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

NCT05027269 | Completed Phase 1 FDA Drug

• 1 other identifier: AOC 1001-CS1
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 8

Brief Summary

AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA). Part A is a single dose design with 1 cohort (dose level). In Part A, the patient duration is 6 months as the treatment period is 1 day followed by a 6 month follow-up period. Part B is a multiple-ascending dose design with 2 cohorts (dose levels). In Part B, the patient duration is 6 months as the treatment period is 3 months followed by a 3 month follow-up period.

Conditions

DM1; Myotonic Dystrophy 1; Myotonic Dystrophy; Myotonic Dystrophy Type 1 (DM1); Dystrophy Myotonic; Myotonic Disorders; Steinert Disease; Myotonic Muscular Dystrophy

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Frequency of treatment emergent adverse events (TEAEs)

  Through study completion, up to Day 183

Secondary Outcomes (8)

• Plasma pharmacokinetic (PK) parameters

  Through study completion, up to Day 183

• Plasma pharmacokinetic (PK) parameters

  Through study completion, up to Day 183

• Plasma pharmacokinetic (PK) parameters

  Through study completion, up to Day 183

• Plasma pharmacokinetic (PK) parameters

  Through study completion, up to Day 183

• Urine pharmacokinetic (PK) parameters

  Through study completion, up to Day 183

Study Arms (4)

Part A Single Dose: AOC 1001 Dose Level 1

EXPERIMENTAL

AOC 1001 will be administered once.

Drug: AOC 1001

Part A Single Dose: Placebo

PLACEBO COMPARATOR

Saline will be administered once.

Drug: Placebo

Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3

EXPERIMENTAL

AOC 1001 will be administered three times.

Drug: AOC 1001

Part B Multiple Ascending Dose: Placebo

PLACEBO COMPARATOR

Saline will be administered three times.

Drug: Placebo

Interventions

AOC 1001 DRUG

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

Part A Single Dose: AOC 1001 Dose Level 1; Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3

Placebo DRUG

Placebo will be administered by intravenous (IV) infusion.

Part A Single Dose: Placebo; Part B Multiple Ascending Dose: Placebo

Eligibility Criteria

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

You may qualify if:

• Genetic diagnosis of DM1 (CTG repeat length ≥ 100)
• Clinician assessed signs of DM1
• Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening

You may not qualify if:

• Diabetes that is not adequately controlled
• BMI \> 35 kg/m2
• Uncontrolled hypertension
• Congenital DM1
• History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during study period
• Recently treated with an investigational drug
• Treatment with anti-myotonic medication within 14 days of Day 1

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

Related Publications (1)

• Johnson NE, Tai LJ, Hamel JI, Day JW, Statland JM, Soltanzadeh P, Subramony SH, Thornton CA, Arnold WD, Wicklund M, Freimer ML, Eichinger K, Dekdebrun J, Chen CY, Goel V, McEvoy B, Zhu Y, Hughes SG, Ackermann EJ, Levin AA. An Antibody-Oligonucleotide Conjugate for Myotonic Dystrophy Type 1. N Engl J Med. 2026 Feb 19;394(8):763-772. doi: 10.1056/NEJMoa2407326.

  PMID: 41707138 DERIVED

Related Links

• Related Info

MeSH Terms

Conditions

Myotonic Dystrophy; Myotonic Disorders

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Li Tai, MD

  Avidity Biosciences, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 24, 2021
• First Posted: August 30, 2021
• Study Start: October 28, 2021
• Primary Completion: February 14, 2023
• Study Completion: February 14, 2023
• Last Updated: March 12, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)