A Phase 3 Global Study to Evaluate AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

• Research type

  Research Study

• Full title

  A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

• IRAS ID

  1010023

• Contact name

  Jazmine Cruz-Maddox

• Contact email

  jazmine.cruz-maddox@aviditybio.com

• Sponsor organisation

  Avidity Biosciences, Inc.

• Clinicaltrials.gov Identifier

  NCT06411288

• Research summary

  HARBOR: A Phase 3 Study to Evaluate AOC 1001 in Patients with DM1

  Study Objective:
  The main purpose of this study is to measure efficacy and safety of AOC 1001 in patients with DM1.

  Study Design:
  The HARBOR study is planned to be conducted in multiple different clinics in several countries. The study will test AOC 1001 or placebo in a total of 150 participants with DM1. Patients may be eligible to participate if they are between 16 and 65 years old, are confirmed to have DM1 by clinical and genetic diagnosis, can walk on their own for 10 meters, are willing and able to perform the tests in the study, and have agreed to take part in the study. If a patient has any medical conditions that may pose a safety risk or make it difficult for them to participate in the study, they may not be included. Pregnant people or people who are breastfeeding will not be included. Study staff will explain all details of the study to participants.

  Study drug will be given by intravenous infusion (through a needle directly into the blood stream). The study will randomly assign participants to be treated with AOC 1001 or placebo for a total of 7 doses. Participants and study staff will not know which participants receive AOC 1001 or the placebo.

  Efficacy endpoints include measures of hand function, grip strength, strength of arm and leg muscles, and time to walk 10 meters. Participants will also answer questionnaires during the study about their quality of life and ability to perform normal functions of daily living while on the study. The efficacy endpoints before and after treatment will be compared between AOC 1001 and placebo groups.

  Study staff will collect information on changes in medical status reported by participants. Study participants will provide blood samples to study staff according to the study schedule to measure drug levels and to assess safety based on laboratory analyses.

• REC name

  North of Scotland Research Ethics Committee 1

• REC reference

  24/NS/0073

• Date of REC Opinion

  25 Jul 2024

• REC opinion

  Further Information Favourable Opinion