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

NCT05479981 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 37

Last updated 2025-10-08

No results posted yet for this study

Summary

AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients

Conditions

  • DM1
  • Muscular Dystrophies
  • Myotonic Dystrophy
  • Myotonic Dystrophy 1
  • Myotonic Disorders
  • Muscular Disorders, Atrophic
  • Muscular Diseases
  • Musculoskeletal Diseases
  • Neuromuscular Diseases
  • Nervous System Diseases
  • Genetic Diseases, Inborn
  • Heredodegenerative Disorders, Nervous System
  • Neurodegenerative Diseases

Interventions

DRUG

Placebo

Placebo will be administered by intravenous (IV) infusion.

DRUG

AOC 1001

AOC 1001 will be administered by intravenous (IV) infusion.

Sponsors & Collaborators

  • Avidity Biosciences, Inc.

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
66 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-08-04
Primary Completion
2025-09-05
Completion
2025-09-10
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05479981 on ClinicalTrials.gov