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NCT Number: NCT07750990

An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)

The primary purpose of this study is to evaluate the long-term safety and tolerability profile of S-606001 in participants with LOPD.

Participants who completed other S-606001 studies (for example, NCT07123155), including participants in a S-606001 study whose allocated arm was terminated by the sponsor and who are willing to participate in this study, will be enrolled globally.

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Key information

Conditions

Pompe Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disease Attributes Genetic Diseases, Inborn Glycogen Storage Disease Glycogen Storage Disease Type II Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • Participant must be ≥40 kilograms of body weight at the time of signing the informed consent
  • Participants who previously completed a S-606001 Pompe treatment study, including participants whose assigned treatment arm was terminated by the sponsor

Key Exclusion Criteria:

  • Has a medical condition or any other extenuating circumstance that may, in the opinion of the investigator or medical monitor, pose an undue safety risk to the participant or may compromise his/her ability to comply with or adversely impact protocol requirements
  • Current enrollment in any other clinical study involving an investigational intervention or any other type of medical research
  • Participant, if female, is pregnant or breastfeeding at visit 1
  • Participant, whether male or female, is planning to conceive a child during the study

Note: Other protocol-specified inclusion and exclusion criteria may apply.

Treatment and study plan

S-606001

Drug

S-606001 will be administered orally.

ERT

Drug

ERT will be administered as an intravenous infusion.

Primary outcomes

  1. Number of Participants With Treatment-emergent Adverse Events

    Time frame: Baseline through Week 96

Secondary outcomes

  1. Change from Baseline in Percent Forced Vital Capacity (%FVC)

    Time frame: Baseline, Week 96

  2. Change from Baseline in 6-minute Walk Test (6MWT)

    Time frame: Baseline, Week 96

  3. Change from Baseline in Gait, Stair, Gower's Maneuver, and Chair (GSGC) Score

    Time frame: Baseline, Week 96

  4. Change from Baseline in Daily Living Activity Levels as Assessed by the Investigator

    Time frame: Baseline, Week 96

  5. Change from Baseline in Patient-Reported Outcomes Measurement Information System Fatigue Short Form 8a (PROMIS Fatigue-8a)

    Time frame: Baseline, Week 96

  6. Change from Baseline in Patient-Reported Outcomes Measurement Information System Item Bank v2.0 - Physical Function - Short Form 20a (PROMIS-Item Bank-PF-20a)

    Time frame: Baseline, Week 96

  7. Change from Baseline in Patient-Reported Outcomes Measurement Information System Dyspnea Characteristics (PROMIS-Dyspnea)

    Time frame: Baseline, Week 96

  8. Change from Baseline in Patient-Reported Outcomes Measurement Information System v2.0 Pain Intensity 3a (PROMIS v2.0 Pain)

    Time frame: Baseline, Week 96

  9. Change from Baseline in 36-item Short Form Health Survey (SF-36)

    Time frame: Baseline, Week 96

  10. Change from Baseline in Patient Global Impression of Severity (PGI-S)

    Time frame: Baseline, Week 96

Study contacts

Contact information is provided by the study sponsor or research team.

Shionogi Clinical Trials Administrator Clinical Support Help Line

CONTACT

[email protected]

1-800-849-9707

Sponsors and collaborators

Lead sponsor

Shionogi

Industry

Registry information

Official study title

A Phase 2, Open-label, Multicenter, Extension Study to Investigate the Long-term Safety and Efficacy of S-606001 in Patients With Late-onset Pompe Disease

Important dates

Study start
2026
Primary completion
2028
Study completion
2028
First posted
Aug 6, 2026
Registry last updated
Aug 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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