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 Steinert Disease

Summary

Eligibility
for people ages 16 years and up (full criteria)
Location
at UCLA
Dates
study started
study ends around

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 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.

Keywords

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

Eligibility

For people ages 16 years and up

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 Exclusion Criteria:

  • Breastfeeding, pregnancy, or intent to become pregnant during the study
  • Unwilling 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

Note: Additional protocol defined Inclusion and Exclusion criteria apply

Locations

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

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 230 study participants
Last Updated