Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
Completed · Not applicable
Conditions studied: Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
In brief
The purpose of this study is to obtain information pertaining to the occurrence of antibodies to investigational SPK-3006 capsid and GAA, GAA activity and GAA antigen levels in the usual care setting of Late-Onset Pompe Disease (LOPD) participants on an enzyme replacement regimen. Additionally, a careful evaluation of laboratory and functional testing in patients with LOPD may provide information to better understand the disease features and better drive the design of a future interventional investigational gene therapy trial. An understanding of the underlying status of liver and muscle health in individuals with LOPD may also inform best surveillance during the conduct of gene therapy trials.
Key facts
- Study ID
- NCT03893240
- Run by
- Spark Therapeutics, Inc.
- People needed
- 61
- Starts
- 2019-06-12
- Expected to finish
- 2020-10-27
- Last updated by the study team
- 2023-03-24
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify 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 qualify 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.
Where it is running
- Barrow Neurological Institute — Phoenix, Arizona, United States
- University of California Irvine Health — Orange, California, United States
- University of Kansas Medical Center — Kansas City, Kansas, United States
- University of Minnesota Medical School — Minneapolis, Minnesota, United States
- Oregon Health & Science University — Portland, Oregon, United States
- University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States
- Hôpital Raymond-Poincaré — Garches, Hauts-de-Seine, France
- Assistance Publique Hôpitaux de Marseille — Marseille, France
- CHU Nice — Nice, France
- Klinikum der Universität München — München, Germany
- Università degli Studi di Messina — Messina, Italy
- Universita degli Studi di Milano - Clinica Oculistica I — Milan, Italy
- Università degli Studi di Napoli Federico II — Naples, Italy
- Fondazione Mondino Istituto Neurologico Nazionale a Carattere Scientifico — Pavia, Italy
- Azienda Ospedaliero Universitaria Pisana — Pisa, Italy
- Azienda Ospedaliera Universitaria Citta della Salute e della Scienza di Torino — Torino, Italy
- Erasmus University Medical Center — Rotterdam, Netherlands
- Salford Royal NHS Foundation Trust — Salford, United Kingdom
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.