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Reduced Intensity Conditioning in Pediatric Patients and Young Adults With Non-Malignant Disorders Undergoing HSCT
NCT01962415Phase 2Recruiting
- Sponsor
- Paul Szabolcs
- Start
- Feb 2014
- Primary completion
- Nov 2026
- Modality
- Monoclonal Antibodies, Orthosteric Ligands, Metabolically Activated Prodrugs
- Countries
- 1
01
Overview
Phase 2, open-label trial of alemtuzumab, hydroxyurea, fludarabine, melphalan and thiotepa in primary immunodeficiency (PI), Congenital bone marrow failure syndrome (CBMFS), inherited metabolic disorder and 4 more (2 arms), sponsored by Paul Szabolcs. Primary endpoints: Post-transplant treatment-related mortality at 1 year, Neurodevelopmental milestones at 1 year and 3 more. Started Feb 2014; status: recruiting.
02
Drugs
alemtuzumabhydroxyureafludarabinemelphalanthiotepa
03
Conditions
primary immunodeficiency (PI)Congenital bone marrow failure syndrome (CBMFS)inherited metabolic disorderhereditary anemiaInflammatory Disorders
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