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Clinical Trials in the USA / NCT07123155
Recruiting Phase 2

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

NCT07123155 · tracked via the Priya Life Science USA tracker
Sponsor
Shionogi
Phase
Phase 2
Started
2025-10-30
Last updated
2026-06-23

Condition(s) studied

Pompe Disease

Investigational drug(s) / intervention(s)

S-606001Placebo

S-606001: S-606001 administered orally

Placebo: S-606001 matching placebo administered orally

Study 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

Sex
ALL
Min age
18 Years
Max age
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 clinical evaluator, and meets all of the following criteria: 1. Screening values of 6-minute walk distance (6MWD) are ≥75 meters 2. Screening values of 6MWD are ≤90% of the predicted value for healthy adults * Participants must be ERT-experienced, defined as currently receiving ERT and having been receiving ERT for ≥24 months, with no regimen change in the last 6 months. Key Exclusion Criteria: * Has a medical condition or any other extenuating circumstance that may pose an undue safety risk to the participant or may compromise his/her ability to comply with or adversely impact protocol requirements. * Has active infections at screening. * Malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years. * Current or chronic history of liver disease. * Known biallelic loss of function mutations whether in glycogenin gene (GYG) or in glycogen phosphorylase muscle associated gene(PYGM) . * Has received any investigational therapy or pharmacological treatment for Pompe disease, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before day 1 or is anticipated to do so during the study. * Has received gene therapy or small interfering ribonucleic acid (RNA) therapy for Pompe disease. * Participant, if female, is pregnant or breastfeeding at screening. * Participant, whether male or female, is planning to conceive a child during the study. Note: Other protocol-specified inclusion and exclusion criteria may apply.

Primary outcome measure(s)

Trial sites (28)

FacilityCityRegionStatus
University of California - Irvine Medical Center Irvine California Recruiting
University of Florida (UF) - Gainesville Gainesville Florida Recruiting
Emory University Hospital Atlanta Georgia Recruiting
Washington University in St. Louis St Louis Missouri Recruiting
Duke University Medical Center Durham North Carolina Recruiting
Cincinnati Children's Hospital Medical Center Cincinnati Ohio Recruiting
University of Pennsylvania Philadelphia Pennsylvania Recruiting
University of Pittsburgh School of Medicine Pittsburgh Pennsylvania Recruiting
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) Fairfax Virginia Recruiting
UZ Leuven Leuven Belgium Recruiting
Aarhus University Hospital Aarhus Denmark Recruiting
HLC Hopital Pierre Wertheimer Bron France Recruiting
AP-HP Hopital Raymond Poincare Garches France Recruiting
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone Marseille France Recruiting
CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires Nice France Not Yet Recruiting
Universitaetsklinikum Halle (Saale) Halle Germany Recruiting
SphinCS GmbH Höchheim Germany Recruiting
Klinikum der Ludwig-Maximilians-Universitaet Muenchen München Germany Recruiting
A.O.U. Policlinico "G. Martino" Messina Italy Recruiting
AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette Torino Italy Recruiting
Erasmus MC GE Rotterdam GE Rotterdam Recruiting
Hospital Universitario 12 de Octubre Madrid Spain Recruiting
Hospital Universitari i Politecnic La Fe Valencia Spain Recruiting
Queen Elizabeth Hospital Birmingham Birmingham United Kingdom Recruiting
National Hospital for Neurology & Neurosurgery London United Kingdom Recruiting
Royal Free London NHS Foundation Trust London United Kingdom Recruiting
Royal Victoria Infirmary Newcastle upon Tyne United Kingdom Recruiting
Salford Royal Hospital Salford United Kingdom Recruiting

More Shionogi trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07123155 on ClinicalTrials.gov ↗ ← All trials in the USA