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

Avidity Biosciences, Inc.38 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

DM1 Myotonic Dystrophy 1 Myotonic Dystrophy Myotonic Dystrophy Type 1 (DM1)

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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.

Locations (8)

University of California Los Angeles

Los Angeles, California, United States

Stanford University

Palo Alto, California, United States

University of Colorado

Denver, Colorado, United States

University of Florida

Gainesville, Florida, United States

Kansas University Medical Center

Kansas City, Kansas, United States

University of Rochester Medical Center

Rochester, New York, United States

Ohio State University

Columbus, Ohio, United States

Virginia Commonwealth University

Richmond, Virginia, United States