RecruitingPhase 2NCT07123155

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

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

Sponsor

Shionogi

Enrollment

45 participants

Start Date

Oct 30, 2025

Study Type

INTERVENTIONAL

Conditions

Pompe Disease

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

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Placebo and a drug called S-606001 for people with pompe disease. The study is currently recruiting participants at 28 locations. People eligible for this study include aged 18 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGS-606001

S-606001 administered orally

DRUGPlacebo

S-606001 matching placebo administered orally

Locations(28)

University of California - Irvine Medical Center

Irvine, California, United States

University of Florida (UF) - Gainesville

Gainesville, Florida, United States

Emory University Hospital

Atlanta, Georgia, United States

Washington University in St. Louis

St Louis, Missouri, United States

Duke University Medical Center

Durham, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

University of Pittsburgh School of Medicine

Pittsburgh, Pennsylvania, United States

Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)

Fairfax, Virginia, United States

UZ Leuven

Leuven, Belgium

Aarhus University Hospital

Aarhus, Denmark

HLC Hopital Pierre Wertheimer

Bron, France

AP-HP Hopital Raymond Poincare

Garches, France

Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone

Marseille, France

CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires

Nice, France

Universitaetsklinikum Halle (Saale)

Halle, Germany

SphinCS GmbH

Höchheim, Germany

Klinikum der Ludwig-Maximilians-Universitaet Muenchen

München, Germany

A.O.U. Policlinico "G. Martino"

Messina, Italy

AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette

Torino, Italy

Erasmus MC

GE Rotterdam, GE Rotterdam, Netherlands

Hospital Universitario 12 de Octubre

Madrid, Spain

Hospital Universitari i Politecnic La Fe

Valencia, Spain

Queen Elizabeth Hospital Birmingham

Birmingham, United Kingdom

National Hospital for Neurology & Neurosurgery

London, United Kingdom

Royal Free London NHS Foundation Trust

London, United Kingdom

Royal Victoria Infirmary

Newcastle upon Tyne, United Kingdom

Salford Royal Hospital

Salford, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT07123155

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