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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
230
Del-desiran will be administered by intravenous (IV) infusion.
University of California, Los Angeles (UCLA)
Los Angeles, California, United States
Stanford University
Stanford, California, United States
University of Colorado
Denver, Colorado, United States
University of Florida
Gainesville, Florida, United States
Indiana University (IU)
Indianapolis, Indiana, United States
Kansas University Medical Center
Kansas City, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States
University of Rochester Medical Center
Rochester, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
...and 10 more locations
TEAEs
Incidence of treatment emergent adverse events
Time frame: From enrollment to the end of treatment at 4 years
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