Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Avidity Biosciences, Inc.
Study ID
NCT07250737
Status
Available

Conditions

  • Genetic Diseases, Inborn
  • Genetic Diseases, X-Linked
  • Muscular Diseases
  • Muscular Disorders, Atrophic
  • Muscular Dystrophies
  • Muscular Dystrophy, Duchenne
  • Musculoskeletal Diseases
  • Nervous System Diseases
  • Neuromuscular Diseases

Eligibility Criteria

Sex
MALE
Age
2 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • delpacibart zotadirsen — DRUG
    Del-zota is administered as an IV infusion every 6 weeks. Doses are administered based on body weight.

Study Details

The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

Key Dates

First listed
Nov 26, 2025
Status verified
Aug 2026

Central Contacts

Locations (19)

FacilityCityStateZIPSite coordinators
University of AlabamaBirminghamAlabama35294-
Arkansas Children's HospitalLittle RockArkansas72202-
UCI Alpha ClinicIrvineCalifornia92612-
UC San Diego HealthSan DiegoCalifornia92121-
Connecticut Children's Medical CenterHartfordConnecticut06106-
Rare Disease ResearchKissimmeeFlorida34746-
Nicklaus Children's HospitalMiamiFlorida33155-
Rare Disease ResearchAtlantaGeorgia30329-
University of Kansas Medical CenterKansas CityKansas66103-
UMass Memorial Health CareWorcesterMassachusetts01583-
Gillette Children'sSaint PaulMinnesota55101-
Washington UniversitySt LouisMissouri63110-
Rare Disease ResearchIselinNew Jersey08830-
Nationwide Children's HospitalColumbusOhio43205-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
University of PennsylvaniaPhiladelphiaPennsylvania19104-
Prisma Health-Midlands Children's HospitalColumbiaSouth Carolina29203-
Neurology & Neuromuscular Care CenterFlower MoundTexas75028-
University of Vermont Medical CenterBurlingtonVermont05401-

Find similar trials in Birmingham, AL

By research site
University of Alabama· Birmingham, ALArkansas Children's Hospital· Little Rock, ARUCI Alpha Clinic· Irvine, CAUC San Diego Health· San Diego, CAConnecticut Children's Medical Center· Hartford, CTRare Disease Research· Kissimmee, FL

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