Trial Now Recruiting: Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) (NCT07123155)
WHY IT MATTERS
This trial is actively recruiting adults with late-onset Pompe disease who are already on enzyme replacement therapy — eligible participants may be able to access an investigational add-on treatment that could potentially improve their muscle function.
Researchers are testing a new drug called S-606001 to see if it can help adults with late-onset Pompe disease when added to their current enzyme replacement therapy (ERT) treatment. Pompe disease is a rare genetic condition where the body can't break down a type of sugar, causing muscle weakness over time. This study will check if the new drug is safe and if it helps patients feel better or move more easily. The trial is currently looking for 45 adult participants to join.
NCT ID: NCT07123155 Status: RECRUITING Conditions: Pompe Disease Phase: PHASE2 Enrollment: 45 Sponsor: Shionogi Summary: The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.
YOU CAN ACT ON THIS
If you have late-onset Pompe disease and are currently on ERT, contact your neurologist or metabolic disease specialist to ask if you meet the eligibility criteria for NCT07123155, or visit clinicaltrials.gov to find participating sites near you.