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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: Recruiting Phase 1 study of Pompe Disease (Late-onset) and LOPD, sponsored by AskBio.
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. According to ClinicalTrials.gov, the official US trial registry.
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 |
What NCT07282847 shows while recruiting
NCT07282847 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 46-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (74% lower).
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 lists 10 locations in 8 states (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.
Learn More About Clinical Trials
Similar trials for Pompe Disease (Late-onset)
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 NCT07282847's enrollment target sits among peer trials
12 7th of 8 higher than 2 of 8 other Pompe Disease (Late-onset) trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Pompe Disease (Late-onset) 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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