A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

Study on Long-Term Effects of an Investigational Medication for Myotonic Dystrophy Type 1 (DM1)

A
Adam Comer

Primary Investigator

Enrolling By Invitation
16 years - 17 years
All
Phase 3
2 participants needed
1 Location

Brief description of study

To evaluate the long-term safety and tolerability of AOC 1001 in DM1 patients.

THIS STUDY IS ENROLLING BY INVITATION ONLY -   The study will invite participants with DM1 who completed a prior study with AOC 1001 to participate.

Detailed description of study

This global Phase 3 open-label extension (OLE) study will continue to evaluate the safety, tolerability, and efficacy of AOC 1001 in DM1 patients who have satisfactorily completed any parent study assessing AOC 1001. All participants who enroll in AOC 1001-CS4 will receive AOC 1001 regardless of whether they received AOC 1001 or placebo in a parent study.

The study consists of a Screening Period of up to either 4-weeks or 8-weeks depending on the prior parent trial, and up to a 4-year Treatment Period. The anticipated duration is 50 months and 2 weeks (4 years and 2.5 months).

Participants will receive an intravenous infusion of del-desiran at the clinical study site every 8 weeks for a total of 7 doses per year. The final dose will occur at Year 4, Visit 7, followed by a final assessment 8 weeks after the last dose.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Steinert Myotonic Dystrophy, Myotonic Dystrophy Type 1, Myotonic Disorders
  • Age: 16 years - 17 years
  • Gender: All

Key Inclusion Criteria:

  • Completion of any prior AOC 1001 studies with satisfactory completion of dosing and follow-up assessments and satisfactory compliance with the protocol requirements of the parent study, as determined by the Investigator.

Key Exclusion Criteria:

  • Breastfeeding, pregnancy, or intent to become pregnant during the study.
  • Unwilling to comply with contraceptive requirements.
  • Any new conditions or worsening of existing condition that in the opinion of the Investigator would make the participant unsuitable for the study.

This study investigates the long-term effects of an investigational medication for Myotonic Dystrophy Type 1 (DM1). DM1 is a genetic disorder that affects muscle function and can cause muscle weakness and stiffness. This study aims to understand how the investigational medication affects people with DM1 over a long period.

Participants will receive the investigational medication through an intravenous infusion at the study site every 8 weeks. This means the medicine is given directly into the blood through a needle. Each participant will get a total of 7 doses each year.

  • Who can participate: Adults who have completed a previous study with the investigational medication and followed all study rules can join. Participants must not be pregnant or planning to become pregnant and must agree to use contraception.
  • Study details: Participants will receive the investigational medication through an intravenous infusion every 8 weeks. The study will monitor the safety and effects of the medication over time.
  • Study timelines: The study will last 50 months. Participants will receive the investigational medication at the study site every 8 weeks, with each participant getting a total of 7 doses each year.
Updated on 03 Nov 2025. Study ID: NEUR-AVIDITY-HARBOR-OLE, 26850
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Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only