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NCT07750990
An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
Conditions: Pompe Disease
Sex: All
Ages: 18 Years – N/A
Healthy volunteers: No
Phase: PHASE2
Enrollment: 60
Sponsor: Shionogi
Summary
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.
Eligibility Criteria
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.
Source: ClinicalTrials.gov (NCT07750990). StuddyBuddy aggregates publicly available trial information.