Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

Recruiting Phase 2 Interventional Study
Pompe Disease
Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
18 and older
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
45 (estimated)
Sponsor
Shionogi · Industry
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About This Trial

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.

Trial Locations
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Eligibility Criteria
Key Inclusion Criteria: * Participant must be ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the informed consent. * Participant must have a diagnosis of LOPD based on documentation of 1 of the following: 1. Deficiency of acid alpha-glucosidase (GAA) enzyme 2. GA…
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Shionogi Clinical Trials Administrator Clinical Support Help Line

800-849-9707

Shionogiclintrials-admin@shionogi.co.jp

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