DM1, Myotonia, Myotonic Disorders, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1, Steinert Disease, Steinert Myotonic Dystrophy
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-OLE, Avidity Biosciences, Avidity, AOC 1001, Del-desiran, Delpacibart etedesiran, HARBOR
Brief summary
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
Detailed description
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.
Interventions
DRUGDel-desiran (AOC 1001)
Del-desiran will be administered by intravenous (IV) infusion.
Sponsors
Avidity Biosciences, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
16 Years to No maximum
Healthy volunteers
No
Inclusion criteria
FROM A PARENT STUDY Key Inclusion Criteria: * Completion of any prior AOC 1001 studies with satisfactory completion of dosing and follow-up assessments and satisfactory compliance with the protocol requirements of the parent study, as determined by the Investigator. Key
Exclusion criteria
* Breastfeeding, pregnancy, or intent to become pregnant during the study. * Unwilling to comply with contraceptive requirements. * Any new conditions or worsening of existing condition that in the opinion of the Investigator would make the participant unsuitable for the study. FIXED-DOSE PK COHORT Key Inclusion Criteria: * Clinical and genetic diagnosis of DM1 and clinically significant hand myotonia. Key
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| TEAEs | From enrollment to the end of treatment at 4 years | Incidence of treatment emergent adverse events |
Countries
Canada, Denmark, France, Germany, Italy, Japan, Netherlands, Spain, United Kingdom, United States
Outcome results
None listed