An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101
Part of paid clinical trials in Los Angeles, California.
- Sponsor
- Satellos Bioscience, Inc.
- Study ID
- NCT06867107
- Phase
- PHASE2
- Status
- Enrolling By Invitation
Conditions
- Duchenne Muscular Dystrophy (DMD)
Eligibility Criteria
- Sex
- MALE
- Age
- 16 Years - 40 Years
- Healthy Volunteers
- Not accepted
Interventions
- SAT-3247 — DRUGAAK1 inhibitor
Study Details
This is an open-label long-term safety and efficacy study of orally administered SAT-3247 in patients with DMD that previously participated in SAT-3247-CL-101. The study will assess the long-term safety, tolerability and potential efficacy of long-term dosing of 60 mg of orally administered SAT-3247 in a 5-days on/2-days off (i.e. weekday dosing) regimen in an open-label design through 25 months- for a total of 24 months of treatment including the duration of the SAT-3247-CL-101 study. The study will enroll up to 30 participants including those that previously participated in the SAT-3247-CL-101 study.
Key Dates
- First listed
- Mar 10, 2025
- Start date
- Aug 20, 2025
- Status verified
- Sep 2026
- Primary completion
- Sep 30, 2027
- Completion
- Sep 30, 2027
Study Design
- Enrollment
- 30 participants (estimated)
- Allocation
- NA
- Intervention model
- SINGLE_GROUP
- Primary purpose
- TREATMENT
Arms
- Experimental: Treatment ArmSAT-3247 60 mg administered orally in a 5-days on/2-days off (weekday) dosing regimen
Primary Outcome Measure
Treatment emergent adverse events [ Time Frame: 24 months ]
Locations (3)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| University of California, Los Angeles | Los Angeles | California | 90095 | - |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | - |
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | - |
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