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 44skipping. The patient's Administering Physician should follow the suggested treatmentguidelines and comply with all local health authority regulations.
Not Provided
Drug: delpacibart zotadirsen
Del-zota is administered as an IV infusion every 6 weeks. Doses are administered based on
body weight.
Other Name: Del-zota,AOC 1044
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 6 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
University of Alabama
Birmingham, Alabama, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Rare Disease Research
Kissimmee, Florida, United States
Rare Disease Research
Atlanta, Georgia, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
UMass Memorial Health Care
Worcester, Massachusetts, United States
Gillette Children's
Saint Paul, Minnesota, United States
Rare Disease Research
Iselin, New Jersey, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Prisma Health-Midlands Children's Hospital
Columbia, South Carolina, United States
University of Vermont Medical Center
Burlington, Vermont, United States
Clinigen Customer Service
1 877 768 4303
usmapcoordinators@clinigengroup.com
Not Provided