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

NCT05027269 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 39

Last updated 2026-03-12

No results posted yet for this study

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

Interventions

DRUG

AOC 1001

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

DRUG

Placebo

Placebo will be administered by intravenous (IV) infusion.

Sponsors & Collaborators

  • Avidity Biosciences, Inc.

    lead INDUSTRY

Principal Investigators

  • Li Tai, MD · Avidity Biosciences, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-10-28
Primary Completion
2023-02-14
Completion
2023-02-14
FDA Drug
Yes

Countries

  • United States

Study Locations

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Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05027269 on ClinicalTrials.gov