DM1, Dystrophy Myotonic, Myotonia, Myotonic Disorders, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1 (DM1), Myotonic Muscular Dystrophy, Steinert, Steinert Disease
Conditions
Keywords
DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Steinert, Myotonic Muscular Dystrophy, HARBOR, Avidity Biosciences, Avidity, AOC 1001, Del-desiran, Delpacibart etedesiran
Brief 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.
Interventions
DRUGAOC 1001 (del-desiran)
Del-desiran will be administered by intravenous (IV) infusion.
DRUGPlacebo
Placebo will be administered by intravenous (IV) infusion.
Sponsors
Avidity Biosciences, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
16 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1 * Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening Key
Exclusion criteria
* Breastfeeding, pregnancy, or intent to become pregnant during the study * Unwilling or unable to comply with contraceptive requirements * Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study * Diabetes that is not adequately controlled * History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded. * Body Mass Index \> 35 kg/m2 at Screening * Recently treated with an investigational drug or biological agent * Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline. Note: Additional protocol defined Inclusion and
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Hand function | Through Week 54 | video Hand Opening Time (vHOT) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Hand grip strength | Through Week 54 | by dynamometer |
| Quantitative Muscle Testing composite score | Through Week 54 | by dynamometer |
| Myotonic Dystrophy Type 1 activity and participation scale c | Through Week 54 | — |
| 10-Meter Walk/Run Test | Through Week 54 | Time (in seconds) to walk or run 10 meters |
Countries
Canada, Denmark, France, Germany, Italy, Japan, Netherlands, Spain, United Kingdom, United States
Outcome results
None listed