Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Spark Therapeutics, Inc.
Age
18–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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.
You may not be if
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.
Clareo Health | Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
