Study design & results
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Trial intelligence / Profile preview
Phase 3 study of fazirsiran in alpha-1 antitrypsin deficiency-associated liver disease
NL-OMON59995Phase 3Recruiting
- Sponsor
- Takeda
- Start
- Oct 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
- 1
01
Overview
Phase 3, randomised, double-blind trial in alpha-1 antitrypsin deficiency (AATD) comparing Fazirsiran with Placebo (2 arms). Primary endpoint: Histologic fibrosis improvement at Week 106. Started Oct 2024; status: recruiting.
02
Drugs
fazirsiran
03
Conditions
alpha-1 antitrypsin deficiency (AATD)
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Competitive context
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