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NCT07123155 · ClinicalTrials.gov registry record · Phase 2

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

A Phase 2 study of Pompe Disease, sponsored by Shionogi.

Recruiting
Registry status
Phase 2
Development phase
45
Enrollment target
20
Study locations

NCT07123155 is a Phase 2 study of Pompe Disease that is actively recruiting participants, run by Shionogi. The registered enrollment target is 45 participants, below the 229-participant average among 14 other Pompe Disease trials with a reported enrollment target (80% lower). The trial reports 20 study locations across 9 states.

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The verdict

NCT07123155, a Phase 2 study of Pompe Disease, is actively recruiting participants, sponsored by Shionogi.

RECRUITING
Registry status
Phase 2
Development phase
45 participants
Enrollment target
20
Study locations

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.

Conditions Studied

Pompe Disease

Interventions

  • DRUG Placebo
  • DRUG S-606001

Study Locations (20)

Other

  • UZ Leuven - Leuven
  • Aarhus University Hospital - Aarhus
  • HCL Hopital Pierre Wertheimer - Bron
  • AP-HP Hopital Raymond Poincare - Garches
  • Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone - Marseille
  • CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires - Nice
  • Universitaetsklinikum Halle (Saale) - Halle
  • SphinCS GmbH - Höchheim
  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen - München
  • A.O.U. Policlinico "G. Martino" - Messina
  • AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette - Torino

Pennsylvania

  • University of Pennsylvania - Philadelphia
  • University of Pittsburgh School of Medicine - Pittsburgh

California

  • University of California - Irvine Medical Center - Irvine

Florida

  • University of Florida (UF) - Gainesville - Gainesville

Georgia

  • Emory University Hospital - Atlanta

Missouri

  • Washington University in St. Louis - St Louis

North Carolina

  • Duke University Medical Center - Durham

Ohio

  • Cincinnati Children's Hospital Medical Center - Cincinnati

Trial Details

FieldValue
Enrollment Target 45 participants
Start Date 2025-10-30
Est. Completion 2027-08-08
Phase Phase 2

Sponsor

Shionogi

54 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT07123155

The ClinicalTrials.gov registry entry for NCT07123155 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 45 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 229-participant average among 14 other Pompe Disease trials with a reported enrollment target (80% lower). The listed sponsor is Shionogi, which has 54 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Pompe Disease appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.

NCT07123155 reports 20 study locations spanning 9 distinct geographic areas - top geographies include Other, Pennsylvania, California.

Frequently Asked Questions

What is clinical trial NCT07123155 about?

NCT07123155 is a clinical study titled "Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)". 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.

What is the current status of trial NCT07123155?

This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 45 participants. The study started on 2025-10-30. Estimated completion is 2027-08-08.

What conditions does trial NCT07123155 study?

This clinical trial studies the following conditions: Pompe Disease.

What interventions are being tested in trial NCT07123155?

The interventions under investigation include: Placebo (DRUG), S-606001 (DRUG).

Who is sponsoring clinical trial NCT07123155?

This trial is sponsored by Shionogi, which has 54 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT07123155 being conducted?

This trial has 20 study locations across California, Florida, Georgia, Missouri, North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.