AOC 1001 for Myotonic Dystrophy
(MARINA-OLE Trial)
Trial Summary
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.
How is the drug AOC 1001 unique for treating myotonic dystrophy type 1?
AOC 1001 is a novel nucleic acid-based therapy, which is different from traditional treatments as it targets the genetic cause of myotonic dystrophy type 1 by potentially correcting the mis-splicing of genes involved in the disease. This approach is part of a new wave of therapies aiming to address the underlying genetic issues rather than just managing symptoms.12345
What is the purpose of this trial?
This trial is testing a new medicine called AOC 1001 to see if it is safe and effective for adults with a muscle disease called Myotonic Dystrophy Type 1. The medicine is given through an IV, and researchers want to know if it helps muscles work better.
Research Team
Li Tai, MD
Principal Investigator
Avidity Biosciences, Inc.
Eligibility Criteria
This trial is for adults with Myotonic Dystrophy Type 1 who completed the MARINA study without major issues. They must be able to continue using contraception and not be pregnant or breastfeeding. Those with new or worsening conditions that could affect their participation are excluded.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive AOC 1001 administered intravenously every 8 weeks, with an additional dose on Day 43
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants may continue active treatment beyond 58 months if extended by the sponsor
Treatment Details
Interventions
- AOC 1001
Find a Clinic Near You
Who Is Running the Clinical Trial?
Avidity Biosciences, Inc.
Lead Sponsor