S-606001 for Pompe Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial examines the safety and effectiveness of a new treatment, S-606001, for adults with late-onset Pompe disease (LOPD), a rare genetic disorder affecting muscle function. Participants will receive either a low or high dose of S-606001 (an experimental treatment) or a placebo (a non-active substance) for 52 weeks. The trial seeks individuals who have been on enzyme replacement therapy (ERT) for at least two years without changing their routine and have been diagnosed with LOPD. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.
Do I have to stop taking my current medications for the trial?
The trial does not specify if you need to stop taking your current medications, but you must continue your existing enzyme replacement therapy (ERT) without changes for at least 6 months before joining the study.
Is there any evidence suggesting that S-606001 is likely to be safe for humans?
Research has shown that S-606001 has undergone safety testing in humans. In a previous study, 121 healthy adults received the treatment to assess its safety. The results indicated that most participants did not experience serious problems, demonstrating that the treatment was generally well-tolerated.
The study did not report any major side effects. So far, evidence suggests the treatment is safe to use. However, since the current study is in Phase 2, researchers are still closely monitoring the treatment. This phase is crucial for checking safety in individuals with the condition, ensuring it is safe for everyone.12345Why do researchers think this study treatment might be promising for Pompe Disease?
Researchers are excited about S-606001 for Pompe disease because it introduces a potentially groundbreaking approach distinct from current enzyme replacement therapies like alglucosidase alfa. Unlike these standard treatments that supplement missing enzymes, S-606001 may offer a novel mechanism by targeting the underlying cellular processes differently. With both low and high dose options being explored, there's potential for tailored treatment that could optimize efficacy and minimize side effects, providing a significant advancement in managing Pompe disease.
What evidence suggests that S-606001 might be an effective treatment for Pompe Disease?
Research has shown that S-606001 might help people with late-onset Pompe disease (LOPD) when combined with enzyme replacement therapy (ERT). This new treatment could work by breaking down glycogen, a type of sugar that accumulates in muscle cells and causes problems in Pompe disease. In this trial, participants will receive either a low dose or a high dose of S-606001, or a placebo. Some patients have demonstrated that treatments like S-606001 might be more effective than alglucosidase alfa, the current standard treatment. While more information is needed, early results appear promising for those with LOPD.12367
Are You a Good Fit for This Trial?
Adults with late-onset Pompe Disease who weigh at least 40 kg and have been on enzyme replacement therapy for over 2 years can join. They must be able to walk a certain distance and have specific lung function values without needing mechanical ventilation.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive S-606001 or placebo as an add-on to enzyme replacement therapy for 52 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- S-606001
Find a Clinic Near You
Who Is Running the Clinical Trial?
Shionogi
Lead Sponsor
Dr. Isao Teshirogi
Shionogi
Chief Executive Officer since 2008
PhD in Pharmaceutical Sciences from the University of Tokyo
Dr. Takuko Sawada
Shionogi
Chief Medical Officer since 2022
MD from a recognized institution (specific details not found)