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

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

Status
AVAILABLE
Phases
Unknown
Study type
Expanded Access
Source
ClinicalTrials.gov
Registry ID
NCT07250737
Enrollment
Unknown
Registered
2025-11-26
Start date
Unknown
Completion date
Unknown
Last updated
2026-09-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

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

Keywords

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

Brief 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.

Interventions

DRUGdelpacibart zotadirsen

Del-zota is administered as an IV infusion every 6 weeks. Doses are administered based on body weight.

Sponsors

Avidity Biosciences, Inc.
Lead SponsorINDUSTRY
Clinigen Healthcare Limited
CollaboratorUNKNOWN

Eligibility

Sex/Gender
MALE
Age
2 Years to No maximum

Inclusion criteria

Key Inclusion Criteria 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 Key

Exclusion criteria

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

Countries

United States

Contacts

CONTACTClinigen Customer Service
usmapcoordinators@clinigengroup.com1 877 768 4303

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Sep 2, 2026