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Phase 3 fazirsiran in PiZZ alpha-1 antitrypsin liver disease with METAVIR F1 fibrosis
2023-504198-19-00Phase 3Authorised
- Sponsor
- Takeda
- Start
- Mar 2024
- 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
- 14
01
Overview
Phase 3, randomised, double-blind trial in alpha-1 antitrypsin deficiency (AATD) comparing Fazirsiran with Placebo (saline) (2 arms). Primary endpoints: Adverse events and serious adverse events, Change in pulmonary function parameters and 4 more. Planned start Mar 2024; status: authorised.
02
Drugs
fazirsiran
03
Conditions
alpha-1 antitrypsin deficiency (AATD)
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