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

March 10, 2026 updated by: Avidity Biosciences, Inc.

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

39

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90095
        • University of California Los Angeles
      • Palo Alto, California, United States, 94304
        • Stanford University
    • Colorado
      • Denver, Colorado, United States, 80045
        • University of Colorado
    • Florida
      • Gainesville, Florida, United States, 32608
        • University of Florida
    • Kansas
      • Kansas City, Kansas, United States, 66205
        • Kansas University Medical Center
    • New York
      • Rochester, New York, United States, 14642
        • University of Rochester Medical Center
    • Ohio
      • Columbus, Ohio, United States, 43221
        • Ohio State University
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Virginia Commonwealth University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years to 61 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Part A Single Dose: AOC 1001 Dose Level 1
AOC 1001 will be administered once.
Drug: AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.
Placebo Comparator: Part A Single Dose: Placebo
Saline will be administered once.
Drug: Placebo
Placebo will be administered by intravenous (IV) infusion.
Placebo Comparator: Part B Multiple Ascending Dose: Placebo
Saline will be administered three times.
Drug: Placebo
Placebo will be administered by intravenous (IV) infusion.
Experimental: Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3
AOC 1001 will be administered three times.
Drug: AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Frequency of treatment emergent adverse events (TEAEs)
Time Frame: Through study completion, up to Day 183
Through study completion, up to Day 183

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Plasma pharmacokinetic (PK) parameters
Time Frame: Through study completion, up to Day 183
Maximum plasma concentration (Cmax)
Through study completion, up to Day 183
Plasma pharmacokinetic (PK) parameters
Time Frame: Through study completion, up to Day 183
Time to maximum plasma concentration (Tmax)
Through study completion, up to Day 183
Plasma pharmacokinetic (PK) parameters
Time Frame: Through study completion, up to Day 183
Terminal Half-life (t1/2)
Through study completion, up to Day 183
Plasma pharmacokinetic (PK) parameters
Time Frame: Through study completion, up to Day 183
Area Under the Concentration-time Curve (AUC)
Through study completion, up to Day 183
Urine pharmacokinetic (PK) parameters
Time Frame: Through study completion, up to Day 183
fraction excreted (fe) in urine
Through study completion, up to Day 183
AOC 1001 levels in muscle tissue
Time Frame: Through study completion, up to Day 183
Through study completion, up to Day 183
Change and percentage change from baseline in DMPK mRNA knockdown
Time Frame: Through study completion, up to Day 183
Through study completion, up to Day 183
Change and percentage change from baseline in Spliceopathy
Time Frame: Through study completion, up to Day 183
Through study completion, up to Day 183

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Avidity Biosciences, Inc.

Investigators

  • Study Director: Li Tai, MD, Avidity Biosciences, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 28, 2021

Primary Completion (Actual)

February 14, 2023

Study Completion (Actual)

February 14, 2023

Study Registration Dates

First Submitted

August 24, 2021

First Submitted That Met QC Criteria

August 24, 2021

First Posted (Actual)

August 30, 2021

Study Record Updates

Last Update Posted (Actual)

March 12, 2026

Last Update Submitted That Met QC Criteria

March 10, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AOC 1001-CS1

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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