Global Study of Del-desiran for the Treatment of DM1

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

Conditions

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

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Study Overview

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Study Details

Study overview

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

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

Global Study of Del-desiran for the Treatment of DM1

Condition
DM1
Intervention / Treatment

-

Contacts and Locations

Los Angeles

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

San Diego

University of California, San Diego (UCSD), San Diego, California, United States, 92093

Stanford

Stanford University, Stanford, California, United States, 94305

Denver

University of Colorado, Denver, Colorado, United States, 80045

Gainesville

University of Florida, Gainesville, Florida, United States, 32608

Tampa

University Research Center of South Florida, Tampa, Florida, United States, 33612

Indianapolis

Indiana University (IU), Indianapolis, Indiana, United States, 46202

Kansas City

Kansas University Medical Center, Kansas City, Kansas, United States, 66205

Baltimore

Kennedy Krieger Institute, Baltimore, Maryland, United States, 21205

Boston

Beth Israel Deaconess Medical Center, Boston, Massachusetts, United States, 02215

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.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * 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
  • * 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.

Ages Eligible for Study

16 Years to 65 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Avidity Biosciences, Inc.,

Study Record Dates

2027-04