CompletedPhase 1/2

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

United States39 participantsStarted 2021-10-28

Plain-language 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.

Who can participate

Age range18 Years – 65 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

Frequency of treatment emergent adverse events (TEAEs)

Timeframe: Through study completion, up to Day 183

Trial details

NCT IDNCT05027269

SponsorAvidity Biosciences, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-02-14

View on ClinicalTrials.gov More DM1 trials