 4.0 mg/kg/day IV divided into four doses daily for four days; total dose = 16 mg/kg.

BIOLOGICAL

Campath 1H

Day -5 through Day -2; Campath-1H dosed as per institutional guidelines.

DRUG

Cyclophosphamide and MESNA

Day -5 through Day -2; Cyclophosphamide 50 mg/kg + MESNA.

Sponsors & Collaborators

  • Baylor College of Medicine

    collaborator OTHER

  • The Methodist Hospital Research Institute

    collaborator OTHER

  • Tami D. John

    lead OTHER

Principal Investigators

  • 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
40 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2005-07-31
Primary Completion
2012-07-31
Completion
2012-07-31

Countries

  • United States

Study Locations

More Related Trials

Entities

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 NCT00578344 on ClinicalTrials.gov