SALT: Alternative Donor Bone Marrow and Cord Blood Transplantation for High Risk Sickle Cell Disease
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00257543|
Recruitment Status : Terminated (lack of enrollment)
First Posted : November 23, 2005
Last Update Posted : September 17, 2013
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Procedure: Alternative donor bone marrow and cord blood||Early Phase 1|
Unfortunately, less than 1/4th of patients with severe SCD will have a matched sibling donor that can serve as a BMT donor. This research protocol proposes to study the safety and usefulness of "alternate donor transplant" (using donors other than matched siblings). We will offer this treatment to children with severe sickle cell disease that do not have a matched sibling BMT donor. Alternative donors can be family members who are slightly less than completely matched, unrelated volunteer donors who are completely matched, and donated banked umbilical cord blood that is completely or nearly completely matched.
Alternative donor transplant has been performed commonly in patients with cancer, and also provides curative therapy for several non-malignant diseases (severe immunodeficiency, marrow failure and metabolic storage diseases). Alternate donor transplants carry higher risks of complications, including graft-versus-host disease, infection, and graft failure. Therefore, we will be selective about which patients are invited to participate, limiting eligibility to those patients that have had a severe SCD related problem (rather than those who are doing well and are likely to have few SCD related problems), but excluding patients who have such severe organ damage that they are more likely to die during transplant, and limiting eligibility to a young age group. A multi-step review algorithm that includes internal, local and external expert review has been constructed to provide a thorough, safe and ethical accrual process. We will treat patients using drugs and methods commonly used in alternate donor transplant for other diseases such as leukemia, and incorporate lessons learned from our previous experience in BMT for sickle cell by modifying supportive care measures. Special attention will be given to evaluation of post-BMT effects in this population, as well as potential reasons for adverse effects (such as graft failure).
We think that Atlanta is a particularly good place to study this kind of transplant for several reasons. One reason is experience: our program has transplanted more children with SCD than any other single institution in North America, with excellent outcomes. Additionally, SCD patients in our area often have been treated on a special red cell transfusion program that limits the number of people donating the blood; we think this is likely to reduce the chance of graft failure.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||3 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||SALT - Alternative Donor Bone Marrow and Cord Blood Transplantation for High Risk Sickle Cell Disease|
|Study Start Date :||January 2006|
|Primary Completion Date :||August 2012|
|Study Completion Date :||August 2012|
Experimental: single arm study
this is a single arm study
Procedure: Alternative donor bone marrow and cord blood
bone marrow transplant - alternative donors for bone marrow and cord blood transplants
- To determine the frequency of donor engraftment after alternate donor transplantation in children with severe sickle cell disease. [ Time Frame: 1 year after completion of study accrual ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00257543
|United States, Georgia|
|Children's Healthcare of Altanta|
|Atlanta, Georgia, United States, 30322|
|Study Chair:||Ann Haight, M.D.||Children's Healthcare of Atlanta/Emory University|