A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency

In brief

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.

Key facts

Study ID

NCT04093349

Run by

Spark Therapeutics, Inc.

People needed

4

Starts

2020-10-01

Expected to finish

2032-04-01

Last updated by the study team

2024-11-27

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• 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

Full record on ClinicalTrials.gov

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