A Phase 3 interventional study of Del-desiran (AOC 1001) in Myotonic Dystrophy Type 1, DM1 and Myotonic Dystrophy, sponsored by Avidity Biosciences, Inc.. Enrolling by invitation at 35 sites in 10 countries. Open to participants aged 16 Years and older. Per ClinicalTrials.gov, last updated 2026-08-19.
Sponsored by Avidity Biosciences, Inc. (part of Novartis) · Phase 3, Interventional, and Treatment
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy 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 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.
An additional subgroup of de novo participants will also be included in a Fixed-Dose PK cohort.
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 planned enrollment of 199 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.
FROM A PARENT STUDY
Key Inclusion Criteria:
Key Exclusion Criteria:
FIXED-DOSE PK COHORT
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.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
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.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
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 per year for up to 4 years.
Drug: Del-desiran (AOC 1001)
Del-desiran will be administered by intravenous (IV) infusion.
TEAEs
Incidence of treatment emergent adverse events
Time frame: From enrollment to the end of treatment at 4 years
Plan to share: Undecided — Not yet determined
No publications or documents are linked to this record.
Started Jun 2025 (actual). Primary completion is estimated for Oct 2030, 64 months after the start.
How these figures are worked out, and which studies are left out of them.
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