Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

Part of paid clinical trials in Los Angeles, California.

Sponsor
Avidity Biosciences, Inc.
Study ID
NCT07008469
Phase
PHASE3
Status
Enrolling By Invitation

Conditions

  • DM1
  • Myotonia
  • Myotonic Disorders
  • Myotonic Dystrophy
  • Myotonic Dystrophy 1
  • Myotonic Dystrophy Type 1
  • Steinert Disease
  • Steinert Myotonic Dystrophy

Eligibility Criteria

Sex
ALL
Age
16 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Del-desiran (AOC 1001) — DRUG
    Del-desiran will be administered by intravenous (IV) infusion.

Study Details

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

Key Dates

First listed
Jun 6, 2025
Start date
Jun 26, 2025
Status verified
Aug 2026
Primary completion
Oct 31, 2030
Completion
Oct 31, 2030

Study Design

Enrollment
199 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Del-desiran
    Del-desiran (AOC 1001) will be administered seven times per year for up to 4 years.

Primary Outcome Measure

TEAEs [ Time Frame: From enrollment to the end of treatment at 4 years ]

Locations (19)

FacilityCityStateZIPSite coordinators
University of California, Los Angeles (UCLA)Los AngelesCalifornia90095-
Stanford UniversityStanfordCalifornia94305-
University of ColoradoDenverColorado80045-
University of FloridaGainesvilleFlorida32608-
University Research Center of South FloridaTampaFlorida33612-
Indiana University (IU)IndianapolisIndiana46202-
Kansas University Medical CenterKansas CityKansas66205-
Kennedy Krieger InstituteBaltimoreMaryland21205-
Beth Israel Deaconess Medical CenterBostonMassachusetts02215-
University of MinnesotaMinneapolisMinnesota55455-
University of Rochester Medical CenterRochesterNew York14642-
Duke University Medical CenterDurhamNorth Carolina27708-
Wake ForestWinston-SalemNorth Carolina27157-
University of Cincinnati Gardner Neuroscience InstituteCincinnatiOhio45219-
Ohio State UniversityColumbusOhio43221-
University of PennsylvaniaPhiladelphiaPennsylvania19104-
Houston Methodist Neurological InstituteHoustonTexas77030-
Virginia Commonwealth UniversityRichmondVirginia23298-
University of WashingtonSeattleWashington98104-

Find similar trials in Los Angeles, CA

By research site
University of California, Los Angeles (UCLA)· Los Angeles, CAStanford University· Stanford, CAUniversity of Colorado· Denver, COUniversity of Florida· Gainesville, FLUniversity Research Center of South Florida· Tampa, FLIndiana University (IU)· Indianapolis, IN

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