Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
Sponsored by Avidity Biosciences, Inc.
About this trial
Last updated 16 days ago
Study ID
Status
Type
Placebo
Accepting
What is this trial about?
What are the Participation Requirements?
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
