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NCT04174105 · ClinicalTrials.gov registry record · Phase 1
Gene Transfer Study in Patients With Late Onset Pompe Disease
A Phase 1 study of Pompe Disease (Late-onset), sponsored by Astellas Gene Therapies.
- Active
- Registry status
- Phase 1
- Development phase
- 11
- Enrollment target
- 4
- Study locations
NCT04174105 is a Phase 1 study of Pompe Disease (Late-onset) that is active but no longer recruiting, run by Astellas Gene Therapies. The registered enrollment target is 11 participants, below the 46-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (76% lower). The trial reports 4 study locations across 3 states.
The verdict
NCT04174105, a Phase 1 study of Pompe Disease (Late-onset), is active but no longer recruiting, sponsored by Astellas Gene Therapies.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 11 participants
- Enrollment target
- 4
- Study locations
Study Summary
This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).
Conditions Studied
Interventions
- GENETIC zocaglusagene nuzaparvovec
Study Locations (4)
California
- University of California Irvine, Department of Neurology - Orange
- Stanford University - Palo Alto
Utah
- University of Utah, Division of Medical Genetics - Salt Lake City
Other
- Newcastle Upon Tyne Hospitals Foundation Trust Clinical Research Facility - Newcastle upon Tyne
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 11 participants |
| Start Date | 2020-10-27 |
| Est. Completion | 2035-02-28 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04174105
The ClinicalTrials.gov registry entry for NCT04174105 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 11 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 46-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (76% lower). The listed sponsor is Astellas Gene Therapies, which has 7 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Pompe Disease (Late-onset) appearing as the primary indexed condition, and to 1 intervention - of which zocaglusagene nuzaparvovec is the first listed.
NCT04174105 reports 4 study locations spanning 3 distinct geographic areas - top geographies include California, Utah, Other.
Frequently Asked Questions
What is clinical trial NCT04174105 about?
NCT04174105 is a clinical study titled "Gene Transfer Study in Patients With Late Onset Pompe Disease". This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).
What is the current status of trial NCT04174105?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 11 participants. The study started on 2020-10-27. Estimated completion is 2035-02-28.
What conditions does trial NCT04174105 study?
This clinical trial studies the following conditions: Pompe Disease (Late-onset).
What interventions are being tested in trial NCT04174105?
The interventions under investigation include: zocaglusagene nuzaparvovec (GENETIC).
Who is sponsoring clinical trial NCT04174105?
This trial is sponsored by Astellas Gene Therapies, which has 7 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04174105 being conducted?
This trial has 4 study locations across California, Utah. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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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.