Pompe Disease Clinical Trial
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
Verified date | October 2023 |
Source | Spark Therapeutics |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Active, not recruiting |
Enrollment | 4 |
Est. completion date | April 2032 |
Est. primary completion date | April 2032 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - 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. Exclusion Criteria: - 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. |
Country | Name | City | State |
---|---|---|---|
Canada | Montreal Neurological Hospital | Montréal | Quebec |
Canada | The Ottawa Hospital | Ottawa | Ontario |
Canada | Vancouver General Hospital | Vancouver | British Columbia |
Denmark | Rigshospitalet | Copenhagen | |
France | Centre Hospitalier Universitaire d'Angers | Angers | |
France | CHU Paris IdF Ouest - Hôpital Raymond Poincaré | Garches | |
France | Centre Hospitalier Régional Universitaire de Lille | Lille Cedex | |
France | Assistance Publique Hôpitaux de Marseille | Marseille | |
France | Nice University Hospital | Nice | |
Germany | Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München | Munchen | |
Italy | 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 | UO Neurologia Azienda Ospedaliera Universitaria Pisana | Pisa | |
Italy | Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino - Neurology, Osp. Molinette | Torino | |
Netherlands | Erasmus Medical Center | Rotterdam | |
United Kingdom | 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 States | Emory University School of Medicine | Atlanta | Georgia |
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 | University of Minnesota | Minneapolis | Minnesota |
United States | University of California Irvine Health | Orange | California |
United States | University of Pennsylvania | Philadelphia | Pennsylvania |
United States | Barrow Neurological Institute | Phoenix | Arizona |
United States | University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania |
United States | Oregon Health & Science University | Portland | Oregon |
United States | University of Utah | Salt Lake City | Utah |
Lead Sponsor | Collaborator |
---|---|
Spark Therapeutics |
United States, Canada, Denmark, France, Germany, Italy, Netherlands, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values. | Adverse events. | Up to 5 years | |
Primary | Occurrence of immune response against AAV capsid | Up to 5 years | ||
Primary | Occurrence of immune response against GAA transgene | Up to 5 years |
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