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Radiation- and Alkylator-free HCT for DC/Telomere Disease
NCT01659606Phase 2Active, not recruiting
- Sponsor
- Boston Children's Hospital
- Start
- Jul 2012
- Primary completion
- Dec 2026
- Modality
- Monoclonal Antibodies, Orthosteric Ligands, Metabolically Activated Prodrugs
- Countries
- 3
01
Overview
Phase 2, open-label, single-arm trial of alemtuzumab and fludarabine in dyskeratosis congenita (DC), Hoyeraal Hreidarsson syndrome (HHS), Revesz syndrome and 1 more, sponsored by Boston Children's Hospital. Primary endpoint: Primary engraftment. Started Jul 2012; status: active, not recruiting.
02
Drugs
alemtuzumabfludarabine
03
Conditions
dyskeratosis congenita (DC)Hoyeraal Hreidarsson syndrome (HHS)Revesz syndromeaplastic anemia
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