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A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
NCT04093349 · View on ClinicalTrials.gov ↗
Study 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.
Conditions Studied
Interventions
- GENETIC SPK-3006
Study Locations (20)
Other
- 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
Pennsylvania
- University of Pennsylvania — Philadelphia
- University of Pittsburgh Medical Center — Pittsburgh
Arizona
- Barrow Neurological Institute — Phoenix
California
- University of California Irvine Health — Orange
Georgia
- Emory University School of Medicine — Atlanta
Kansas
- University of Kansas Medical Center Research Institute — Kansas City
Minnesota
- University of Minnesota — Minneapolis
Oregon
- Oregon Health & Science University — Portland
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2020-10-01 |
| Est. Completion | 2032-04 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04093349
The ClinicalTrials.gov registry entry for NCT04093349 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 4 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Spark Therapeutics, which has 6 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 7 conditions, with Pompe Disease appearing as the primary indexed condition, and to 1 intervention — of which SPK-3006 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04093349 reports 20 study locations spanning 13 distinct geographic areas — top geographies include Other, Pennsylvania, Arizona. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04093349 about?
NCT04093349 is a clinical study titled "A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)". 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.
What is the current status of trial NCT04093349?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2020-10-01. Estimated completion is 2032-04.
What conditions does trial NCT04093349 study?
This clinical trial studies the following conditions: Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type II, LOPD, Acid Maltase Deficiency. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04093349?
The interventions under investigation include: SPK-3006 (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04093349?
This trial is sponsored by Spark Therapeutics, which has 6 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04093349 being conducted?
This trial has 20 study locations across Arizona, California, Georgia, Kansas, Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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