Trials / Active Not Recruiting
Active Not RecruitingNCT06411288
Global Study of Del-desiran for the Treatment of DM1
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
- Status
- Active Not Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 159 (actual)
- Sponsor
- Avidity Biosciences, Inc. · Industry
- Sex
- All
- Age
- 16 Years – 65 Years
- Healthy volunteers
- Not accepted
Summary
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
Detailed description
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.
Conditions
- DM1
- Myotonic Dystrophy
- Myotonic Dystrophy 1
- Myotonia
- Myotonic Dystrophy Type 1 (DM1)
- Dystrophy Myotonic
- Myotonic Disorders
- Steinert Disease
- Steinert
- Myotonic Muscular Dystrophy
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | AOC 1001 (del-desiran) | Del-desiran will be administered by intravenous (IV) infusion. |
| DRUG | Placebo | Placebo will be administered by intravenous (IV) infusion. |
Timeline
- Start date
- 2024-05-30
- Primary completion
- 2026-08-01
- Completion
- 2026-09-01
- First posted
- 2024-05-13
- Last updated
- 2026-01-22
Locations
34 sites across 10 countries: United States, Canada, Denmark, France, Germany, Italy, Japan, Netherlands, Spain, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06411288. Inclusion in this directory is not an endorsement.