A Phase 2 interventional study of S-606001 and ERT in Pompe Disease, sponsored by Shionogi. Not yet recruiting. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-13.
Sponsored by Shionogi · Phase 2, Interventional, and Treatment
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.
162 studies on the registry are indexed under Glycogen Storage Disease Type II; 34 are open to participants now.
This study's planned enrollment of 60 is above the median of 20 across 89 interventional studies indexed under Glycogen Storage Disease Type II.
Browse Glycogen Storage Disease Type II studies →Shionogi is the lead sponsor of 105 studies on the registry; 10 are open to participants now.
Of its 39 completed or terminated interventional studies of FDA-regulated products, 21 (54%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Other protocol-specified inclusion and exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive S-606001. Participants who were previously using enzyme replacement therapy (ERT) at the time of enrollment will receive the same regimen of ERT as standard of care.
Drug: S-606001 · Drug: ERT
Number of Participants With Treatment-emergent Adverse Events
Time frame: Baseline through Week 96
Change from Baseline in Percent Forced Vital Capacity (%FVC)
Time frame: Baseline, Week 96
Change from Baseline in 6-minute Walk Test (6MWT)
Time frame: Baseline, Week 96
Change from Baseline in Gait, Stair, Gower's Maneuver, and Chair (GSGC) Score
Time frame: Baseline, Week 96
Change from Baseline in Daily Living Activity Levels as Assessed by the Investigator
Time frame: Baseline, Week 96
Change from Baseline in Patient-Reported Outcomes Measurement Information System Fatigue Short Form 8a (PROMIS Fatigue-8a)
Time frame: Baseline, Week 96
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
Change from Baseline in Patient-Reported Outcomes Measurement Information System Dyspnea Characteristics (PROMIS-Dyspnea)
Time frame: Baseline, Week 96
Change from Baseline in Patient-Reported Outcomes Measurement Information System v2.0 Pain Intensity 3a (PROMIS v2.0 Pain)
Time frame: Baseline, Week 96
Change from Baseline in 36-item Short Form Health Survey (SF-36)
Time frame: Baseline, Week 96
Change from Baseline in Patient Global Impression of Severity (PGI-S)
Time frame: Baseline, Week 96
No study locations are listed for this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is not yet recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: S-606001
Glycogen Storage Disease Type II →