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NCT Number: NCT05479981

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

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

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Key information

Conditions

DM1 Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Myotonic Disorders Myotonic Dystrophy Myotonic Dystrophy 1 Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases

Age range

18 year–66 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of California Los Angeles, Los Angeles, California, United States

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About this study

This study will continue to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AOC 1001 in participants that enrolled in the randomized, placebo-controlled, First-In-Human Phase 1/2 AOC 1001-CS1 (MARINA) clinical study.

Participants from AOC 1001-CS1 are eligible to enroll in AOC 1001-CS2 if they have satisfactorily completed AOC 1001-CS1.

After completion of Q8W Visit 6 assessments, participants will have the option to enroll in another AOC 1001 open-label extension (OLE) study. Participants who do not enroll in an AOC 1001 OLE study will be monitored for safety for a total of 8 weeks from the last dose of study drug. Total duration on study is up to 37 months (3 years 1 month).

As of September 2024, the dosing regimen was updated to every 8 weeks.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • Completion of AOC 1001-CS1 (MARINA) study with satisfactory compliance and no significant tolerability issues

Key Exclusion Criteria:

  • Pregnancy, intent to become pregnant, or active breastfeeding
  • Unwilling or unable to continue to comply with contraceptive requirements
  • Any new conditions or worsening of existing conditions that in the opinion of the investigator or sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study

Treatment and study plan

Placebo

Drug

Placebo will be administered by intravenous (IV) infusion.

Other names: Saline

AOC 1001

Drug

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

Primary outcomes

  1. Number and severity of treatment-emergent adverse events (TEAEs)

    Time frame: Through study completion

Secondary outcomes

  1. AOC 1001 levels in muscle tissue

    Time frame: Through Day 183

  2. Change and percentage change from baseline in DMPK mRNA knockdown

    Time frame: Through Day 183

  3. Change and percentage change from baseline in Spliceopathy

    Time frame: Through Day 183

  4. Plasma pharmacokinetic (PK) parameters

    Time frame: Up to Day 729

    Maximum and trough plasma concentration

Sponsors and collaborators

Lead sponsor

Avidity Biosciences, Inc.

Industry

Registry information

Official study title

A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients

Acronym: MARINA-OLE

Important dates

Study start
2022
Primary completion
2025
Study completion
2025
First posted
Jul 29, 2022
Registry last updated
Oct 8, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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