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A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Sponsor: Spark Therapeutics, Inc.
Summary
The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.
Official title: Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
4
Start Date
2020-10-01
Completion Date
2032-04
Last Updated
2024-11-27
Healthy Volunteers
No
Conditions
Interventions
SPK-3006
adeno-associated viral (AAV) vector
Locations (29)
Barrow Neurological Institute
Phoenix, Arizona, United States
University of California Irvine Health
Orange, California, United States
Emory University School of Medicine
Atlanta, Georgia, United States
University of Kansas Medical Center Research Institute
Kansas City, Kansas, United States
University of Minnesota
Minneapolis, Minnesota, United States
Oregon Health & Science University
Portland, Oregon, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
University of Utah
Salt Lake City, Utah, United States
Lysosomal and Rare Disorders Research & Treatment Center
Fairfax, Virginia, United States
Vancouver General Hospital
Vancouver, British Columbia, Canada
The Ottawa Hospital
Ottawa, Ontario, Canada
Montreal Neurological Hospital
Montreal, Quebec, Canada
Rigshospitalet
Copenhagen, Denmark
Centre Hospitalier Universitaire d'Angers
Angers, France
CHU Paris IdF Ouest - Hôpital Raymond Poincaré
Garches, France
Centre Hospitalier Régional Universitaire de Lille
Lille, France
Assistance Publique Hôpitaux de Marseille
Marseille, France
Nice University Hospital
Nice, France
Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München
München, Germany
Universita Degli Studi Di Messina - Dipartimento di Medicina Clinica e Sperimentale
Messina, Italy
Universita Degli Studi Di Milano, Laboratorio di Biochimica e Genetica della Malattie Neuromuscolari
Milan, Italy
Malattie Metaboliche Universita Degli Studi Di Napoli Federico II
Naples, Italy
UO Neurologia Azienda Ospedaliera Universitaria Pisana
Pisa, Italy
Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino - Neurology, Osp. Molinette
Torino, Italy
Erasmus Medical Center
Rotterdam, Netherlands
New Queen Elizabeth Hospital Birmingham
Birmingham, GBR, United Kingdom
The Royal Free London NHS Foundation Trust
London, GBR, United Kingdom
Salford Royal MHS Foundation Trust
Salford, United Kingdom