Trials / Available
AvailableNCT07250737
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
- Phase
- —
- Study type
- Expanded Access
- Enrollment
- —
- Sponsor
- Avidity Biosciences, Inc. · Industry
- Sex
- Male
- Age
- 6 Years
- Healthy volunteers
- —
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.
Conditions
- Muscular Disorders, Atrophic
- Muscular Diseases
- Musculoskeletal Diseases
- Neuromuscular Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Muscular Dystrophies
- Muscular Dystrophy, Duchenne
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | delpacibart zotadirsen | Del-zota is administered as an IV infusion every 6 weeks. Doses are administered based on body weight. |
Timeline
- First posted
- 2025-11-26
- Last updated
- 2026-04-08
Locations
4 sites across 1 country: United States
Source: ClinicalTrials.gov record NCT07250737. Inclusion in this directory is not an endorsement.