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

AC

Adam Comer

Principal Investigator

Status: Enrolling By Invitation Ages: 16 Years - 17 Years Gender: All Genders Phase: 3 1 Locations

Brief Description

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

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

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.

Interested in participating?

Enrollment in IU School of Medicine clinical trials is managed through our All IN for Health program. Many studies only accept people who are part of an invited group. Visit allinforhealth.info to connect with the study team, or contact your doctor to see if you are eligible.