Avidity Biosciences, Inc.
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…
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…
Del-desiran will be administered by intravenous (IV) infusion.