An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD) | NCT07750990

NCT07750990

Overview

NCT ID: NCT07750990
Lead Sponsor: ShionogiINDUSTRY
Official 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
Brief 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.
Conditions: Pompe Disease
Interventions

Status and Timeline

Status: Not yet recruiting
Last Update Posted: Aug 6, 2026Actual
Start Date: Aug 31, 2026Estimated
Primary Completion Date: Aug 31, 2028Estimated

Participation Criteria

Ages Eligible for Study: Adult (18-64)Older Adult (65+)
Sexes Eligible for Study: All
Accepts Healthy Volunteers?: No
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.

Study Plan

Study Type: Interventional
Phase: Phase 2
Enrollment: 60Estimated
Primary Purpose: Treatment
Allocation: N/A
Interventional Model: Single Group Assignment
Masking: None (Open Label)

Contacts and Locations

Central Contacts: Shionogi Clinical Trials Administrator Clinical Support Help LineContact
Shionogiclintrials-admin@shionogi.co.jp

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