AOC 1001

DRUG4 trials

Sponsors

Avidity Biosciences Inc., Avidity Biosciences, Inc.

Conditions

DM1Dystrophy MyotonicGenetic Diseases, InbornHeredodegenerative Disorders, Nervous SystemMuscular DiseasesMuscular Disorders, AtrophicMuscular DystrophiesMusculoskeletal Diseases

Phase 1

Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

CompletedNCT05027269

Avidity Biosciences, Inc.DM1, Dystrophy Myotonic, Myotonic Disorders +5

Start: 2021-10-28End: 2023-02-14Updated: 2026-03-12

Phase 2

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

CompletedNCT05479981

Avidity Biosciences, Inc.DM1, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System +10

Start: 2022-08-04End: 2025-09-10Updated: 2026-09-11

Phase 3

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

Active, not recruitingCTIS2024-511378-60-00

Avidity Biosciences Inc.Myotonic Dystrophy Type 1

Start: 2024-12-11Target: 39Updated: 2025-11-18

A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

Not yet recruitingCTIS2025-520766-22-00

Avidity Biosciences Inc.Myotonic Dystrophy Type 1

Target: 46Updated: 2025-11-18

Related Papers

Erythromycin for myotonic dystrophy type 1: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.

2023-12-269 citations

Preliminary Assessment of the Phase 1/2 Clinical Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Patients with Myotonic Dystrophy Type 1 (DM1) (MARINA) (S48.002)

Neurology2023-04-252 citations