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Unrelated Donor HSCT for Severe Sickle Cell Disease
NCT01279616Phase 2
Key facts
- Status
- Terminated
- Start
- Sep 2010
- Primary completion
- Jan 2015
- Countries
- 1
- Sponsor
- Nationwide Children's Hospital
- Modality
- Monoclonal Antibodies, Recombinant Proteins and Enzymes, Orthosteric Ligands
- Conditions
- sickle cell disease (SCD)
01
Overview
Phase 2, open-label, single-arm trial of fludarabine, rituximab, busulfan, antithymocyte globulin rabbit, cyclophosphamide and 2 more in sickle cell disease (SCD), sponsored by Nationwide Children's Hospital. Primary endpoint: Event-Free Survival at 1 year. Started Sep 2010; status: terminated.
02
Design
1 arm- Study design
- Non-randomizedOpen-labelSingle group
Hematopoietic Stem Cell Transplant
Experimental arm receiving conditioning regimen followed by unrelated donor HSCT
03
Who it enrols
Patients under 21 years old with severe sickle cell disease (Hb SS or Sß° thalassemia) and at least one severe clinical complication, lacking a matched family donor.
04
Endpoints
Primary
Event-Free Survival
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