Stem Cell Transplantation With Identical Donors for Patients With Sickle Cell Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 17 October 2012
• Main ID: NCT00186810
• Date of registration: 09/09/2005
• Primary sponsor: St. Jude Children's Research Hospital
• Public title: Stem Cell Transplantation With Identical Donors for Patients With Sickle Cell Disease
• Scientific title: Allogeneic Stem Cell Transplantation From HLA/MLC Genotype Identical Donors for Patients With High Risk Sickle Cell Disease
• Date of first enrolment: December 1992
• Target sample size: 15
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00186810
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Countries of recruitment

United States

Contacts

• Name: Gregory Hale, MD
• Affiliation: St. Jude Children's Research Hospital

Key inclusion & exclusion criteria

Inclusion criteria:

Diagnosis of severe' disease is denoted by one of the following:

- Previous central nervous system vaso-occlusive episode with or without residual
neurologic findings or

- Frequent painful vaso-occlusive episodes with significant interference with normal
life activities and which necessitates chronic transfusion therapy or

- Recurrent SCD chest syndrome events which necessitate chronic transfusion therapy.

Exclusion criteria:

- Patient with SCD chronic lung disease greater than or equal to stage 3

- Patient with severe renal dysfunction defined as creatinine clearance < 40
ml/min/1.73m2.

- Patient with severe cardiac dysfunction defined as echocardiogram shortening fraction
< 25%.

- Patient with HIV infection.

- Pregnant or lactating.

- Patient with unspecified chronic toxicity that in the opinion of the Principal
Investigator is serious enough to detrimentally affect the patient's capacity to
tolerate SCT.

- Patient or patient's guardian(s) unable to understand the nature and risks inherent
in the BMT process



Age minimum: N/A
Age maximum: 21 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Sickle Cell Disease

Intervention(s)

Drug: Busulfan, Cyclophosphamide, Horse ATG

Procedure: Allogeneic stem cell transplant

Primary Outcome(s)

To evaluate engraftment, GVHD, hematopoietic and immune reconstitution, and regimen-related mortality and morbidity in patients with severe sickle cell disease undergoing transplant using either HLA matched sibling bone marrow or cord blood grafts. [Time Frame: March 2007]

Secondary ID(s)

SCALLO

Source(s) of Monetary Support

Please refer to primary and secondary sponsors