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Clinical trialNCT04093349Active, not recruitingPhase 1/PHASE2Interventional

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

SponsorSpark Therapeutics, Inc.Enrollment4Acid Maltase DeficiencyGlycogen Storage Disease Type 2Glycogen Storage Disease Type IILOPDLysosomal Storage DiseasesPompe Disease+1 more conditions
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

29 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

Canada
  • Vancouver General Hospital

    Vancouver, British Columbia, Canada

  • the Ottawa Hospital

    Ottawa, Ontario, Canada

  • Montreal Neurological Hospital

    Montreal, Quebec, Canada

Denmark
  • Rigshospitalet

    Copenhagen, Denmark

France
  • 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

Germany
  • Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München

    München, Germany

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

    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

Netherlands
  • Erasmus Medical Center

    Rotterdam, Netherlands

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 Kingdom

United States
  • 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

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

No drug or biological interventions are in our data for this study. It may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start
1 Oct 2020
Primary completion
30 Apr 2032
Completion
30 Apr 2032
Last update posted
27 Nov 2024
Registered enrollment
4
Registry id
NCT04093349

Cross-registrations

EU-CTR / CTIS (EudraCT)2019-001283-30

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.