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This study evaluates how often neutralizing antibodies against the carrier (capsid) of the experimental gene therapy SPK-3006 are present in people with late-onset Pompe disease. It included patients aged 18 and older who had been on regular enzyme replacement therapy (ERT) for at least 18 months. It was conducted as a study with no assigned phase and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Provide written informed consent and authorization to use protected health information in accordance with national and local privacy regulations * Male or females ≥18 years of age * Currently on ERT using regular recombinant human GAA infusions for at least 18 months prior to screening * Documented history of clinically moderate late-onset Pompe disease. Exclusion Criteria: * History of HIV infection * Requires any invasive ventilation (other than BiPAP at night) or noninvasive ventilation while awake and upright * Previously received SPK-3006 * Previously dosed with any investigational or approved gene therapy product at any time or treated with an investigational drug within the last 12 weeks (vaccination studies are accepted) * Any concurrent clinically significant condition that would not allow the potential participant to complete the Day 1 examinations, or other condition that, in the opinion of the Investigator and/or Sponsor, makes the subject unsuitable for participation in the study * Unable or unwilling to comply with the schedule of visits and/or study assessments described in the clinical protocol.