AOC 1044 Exon 44 Skipping Therapy for Duchenne Muscular Dystrophy: SAFARI44 Clinical Trial
Not Yet RecruitingPhase 3Exon Skipping
Study Overview
- Age
- 6–16 years
- Phase
- Phase 3
- Sponsor
- Avidity Biosciences
- Therapeutic Approach
- Exon Skipping
- Variant Requirement
- Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping.
- Eligible Sex
- Male
- Ambulation
- Ambulatory
- Study Start (Actual)
- 2026-06
- Primary Completion (Estimated)
- 2029-05
- Study Completion (Estimated)
- 2030-07
- Enrollment (Estimated)
- 70
- Countries
- United States
Study Requirements and Criteria
Steroid Use
On a stable regimen of corticosteroids (including Vamolorone) for at least 6 months prior to Day 1. Steroid regimen must be anticipated to remain stable.
Inclusion Criteria
- Ambulatory males with clinical and genetic diagnosis of DMD
- 7 to 16 years of age at time of consent
- TTR and NSAA assessment completed within the protocol specified parameters at Screening
Exclusion Criteria
- Previous treatment cell or gene therapy.
- Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines).
- Lab values outside of the protocol specified range at Screening
- If on any of the following treatments (growth hormone, testosterone or givinostat), participants must be on a stable regimen and must plan to maintain it for the duration of the study. Participants will be excluded if regimen stability prior to informed consent is as follows:
- Less than 1 month, for growth hormone and/or testosterone
- Less than 6 months for givinostat
Contact Information
This section provides contact details for people who can answer questions about joining this study
- Name: Avidity Bioscience, Inc., A Novartis Company
- Phone Number: 858-771-7038
- Email: [email protected]
Clinical Trial Registry
NCT ID
NCT07587242This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.