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Study identifier: NCT07282847 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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)

Condition: Pompe Disease (Late-onset) · Pompe Disease Late-Onset · LOPD  ·  Sponsor: AskBio Inc

PhasePhase 1/Phase 2
Planned participants12
Who can joinAll sexes, 18 Years to 65 Years
Healthy volunteersNo

About this study

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).

This description comes directly from the study's public registry record.

Talk to the study team

AskFirst Patient Engagement  ·  919-561-6210  ·  AskFirst@AskBio.com

Always discuss trial participation with your own doctor first.

Locations (9)

Barrow Neurological InstitutePhoenix, Arizona, United StatesNot Yet Recruiting
University of California, Irvine (UCI)Irvine, California, United StatesRecruiting
Stanford Neuroscience Health CenterPalo Alto, California, United StatesNot Yet Recruiting
NYU LangoneNew York, New York, United StatesNot Yet Recruiting
Duke UniversityDurham, North Carolina, United StatesRecruiting
Hospital of the University of PennsylvaniaPhiladelphia, Pennsylvania, United StatesRecruiting
University of Pittsburgh Medical Center (UPMC)Pittsburgh, Pennsylvania, United StatesNot Yet Recruiting
University of Texas Southwest Medical CenterDallas, Texas, United StatesRecruiting
Virginia Commonwealth University (VCU)Richmond, Virginia, United StatesNot Yet Recruiting

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Source record: clinicaltrials.gov/study/NCT07282847