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NCT04093349 · ClinicalTrials.gov registry record · Phase 1
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
A Phase 1 study of Pompe Disease and Pompe Disease (Late-onset), sponsored by Spark Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 4
- Enrollment target
- 20
- Study locations
NCT04093349: Active Phase 1 study of Pompe Disease and Pompe Disease (Late-onset), sponsored by Spark Therapeutics.
NCT04093349 is a Phase 1 study of Pompe Disease and Pompe Disease (Late-onset) that is active but no longer recruiting, run by Spark Therapeutics. The registered enrollment target is 4 participants, below the 232-participant average among 14 other Pompe Disease trials with a reported enrollment target (98% lower). The trial reports 20 study locations across 13 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04093349, a Phase 1 study of Pompe Disease and Pompe Disease (Late-onset), is active but no longer recruiting, sponsored by Spark Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 4 participants
- Enrollment target
- 20
- Study locations
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.
Primary Outcome
Adverse events.
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 |
What the registry record for NCT04093349 still lists
NCT04093349 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 4 participants, a relatively small participant target, below the 232-participant average among 14 other Pompe Disease trials with a reported enrollment target (98% lower).
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.
NCT04093349 names 20 study sites across 13 states, led by Other, Pennsylvania, Arizona.
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.
What interventions are being tested in trial NCT04093349?
The interventions under investigation include: SPK-3006 (GENETIC).
Who is sponsoring clinical trial NCT04093349?
This trial is sponsored by Spark Therapeutics, which has 13 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04093349's enrollment target sits among peer trials
4 14th of 14 higher than 1 of 14 other Pompe Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Pompe Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
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