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NCT07282847 · ClinicalTrials.gov registry record · Phase 1
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)
A Phase 1 study of Pompe Disease (Late-onset) and LOPD, sponsored by AskBio.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 10
- Study locations
NCT07282847 is a Phase 1 study of Pompe Disease (Late-onset) and LOPD that is actively recruiting participants, run by AskBio. The registered enrollment target is 12 participants, below the 46-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (74% lower). The trial reports 10 study locations across 8 states.
The verdict
NCT07282847, a Phase 1 study of Pompe Disease (Late-onset) and LOPD, is actively recruiting participants, sponsored by AskBio.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 10
- Study locations
Study Summary
This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult participants with late-onset Pompe disease (LOPD).
Conditions Studied
Interventions
- GENETIC AB-1009 (GAA Gene)
Study Locations (10)
California
- University of California, Irvine (UCI) - Irvine
- Stanford Neuroscience Health Center - Palo Alto
Pennsylvania
- University of Pennsylvania - Philadelphia
- University of Pittsburgh Medical Center (UPMC) - Pittsburgh
Arizona
- Barrow Neurological Institute - Phoenix
New York
- NYU Langone - New York
North Carolina
- Duke University - Durham
Oregon
- Oregon Health and Science University (OHSU) - Portland
Texas
- University of Texas Southwest Medical Center - Dallas
Virginia
- Virginia Commonwealth University (VCU) - Richmond
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2026-02-10 |
| Est. Completion | 2032-09 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07282847
The ClinicalTrials.gov registry entry for NCT07282847 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 12 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 (74% lower). The listed sponsor is AskBio, which has 8 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Pompe Disease (Late-onset) appearing as the primary indexed condition, and to 1 intervention - of which AB-1009 (GAA Gene) is the first listed.
NCT07282847 reports 10 study locations spanning 8 distinct geographic areas - top geographies include California, Pennsylvania, Arizona.
Frequently Asked Questions
What is clinical trial NCT07282847 about?
NCT07282847 is a clinical study titled "A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)". This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult participants with late-onset Pompe disease (LOPD).
What is the current status of trial NCT07282847?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2026-02-10. Estimated completion is 2032-09.
What conditions does trial NCT07282847 study?
This clinical trial studies the following conditions: Pompe Disease (Late-onset), LOPD.
What interventions are being tested in trial NCT07282847?
The interventions under investigation include: AB-1009 (GAA Gene) (GENETIC).
Who is sponsoring clinical trial NCT07282847?
This trial is sponsored by AskBio, which has 8 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07282847 being conducted?
This trial has 10 study locations across Arizona, California, New York, North Carolina, Oregon. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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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.