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Phase 3 Study of Intravenous AOC 1001 in Myotonic Dystrophy Type 1
2024-511378-60-00Phase 3Completed
- Sponsor
- Avidity Biosciences
- Start
- Aug 2024
- Modality
- 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
- 13
01
Overview
Phase 3, randomised, double-blind trial in myotonic dystrophy type 1 (DM1) comparing AOC 1001 IV with Placebo IV (0.9% Saline) (2 arms). Primary endpoint: Change from baseline to Week 54 in video Hand Opening Time (vHOT). Started Aug 2024; status: completed.
02
Drugs
delpacibart etedesiran
03
Conditions
myotonic dystrophy type 1 (DM1)
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