A Phase 3 interventional study of AOC 1001 (del-desiran) and Placebo in DM1, Myotonic Dystrophy and Myotonic Dystrophy 1, sponsored by Avidity Biosciences, Inc.. Completed at 34 sites in 10 countries. Open to participants aged 16 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-09-01.
Sponsored by Avidity Biosciences, Inc. · Phase 3, Interventional, and Treatment
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1
The study consists of a Screening Period of up to 6 weeks and 54-week Treatment Period. The anticipated duration is approximately 60 weeks.
Participants will be randomized to receive an intravenous infusion of either del-desiran or placebo at the clinical study site every 8 weeks for a total of 7 doses. The final dose will occur at Week 48, followed by a final assessment at Week 54.
After completion of Week 54 assessments, eligible participants will have the option to enroll into an open label extension (OLE) study, pending regulatory approval.
An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.
Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Additional protocol defined Inclusion and Exclusion criteria apply
Del-desiran (AOC 1001) will be administered seven times
Drug: AOC 1001 (del-desiran)
Saline will be administered seven times
Drug: Placebo
Del-desiran will be administered by intravenous (IV) infusion.
Placebo will be administered by intravenous (IV) infusion.
Hand function
video Hand Opening Time (vHOT)
Time frame: Through Week 54
Hand grip strength
by dynamometer
Time frame: Through Week 54
Quantitative Muscle Testing composite score
by dynamometer
Time frame: Through Week 54
Myotonic Dystrophy Type 1 activity and participation scale c
Time frame: Through Week 54
10-Meter Walk/Run Test
Time (in seconds) to walk or run 10 meters
Time frame: Through Week 54
Plan to share: Undecided
No publications or documents are linked to this record.
This study is completed, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Avidity Biosciences, Inc.