In short
Clinical trials are studying SAT-3247 OXALATE in people with Duchenne muscular dystrophy. These studies aim to check safety, tolerability, and whether the treatment may improve muscle force in ambulatory patients. The main trial is a Phase 2 study in patients who can still walk.
Key points
- Clinical trials of SAT-3247 OXALATE are being done in Duchenne muscular dystrophy, a condition that weakens muscles over time. The main study is a Phase 2 trial in ambulatory patients, meaning people who can walk. It compares SAT-3247 OXALATE with placebo in a randomized, double-blind design. The study is looking at both safety and whether muscle force changes after 12 weeks. The trial also checks for changes in physical exams, lab tests, vital signs, ECG, and C-SSRS results.
Trial overview
The available study is an interventional trial of SAT-3247 OXALATE in people with Duchenne muscular dystrophy. The trial title says it is a Phase 2a, randomized, double-blind, placebo-controlled dose comparison and exploratory efficacy study in ambulatory patients. The brief summary says the main goals are to evaluate safety, tolerability, and early signs of benefit on muscle force.
Who can join the study
The study is for ambulatory DMD patients, which means people with Duchenne muscular dystrophy who are still able to walk. No other eligibility details are provided in the source data.
Study design and treatment groups
This is a randomized study, so participants are assigned to study groups by chance. It is also double-blind, which means neither the participants nor the study team knows who is receiving the active study treatment or placebo during the trial. The study compares orally administered SAT-3247 OXALATE tablets with placebo tablets that are made to look the same as the active tablets. The source lists two active tablet strengths, 10 mg and 50 mg, both given by mouth.
What the study measures
The main efficacy endpoint is the change from baseline in muscle force measured by dynamometry at Week 12. Baseline means the starting point before treatment begins, and dynamometry is a test that measures muscle strength. The main safety endpoints include the number, severity, and relationship of adverse events to SAT-3247, plus changes in physical examination, laboratory tests, vital signs, ECG, and C-SSRS results. Adverse events are unwanted health problems during a study, ECG checks the heart’s electrical activity, and C-SSRS is used to check for suicidal thoughts or behavior.
Trial status and size
The trial status is Authorised. The planned enrollment is 51 participants. This is a small early-stage study, so it is mainly designed to learn more about safety and to look for a possible effect on muscle force rather than to give a final answer.