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. (part of Novartis) · 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.
126 studies on the registry are indexed under Myotonic Dystrophy; 50 are open to participants now.
This study's enrollment of 159 is above the median of 40 across 67 interventional studies indexed under Myotonic Dystrophy.
Browse Myotonic Dystrophy studies →Avidity Biosciences, Inc. is the lead sponsor of 11 studies on the registry; 4 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 1 (20%) have results posted.
First submitted before Avidity Biosciences, Inc. became part of Novartis.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Additional protocol defined Inclusion and Exclusion criteria apply
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Ran from May 2024 to primary completion in Jul 2026, 25 months.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: AOC 1001