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HARBOR phase 3 study of intravenous AOC 1001 in myotonic dystrophy type 1
jRCT2051240140Phase 3Active, not recruiting
- Sponsor
- Avidity Biosciences
- Start
- Sep 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
01
Overview
Phase 3, randomised, double-blind trial in myotonic dystrophy type 1 (DM1) comparing AOC 1001 with Placebo (2 arms). Primary endpoint: Hand function. Started Sep 2024; status: active, not recruiting.
02
Drugs
delpacibart etedesiran
03
Conditions
myotonic dystrophy type 1 (DM1)
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