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Phase 2 open-label single-dose study of subcutaneous ALN-CC5 in PNH patients inadequately responding to eculizumab
2016-002943-40Phase 2Terminated
- Sponsor
- Alnylam Pharmaceuticals
- Start
- Sep 2016
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Peptide-Drug Conjugates → Peptide Conjugates → Peptides
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of cemdisiran in paroxysmal nocturnal hemoglobinuria (PNH), sponsored by Alnylam Pharmaceuticals. Primary endpoint: Percent change from baseline in LDH. Started Sep 2016; status: terminated.
02
Drugs
cemdisiran
03
Conditions
paroxysmal nocturnal hemoglobinuria (PNH)
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