This study is not accepting new patients

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

a study on Atrophic Muscular Disorders Myopathy Musculoskeletal Diseases Muscular Dystrophies Duchenne Muscular Dystrophy

Summary

Eligibility
for males ages 2 years and up (full criteria)
Location
at UC Irvine
Dates
study started

Description

Summary

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.

Official Title

Managed Access to Investigational Use of AOC 1044 in Participants With DMD Mutations Amenable to Exon 44 Skipping

Keywords

Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Muscular Dystrophies, Muscular Dystrophy, Duchenne, Avidity, Avidity Biosciences, AOC 1044, AOC 1044-CS1, AOC 1044-CS2, EXPLORE44, EXPLORE44-OLE, Del-zota, delpacibart zotadirsen, DMD, exon skipping therapy, dystrophin, managed access, expanded access, pre-approval access, compassionate use, MAP, EAP, Atrophic Muscular Disorders, Inborn Genetic Diseases, X-Linked Genetic Diseases, Duchenne Muscular Dystrophy

Eligibility

You can join if…

Open to males ages 2 years and up

Rollover Participants

  • Completed Study EXPLORE44-OLE Treatment Period (through W102)
  • No significant tolerability issues with AOC 1044

New (Non-Rollover) Participants

  • Permanently residing in the US and have a US primary health care provider
  • Documented dystrophin gene mutation that is amenable to exon 44 skipping
  • Age 2 or older at the time of consent
  • If previously treated with gene therapy for DMD, treatment and associated immunosuppressive regimen was more than 12 months before consent and in the opinion of the prescriber, participant has had an unsatisfactory treatment response

You CAN'T join if...

Rollover Participants

• Prescence of any new condition or worsening of existing condition that could affect participant's safety or ability to comply with the program requirements

New (Non-Rollover) Participants

  • Recently treated with or on a clinical study for another investigation drug
  • Serious respiratory or cardiac dysfunction, or nearing end of life
  • Screening laboratory parameters do not meet protocol requirements
  • History of multiple drug allergies or to any component of AOC 1044
  • Participants who discontinued early from the treatment period of EXPLORE44 or EXPLORE44-OLE

Locations

  • UCI Alpha Clinic not accepting new patients
    Irvine California 92612 United States
  • University of Kansas Medical Center not accepting new patients
    Kansas City Kansas 66103 United States

Details

Status
not accepting new patients
Start Date
Sponsor
Avidity Biosciences, Inc.
ID
NCT07250737
Study Type
Expanded Access
Last Updated
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