This study is accepting new patients by invitation only

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

a study on Myotonic Dystrophy Myotonia Myotonic Disorders

Summary

Eligibility
for people ages 16 years and up (full criteria)
Location
at UCLA
Dates
study started
study ends around
Principal Investigator
by Payam Soltanzadeh, MD (ucla)
Headshot of Payam Soltanzadeh
Payam Soltanzadeh

Description

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

Official Title

A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

Details

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 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.

Keywords

Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia, Myotonic Dystrophy 1, Myotonic Disorders, Steinert Myotonic Dystrophy, Steinert's Disease, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Steinert Disease, Steinert, Myotonic Muscular Dystrophy, HARBOR-OLE, Avidity Biosciences, Avidity, AOC 1001, Del-desiran, Delpacibart etedesiran, HARBOR, Del-desiran (AOC 1001)

Eligibility

You can join if…

Open to people ages 16 years and up

  • 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.

You CAN'T join if...

  • 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.

Locations

  • University of California, Los Angeles (UCLA)
    Los Angeles 5368361 California 5332921 90095 United States
  • Stanford University
    Stanford 5398563 California 5332921 94305 United States

Lead Scientist at University of California Health

  • Payam Soltanzadeh, MD (ucla)
    HS Associate Clinical Professor, Neurology, Medicine. Authored (or co-authored) 17 research publications

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Avidity Biosciences, Inc.
ID
NCT07008469
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 217 study participants
Last Updated