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)

ClinicalTrials.gov ID: NCT07282847

Public ClinicalTrials.gov record NCT07282847. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

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ClinicalTrials.gov public records Last synced May 5, 2026, 2:34 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

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)

Study identification

NCT ID: NCT07282847
Recruitment status: Recruiting
Study type: Interventional
Phase: Phase 1, Phase 2
Lead sponsor: AskBio Inc; Industry
Enrollment: 12 participants

Conditions and interventions

Conditions

Interventions

AB-1009 (GAA Gene) Genetic

Genetic

Eligibility (public fields only)

Age range: 18 Years and older
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Feb 9, 2026
Primary completion: Aug 31, 2028
Completion: Aug 31, 2032
Last update posted: May 3, 2026

2026 – 2032

United States locations

U.S. sites: 10
U.S. states: 8
U.S. cities: 10

Facility	City	State	ZIP	Site status
Barrow Neurological Institute	Phoenix	Arizona	85013	Not yet recruiting
University of California, Irvine (UCI)	Irvine	California	92697	Recruiting
Stanford Neuroscience Health Center	Palo Alto	California	94304	Not yet recruiting
NYU Langone	New York	New York	10016	Not yet recruiting
Duke University	Durham	North Carolina	27705	Not yet recruiting
Oregon Health and Science University (OHSU)	Portland	Oregon	97239	Not yet recruiting
Hospital of the University of Pennsylvania	Philadelphia	Pennsylvania	19104	Recruiting
University of Pittsburgh Medical Center (UPMC)	Pittsburgh	Pennsylvania	15213	Not yet recruiting
University of Texas Southwest Medical Center	Dallas	Texas	75235	Not yet recruiting
Virginia Commonwealth University (VCU)	Richmond	Virginia	23298	Not yet recruiting

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT07282847, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted May 3, 2026 · Synced May 5, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT07282847 live on ClinicalTrials.gov.

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