A phase 2a study of SAT-3247 in Ambulatory DMD patients (BASECAMP)
Research type
Research Study
Full title
A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients
IRAS ID
1012904
Contact name
Wildon Farwell
Contact email
Sponsor organisation
Satellos Bioscience, Inc.
Eudract number
2025-522522-13
Research summary
The SAT-3247-CL-201 study is exploring a potential new treatment for Duchenne muscular dystrophy (DMD), a rare, progressive genetic condition that causes muscle weakness and loss of mobility primarily in boys, with onset usually occurring early in childhood. DMD is caused by a mutation in the DMD gene and usually leads to increasing difficulty with movement as the disease progresses. While current treatments can help slow progression, there is still an urgent need for new therapies.
This Phase 2a clinical trial will test an investigational medicine called SAT-3247 to see if it is safe, well-tolerated, and has positive effects on muscle strength and function. The study will involve up to 51 boys aged 7 to under 10 years who can still walk and meet other health criteria. Some participants may have previously received other DMD treatments, such as gene therapy or exon-skipping therapy.
Participants will be randomly assigned to receive either SAT-3247 at one of two doses or placebo (a look-alike tablet with no active medicine). SAT-3247 or placebo will be taken by mouth once daily for five days a week over twelve weeks, with placebo tablets on the weekend to keep a consistent daily schedule.
The main goal is to see whether SAT-3247 improves muscle strength, measured using a specialized test called dynamometry. Other key measures include muscle composition and health using MRI scans, muscle biopsies, and tests of everyday physical ability. Researchers will also monitor breathing, muscle cell changes, and other markers to understand how the medicine may be working.
Participants will have regular check-ups, heart and lung monitoring, and blood tests. An independent Data Safety Monitoring Board will review safety information throughout the study.
The results will help determine if SAT-3247 shows potential to move forward into larger trials.REC name
London - Hampstead Research Ethics Committee
REC reference
25/LO/0741
Date of REC Opinion
29 Oct 2025
REC opinion
Further Information Favourable Opinion