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Phase 3 randomized double-blind placebo-controlled study of fazirsiran in alpha-1 antitrypsin deficiency-associated liver disease
2022-501943-34-00Phase 3Authorised
- Sponsor
- Takeda
- Start
- Jun 2023
- 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, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 19
01
Overview
Phase 3, randomised, double-blind trial in alpha-1 antitrypsin deficiency (AATD) comparing Fazirsiran with Placebo saline (2 arms). Primary endpoint: Histologic fibrosis improvement at Week 106 in F2 to F3 population. Planned start Jun 2023; status: authorised.
02
Drugs
fazirsiran
03
Conditions
alpha-1 antitrypsin deficiency (AATD)
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