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Currently Recruiting
This trial is actively seeking participants in the UK. Discuss eligibility with your patient.
Status
Recruiting
Phase
PHASE2
NCT ID
NCT07123155

Trial Summary

The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.

Sponsor: Shionogi
Participants: ALL
Start: 2025-10-30
Completion: 2027-08-08
Min Age: 18 Years

Eligibility Criteria

Key Inclusion Criteria: * Participant must be ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the informed consent. * Participant must have a diagnosis of LOPD based on documentation of 1 of the following: 1. Deficiency of acid alpha-glucosidase (GAA) enzyme 2. GAA genotype * Participant has a %FVC ≥30% and ≤80% in an upright position without mechanical ventilation at screening; or Participant has ≥10% %FVC drop from upright position to supine position and %FVC ≥20% in a supine position. * Participant performs the 6MWT at screening, as determined by the clinical evaluator, and meets all of the following criteria: 1. Screening values of 6-minute walk distance (6MWD) are ≥75 meters 2. Screening values of 6MWD are ≤90% of the predicted value for healthy adults * Participants must be ERT-experienced, defined as currently receiving ERT and having been receiving ERT for ≥24 months, with no regimen change in the last 6 months. Key Exclusion Criteria: * Has a medical condition or any other extenuating circumstance that may pose an undue safety risk to the participant or may compromise his/her ability to comply with or adversely impact protocol requirements. * Has active infections…

Data from ClinicalTrials.gov. Always verify on the primary source before clinical use.

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