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Completed

NCT Number: NCT05027269

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

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.

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Key information

Conditions

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

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

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

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

  • 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

Key Exclusion Criteria:

  • 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

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

AOC 1001

Drug

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

Placebo

Drug

Placebo will be administered by intravenous (IV) infusion.

Primary outcomes

  1. Frequency of treatment emergent adverse events (TEAEs)

    Time frame: Through study completion, up to Day 183

Secondary outcomes

  1. Plasma pharmacokinetic (PK) parameters

    Time frame: Through study completion, up to Day 183

    Maximum plasma concentration (Cmax)

  2. Plasma pharmacokinetic (PK) parameters

    Time frame: Through study completion, up to Day 183

    Time to maximum plasma concentration (Tmax)

  3. Plasma pharmacokinetic (PK) parameters

    Time frame: Through study completion, up to Day 183

    Terminal Half-life (t1/2)

  4. Plasma pharmacokinetic (PK) parameters

    Time frame: Through study completion, up to Day 183

    Area Under the Concentration-time Curve (AUC)

  5. Urine pharmacokinetic (PK) parameters

    Time frame: Through study completion, up to Day 183

    fraction excreted (fe) in urine

  6. AOC 1001 levels in muscle tissue

    Time frame: Through study completion, up to Day 183

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

    Time frame: Through study completion, up to Day 183

  8. Change and percentage change from baseline in Spliceopathy

    Time frame: Through study completion, up to Day 183

Sponsors and collaborators

Lead sponsor

Avidity Biosciences, Inc.

Industry

Registry information

Official study title

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

Acronym: MARINA

Important dates

Study start
2021
Primary completion
2023
Study completion
2023
First posted
Aug 30, 2021
Registry last updated
Mar 12, 2026

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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