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Patisiran-LNP in hATTR amyloidosis patients with disease progression after liver transplant
2018-003519-24Phase 3Completed
- Sponsor
- Alnylam Pharmaceuticals
- Start
- Nov 2018
- Modality
- Gene Therapies, 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
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of patisiran in hereditary transthyretin amyloidosis (hATTR) and amyloid cardiomyopathy, sponsored by Alnylam Pharmaceuticals. Primary endpoint: Percent change from baseline in serum transthyretin (TTR) levels. Started Nov 2018; status: completed.
02
Drugs
patisiran
03
Conditions
hereditary transthyretin amyloidosis (hATTR)amyloid cardiomyopathy
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