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.
You may not be if
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 at screening.
Malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.
Current or chronic history of liver disease.
Known biallelic loss of function mutations whether in glycogenin gene (GYG) or in glycogen phosphorylase muscle associated gene(PYGM) .
Has received any investigational therapy or pharmacological treatment for Pompe disease, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before day 1 or is anticipated to do so during the study.
Has received gene therapy or small interfering ribonucleic acid (RNA) therapy for Pompe disease.
Participant, if female, is pregnant or breastfeeding at screening.
Participant, whether male or female, is planning to conceive a child during the study.
Clareo Health | Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
