Summary

Eligibility
for people ages 18-66 (full criteria)
Location
at UCLA
Dates
study started
completion around

Description

Summary

AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients

Official Title

A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients

Details

This study will continue to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AOC 1001 in participants that enrolled in the randomized, placebo-controlled, First-In-Human Phase 1/2 AOC 1001-CS1 (MARINA) clinical study.

Participants from AOC 1001-CS1 are eligible to enroll in AOC 1001-CS2 if they have satisfactorily completed AOC 1001-CS1.

The total duration of active treatment in AOC 1001-CS2 is approximately 24 months. Once participants have completed active treatment, they will be followed through a 9-month safety follow-up period. The sponsor may extend active treatment beyond 24 months at a future timepoint.

Keywords

DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Disorders, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases, Myotonic Dystrophy Type 1 (DM1), DM, Dystrophy Myotonic, Steinert Disease, MARINA-OLE, MARINA, Avidity Biosciences, Avidity, AOC 1001, Myotonic Muscular Dystrophy, Atrophic Muscular Disorders, Nervous System Heredodegenerative Disorders, Inborn Genetic Diseases

Eligibility

You can join if…

Open to people ages 18-66

- Completion of AOC 1001-CS1 (MARINA) study with satisfactory compliance and no significant tolerability issues

You CAN'T join if...

  • Pregnancy, intent to become pregnant, or active breastfeeding
  • Unwilling or unable to continue to comply with contraceptive requirements
  • Any new conditions or worsening of existing conditions that in the opinion of the investigator or sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study

Locations

  • University of California Los Angeles
    Los Angeles California 90095 United States
  • Stanford University
    Palo Alto California 94304 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Avidity Biosciences, Inc.
ID
NCT05479981
Phase
Phase 2 research study
Study Type
Interventional
Participants
About 37 people participating
Last Updated