A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
- Conditions
- Glycogen Storage Disease Type IILysosomal Storage DiseasesGlycogen Storage Disease Type 2LOPDPompe DiseasePompe Disease (Late-onset)Acid Maltase Deficiency
- Interventions
- Genetic: SPK-3006
- Registration Number
- NCT04093349
- Lead Sponsor
- Spark Therapeutics, Inc.
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 4
- Provide written informed consent;
- Males and Females ≥18 years of age with late-onset Pompe disease;
- Received ERT for at least the previous 24 months
- Have clinically moderate, late-onset Pompe disease characteristics;
- Agree to use reliable contraception.
- Active hepatitis B and/or C;
- Significant underlying liver disease;
- Human immunodeficiency virus (HIV) infection;
- Prior hypersensitivity to rhGAA;
- Pre-existing anti-AAV neutralizing antibody titers;
- High titer antibody responses to rhGAA;
- Requires any invasive ventilation or requires noninvasive ventilation while awake and upright;
- Received any prior vector or gene transfer agent;
- Active malignancy (except non-melanoma skin cancer);
- History of liver cancer;
- Pregnant or nursing women;
- Any evidence of active infection at the time of SPK-3006 infusion.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description SPK-3006 SPK-3006 All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.
- Primary Outcome Measures
Name Time Method Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values. Up to 5 years Adverse events.
Occurrence of immune response against GAA transgene Up to 5 years Occurrence of immune response against AAV capsid Up to 5 years
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (29)
Centre Hospitalier Régional Universitaire de Lille
🇫🇷Lille Cedex, France
Salford Royal MHS Foundation Trust
🇬🇧Salford, United Kingdom
New Queen Elizabeth Hospital Birmingham
🇬🇧Birmingham, GBR, United Kingdom
The Royal Free London NHS Foundation Trust
🇬🇧London, GBR, United Kingdom
Centre Hospitalier Universitaire d'Angers
🇫🇷Angers, France
Vancouver General Hospital
🇨🇦Vancouver, British Columbia, Canada
Assistance Publique Hôpitaux de Marseille
🇫🇷Marseille, France
Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München
🇩🇪Munchen, Germany
University of California Irvine Health
🇺🇸Orange, California, United States
Emory University School of Medicine
🇺🇸Atlanta, Georgia, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
🇺🇸Pittsburgh, Pennsylvania, United States
Lysosomal and Rare Disorders Research & Treatment Center
🇺🇸Fairfax, Virginia, United States
University of Kansas Medical Center Research Institute
🇺🇸Kansas City, Kansas, United States
Rigshospitalet
🇩🇰Copenhagen, Denmark
Montreal Neurological Hospital
🇨🇦Montréal, Quebec, Canada
CHU Paris IdF Ouest - Hôpital Raymond Poincaré
🇫🇷Garches, France
Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino - Neurology, Osp. Molinette
🇮🇹Torino, Italy
UO Neurologia Azienda Ospedaliera Universitaria Pisana
🇮🇹Pisa, Italy
University of Utah
🇺🇸Salt Lake City, Utah, United States
The Ottawa Hospital
🇨🇦Ottawa, Ontario, Canada
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
🇮🇹Milano, Italy
Malattie Metaboliche Universita Degli Studi Di Napoli Federico II
🇮🇹Naples, Italy
Erasmus Medical Center
🇳🇱Rotterdam, Netherlands
Nice University Hospital
🇫🇷Nice, France
Barrow Neurological Institute
🇺🇸Phoenix, Arizona, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States