🏛️ Verified NIH Record • Updated 2024-11-25
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) (NCT04093349)
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
🔵 Active, Not Recruiting Phase1 / Phase2 📋 Clinical Research Protocol Sponsor: Spark Therapeutics, Inc.
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Barrow Neurological Institute — Phoenix, Arizona
Age range: 18 Years
🧪 Interventions in This Study
- SPK-3006 (GENETIC)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (29 Sites)
Showing the first 25 of 29 sites.
- Barrow Neurological Institute — Phoenix, Arizona
- University of California Irvine Health — Orange, California
- Emory University School of Medicine — Atlanta, Georgia
- University of Kansas Medical Center Research Institute — Kansas City, Kansas
- University of Minnesota — Minneapolis, Minnesota
- Oregon Health & Science University — Portland, Oregon
- University of Pennsylvania — Philadelphia, Pennsylvania
- University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania
- University of Utah — Salt Lake City, Utah
- Lysosomal and Rare Disorders Research & Treatment Center — Fairfax, Virginia
- Vancouver General Hospital — Vancouver, British Columbia
- The Ottawa Hospital — Ottawa, Ontario
- Montreal Neurological Hospital — Montreal, Quebec
- Rigshospitalet — Copenhagen,
- Centre Hospitalier Universitaire d'Angers — Angers,
- CHU Paris IdF Ouest - Hôpital Raymond Poincaré — Garches,
- Centre Hospitalier Régional Universitaire de Lille — Lille,
- Assistance Publique Hôpitaux de Marseille — Marseille,
- Nice University Hospital — Nice,
- Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München — München,
- Universita Degli Studi Di Messina - Dipartimento di Medicina Clinica e Sperimentale — Messina,
- Universita Degli Studi Di Milano, Laboratorio di Biochimica e Genetica della Malattie Neuromuscolari — Milan,
- Malattie Metaboliche Universita Degli Studi Di Napoli Federico II — Naples,
- UO Neurologia Azienda Ospedaliera Universitaria Pisana — Pisa,
- Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino - Neurology, Osp. Molinette — Torino,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Pompe Disease
- Developing a Management Approach for Patients With "Late-Onset" Pompe Disease — Durham, North Carolina
- A Global Prospective Observational Registry of Patients With Pompe Disease — Little Rock, Arkansas
- Cognitive and Neurological Pathologies in Pompe Disease — Durham, North Carolina
- Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease — Durham, North Carolina
- Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) — Irvine, California
Explore by condition: All Pompe Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.