Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 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.
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.
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 |
Interested in This Trial?
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, categorized as Phase 1. The registered enrollment target is 4 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 232-participant average among 14 other Pompe Disease trials with a reported enrollment target (98% lower). The listed sponsor is Spark Therapeutics, which has 13 total studies on file at ClinicalTrials.gov.
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 reports 20 study locations spanning 13 distinct geographic areas - top geographies include 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
Similar trials for Pompe Disease
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.
-
AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease
ACTIVE NOT RECRUITING · Phase 1
-
Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
COMPLETED · Phase 1
-
Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
COMPLETED · Phase 1
-
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
RECRUITING · Phase 2
-
Cognitive and Neurological Pathologies in Pompe Disease
RECRUITING
-
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
RECRUITING
Explore more on PlainTrial
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.