Global Study of Del-desiran for the Treatment of DM1

a study on Myotonic Dystrophy Myotonia Myotonic Disorders Steinert Disease Steinert

Summary

Eligibility
for people ages 16-65 (full criteria)
Location
at UCLA UCSD
Dates
study started
completion around

Description

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

Official Title

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

Details

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.

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, Muscular Dystrophies, AOC 1001 (del-desiran)

Eligibility

You can join if…

Open to people ages 16-65

  • 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

You CAN'T join if...

  • 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 Exclusion criteria apply

Locations

  • University of California, Los Angeles (UCLA) not yet accepting patients
    Los Angeles California 90095 United States
  • University of California, San Diego (UCSD) accepting new patients
    San Diego California 92093 United States
  • Stanford University accepting new patients
    Stanford California 94305 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Avidity Biosciences, Inc.
Links
HARBOR Study Website
ID
NCT06411288
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 150 study participants
Last Updated