Summary

Eligibility
for people ages 18-65 (full criteria)
Location
at UCLA
Dates
study started
estimated completion

Description

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 3 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.

Official 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

Keywords

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

Eligibility

You can join if…

Open to people ages 18-65

  • 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 CAN'T join 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

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

Locations

  • University of California Los Angeles accepting new patients
    Los Angeles California 90095 United States
  • Stanford University accepting new patients
    Palo Alto California 94304 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Avidity Biosciences, Inc.
Links
Related Info
ID
NCT05027269
Phase
Phase 1/2 research study
Study Type
Interventional
Participants
Expecting 44 study participants
Last Updated