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Phase 3 randomized double-blind placebo-controlled study of AOC 1020 in facioscapulohumeral muscular dystrophy
2025-521012-18-00Phase 3Authorised
- Sponsor
- Avidity Biosciences
- Start
- Apr 2026
- Modality
- Cytotoxic ADCs → Antibody-Drug Conjugates (ADCs) → Antibody Conjugates → Antibody-Based Therapeutics, 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
- 10
01
Overview
Phase 3, randomised, double-blind trial in facioscapulohumeral muscular dystrophy (FSHD) comparing AOC 1020 with Placebo (2 arms). Primary endpoint: Change from baseline to Week 78 in 10-meter walk/run test velocity. Planned start Apr 2026; status: authorised.
02
Drugs
delpacibart braxlosiran
03
Conditions
facioscapulohumeral muscular dystrophy (FSHD)
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