ACTIVE_NOT_RECRUITING

Global Study of Del-desiran for the Treatment of DM1

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Conditions

DM1Myotonic DystrophyMyotonic Dystrophy 1MyotoniaMyotonic Dystrophy Type 1 (DM1)Dystrophy MyotonicMyotonic DisordersSteinert DiseaseSteinertMyotonic Muscular DystrophyHARBORAvidity BiosciencesAvidityAOC 1001Del-desiranDelpacibart etedesiran

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Quick Facts

Study Start:2024-05-30
Study Completion:2026-09
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT06411288

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:16 Years to 65 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1
  2. * Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening
  1. * Breastfeeding, pregnancy, or intent to become pregnant during the study
  2. * Unwilling or unable to comply with contraceptive requirements
  3. * Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
  4. * Diabetes that is not adequately controlled
  5. * History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded.
  6. * Body Mass Index \> 35 kg/m2 at Screening
  7. * Recently treated with an investigational drug or biological agent
  8. * Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline.

Contacts and Locations

Study Locations (Sites)

Stanford University
Stanford, California, 94305
United States
University of Colorado
Denver, Colorado, 80045
United States
University of Florida
Gainesville, Florida, 32608
United States
University Research Center of South Florida
Tampa, Florida, 33612
United States
Indiana University (IU)
Indianapolis, Indiana, 46202
United States
Kansas University Medical Center
Kansas City, Kansas, 66205
United States
Kennedy Krieger Institute
Baltimore, Maryland, 21205
United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
United States
University of Minnesota
Minneapolis, Minnesota, 55455
United States
University of Rochester Medical Center
Rochester, New York, 14642
United States
Duke University Medical Center
Durham, North Carolina, 27708
United States
Wake Forest
Winston-Salem, North Carolina, 27157
United States
University of Cincinnati Gardner Neuroscience Institute
Cincinnati, Ohio, 45219
United States
Ohio State University
Columbus, Ohio, 43221
United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
United States
Houston Methodist Neurological Institute
Houston, Texas, 77030
United States
Virginia Commonwealth University
Richmond, Virginia, 23298
United States
University of Washington
Seattle, Washington, 98104
United States

Collaborators and Investigators

Sponsor: Avidity Biosciences, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-05-30
Study Completion Date2026-09

Study Record Updates

Study Start Date2024-05-30
Study Completion Date2026-09

Terms related to this study

Keywords Provided by Researchers

  • 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

Additional Relevant MeSH Terms

  • DM1
  • Myotonic Dystrophy
  • Myotonic Dystrophy 1
  • Myotonia
  • Myotonic Dystrophy Type 1 (DM1)
  • Dystrophy Myotonic
  • Myotonic Disorders
  • Steinert Disease
  • Steinert
  • Myotonic Muscular Dystrophy