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Radiation- and alkylator-free hematopoietic cell transplantation for bone marrow failure in dyskeratosis congenita / telomere disease
2018-001489-41Phase 2Completed
- Sponsor
- Karolinska University Hospital
- Start
- May 2018
- Modality
- Monoclonal Antibodies → Antibody-Based Therapeutics, Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules, DNA Intercalators/Alkylators → Nucleic Acid-Directed Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of alemtuzumab and fludarabine in bone marrow failure, dyskeratosis congenita (DC), telomere syndrome and 2 more, sponsored by Karolinska University Hospital. Primary endpoint: Neutrophil engraftment. Started May 2018; status: completed.
02
Drugs
alemtuzumabfludarabine
03
Conditions
bone marrow failuredyskeratosis congenita (DC)telomere syndromeaplastic anemiasingle-lineage cytopenia
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