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This study examines antibody levels and biomarkers in the blood of people with late-onset Pompe disease. No treatment is given; patients with a documented diagnosis take part. The study is being conducted as a non-phase observational study; it is not currently recruiting but is ongoing (active, not recruiting).
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
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Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Participant has a documented clinical diagnosis of LOPD. * Participant is enzyme replacement therapy ERT-naïve (ERT-N) or if the participant is currently taking an approved ERT treatment or is participating in an ERT-interventional study, the ERT must have been received for at least 6 months or more (ERT-experienced \[ERT-E\]). * Participant is willing and able to comply with study visits and procedures. * Participant agrees to not start participating in any other clinical study involving an investigational study treatment, including ERT, while participating in this study. Exclusion Criteria: * Participant previously received an AAV-related product (any serotype). * Participant is currently participating in a Pompe-related interventional study (other than ERT-interventional studies) or has received gene or cell therapy. * Participant requires any invasive or noninvasive ventilation support while awake and upright (non-invasive support while sleeping with either continuous positive airway pressure (CPAP) or bilevel positive airway pressure (BiPAP) is acceptable for eligibility). * Participant is unable to ambulate (assistive devices \[e.g., cane or walker\] are acceptable for eligibility). * Participants who have received any ERT for less than 6 months as of the Baseline visit are not eligible.