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ENVISION: Phase 3 study of givosiran in acute hepatic porphyria
jRCT2080223832Phase 3Completed
- Sponsor
- Alnylam Pharmaceuticals
- Start
- Mar 2018
- 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
01
Overview
Phase 3, randomised, double-blind trial in acute porphyria, acute intermittent porphyria (AIP), hereditary coproporphyria (HCP) and 2 more comparing Givosiran with Placebo (2 arms). Primary endpoint: Annualized rate of porphyria attacks. Started Mar 2018; status: completed.
02
Drugs
givosiran
03
Conditions
acute porphyriaacute intermittent porphyria (AIP)hereditary coproporphyria (HCP)variegate porphyria (VP)ALA dehydratase deficiency porphyria (ADP)
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