A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind Study to Investigate the Safety, Pharmacodynamics, and Preliminary Efficacy of S-606001 as an Add-on to Enzyme Replacement Therapy in Patients With Late-onset Pompe Disease
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.
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
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. GAA genotype * Participant has a %FVC ≥30% and ≤80% in an upright position without mechanical ventilation at screening; or Participant has ≥10% %FVC drop from upright position to supine position and %FVC ≥20% in a supine position. * Participant performs the 6MWT at screening, as determined by the clin…
Interventions
- DrugS-606001
S-606001 administered orally
- DrugPlacebo
S-606001 matching placebo administered orally
Locations (28)
- University of California - Irvine Medical CenterIrvine, California
- University of Florida (UF) - GainesvilleGainesville, Florida
- Emory University HospitalAtlanta, Georgia
- Washington University in St. LouisSt Louis, Missouri
- Duke University Medical CenterDurham, North Carolina
- Cincinnati Children's Hospital Medical CenterCincinnati, Ohio