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

NCT05479981 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: AOC 1001-CS2
• Study Type: interventional
• Target: 37
• Locations: 1 country
• Sites: 8

Brief 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

Keywords

DM1; Myotonic Dystrophy 1; Myotonic Dystrophy Type 1 (DM1); Myotonic Dystrophy; DM; Dystrophy Myotonic; Myotonic Disorders; Steinert Disease; MARINA-OLE; MARINA; Avidity Biosciences; Avidity; AOC 1001; Myotonic Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

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

  Through study completion

Secondary Outcomes (4)

• AOC 1001 levels in muscle tissue

  Through Day 183

• Change and percentage change from baseline in DMPK mRNA knockdown

  Through Day 183

• Change and percentage change from baseline in Spliceopathy

  Through Day 183

• Plasma pharmacokinetic (PK) parameters

  Up to Day 729

Study Arms (2)

AOC 1001

EXPERIMENTAL

AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant did not receive AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive AOC 1001 treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks.

Drug: AOC 1001

AOC 1001 (with Placebo at Day 43)

EXPERIMENTAL

AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant received AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive blinded placebo treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks.

Drug: Placebo; Drug: AOC 1001

Interventions

Placebo DRUG

Placebo will be administered by intravenous (IV) infusion.

Also known as: Saline

AOC 1001 (with Placebo at Day 43)

AOC 1001 DRUG

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

AOC 1001; AOC 1001 (with Placebo at Day 43)

Eligibility Criteria

• Age: 18 Years - 66 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Avidity Biosciences, Inc.: lead

Study Sites (8)

University of California Los Angeles

Los Angeles, California, 90095, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

University of Colorado

Denver, Colorado, 80045, United States

Location

University of Florida

Gainesville, Florida, 32608, United States

Location

Kansas University Medical Center

Kansas City, Kansas, 66205, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

Ohio State University

Columbus, Ohio, 43221, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

MeSH Terms

Conditions

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

Interventions

Sodium Chloride

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Masking Details: Participants will receive one blinded treatment at Day 43. Treatment assignment on this day will be based on treatment received in AOC 1001-CS1 so that participants who did not receive active drug on Day 43 in AOC 1001-CS1 will received active drug on Day 43 in AOC 1001-CS2.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 27, 2022
• First Posted: July 29, 2022
• Study Start: August 4, 2022
• Primary Completion: September 5, 2025
• Study Completion: September 10, 2025
• Last Updated: October 8, 2025

Record last verified: 2025-10

Locations

US (8)