Can an oral pill boost muscle strength in pompe disease?
NCT ID NCT07750990
First seen Aug 06, 2026 · Last updated Aug 07, 2026 · Updated 1 time
Summary
This study tests the long-term safety and effectiveness of an experimental oral drug called S-606001 in adults with late-onset Pompe disease, a genetic condition that causes progressive muscle weakness. Participants who completed a prior S-606001 study can join, and they will receive S-606001 alongside their standard enzyme replacement therapy. Researchers will track side effects and measure changes in breathing, walking ability, and daily activities over time.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- S-606001, an oral drug, plus standard enzyme replacement therapy (ERT) given intravenously
- What this could lead to
- If successful, this could support S-606001 as a long-term add-on treatment to improve muscle function and daily living in people with late-onset Pompe disease.
- What could go wrong
- This is an early-phase extension study with a small number of participants, so long-term benefits are uncertain. Possible side effects and the need for continued ERT remain.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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Other studies related to the condition(s) this trial covers.
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