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
Status: Recruiting
Location: See all (28) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:
• 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:
‣ Deficiency of acid alpha-glucosidase (GAA) enzyme
⁃ 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:
‣ Screening values of 6-minute walk distance (6MWD) are ≥75 meters
⁃ 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.
Locations
United States
California
University of California - Irvine Medical Center
NOT_YET_RECRUITING
Irvine
Florida
University of Florida (UF) - Gainesville
NOT_YET_RECRUITING
Gainesville
Georgia
Emory University Hospital
NOT_YET_RECRUITING
Atlanta
Missouri
Washington University in St. Louis
NOT_YET_RECRUITING
St Louis
North Carolina
Duke University Medical Center
NOT_YET_RECRUITING
Durham
Ohio
Cincinnati Children's Hospital Medical Center
NOT_YET_RECRUITING
Cincinnati
Pennsylvania
University of Pennsylvania
NOT_YET_RECRUITING
Philadelphia
University of Pittsburgh School of Medicine
NOT_YET_RECRUITING
Pittsburgh
Virginia
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)
RECRUITING
Fairfax
Other Locations
Belgium
UZ Leuven
NOT_YET_RECRUITING
Leuven
Denmark
Aarhus University Hospital
RECRUITING
Aarhus
France
HCL Hopital Pierre Wertheimer
NOT_YET_RECRUITING
Bron
AP-HP Hopital Raymond Poincare
NOT_YET_RECRUITING
Garches
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
NOT_YET_RECRUITING
Marseille
CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
NOT_YET_RECRUITING
Nice
Germany
Universitaetsklinikum Halle (Saale)
NOT_YET_RECRUITING
Halle
SphinCS GmbH
NOT_YET_RECRUITING
Höchheim
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
RECRUITING
München
Italy
A.O.U. Policlinico G. Martino
NOT_YET_RECRUITING
Messina
AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette
NOT_YET_RECRUITING
Torino
Netherlands
Erasmus MC
NOT_YET_RECRUITING
Ge Rotterdam
Spain
Hospital Universitario 12 de Octubre
NOT_YET_RECRUITING
Madrid
Hospital Universitari i Politecnic La Fe
NOT_YET_RECRUITING
Valencia
United Kingdom
Queen Elizabeth Hospital Birmingham
NOT_YET_RECRUITING
Birmingham
National Hospital for Neurology & Neurosurgery
NOT_YET_RECRUITING
London
Royal Free London NHS Foundation Trust
NOT_YET_RECRUITING
London
Royal Victoria Infirmary
NOT_YET_RECRUITING
Newcastle Upon Tyne
Salford Royal Hospital
NOT_YET_RECRUITING
Salford
Contact Information
Primary
Shionogi Clinical Trials Administrator Clinical Support Help Line
Shionogiclintrials-admin@shionogi.co.jp
800-849-9707
Time Frame
Start Date:2025-10-30
Estimated Completion Date:2027-08-08
Participants
Target number of participants:45
Treatments
Experimental: S-606001 Low Dose
Participants will receive S-606001 at a low dose level twice daily (BID) after a meal for 52 weeks.
Experimental: S-606001 High Dose
Participants will receive S-606001 at a high dose level BID after a meal for 52 weeks.
Placebo_comparator: Placebo
Participants will receive S-606001 matching placebo BID after a meal for 52 weeks.