An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
ConditionPompe Disease
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorShionogi
About this trial
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
Qualifiers
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
Disqualifiers
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
Trial design
Treatments tested in this trial
- S-606001
- ERT
Treatment groups
60 Participants
are divided into 1 treatment groupLocations
Sponsors and collaborators
Source ClinicalTrials.gov