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 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 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).

Study Overview

• Status: Recruiting
• Conditions: Pompe Disease (Late-onset); LOPD; Pompe Disease Late-Onset
• Intervention / Treatment: Genetic: AB-1009 (GAA Gene)

Detailed Description

This is an open-label study, up to 12 participants will receive a single IV infusion of AB-1009. Participants will be assigned to either cohort 1 (1.0E13 vg/kg) or Cohort 2 (1.5E13 vg/kg) based on enrollment in the study.

Study duration will include a screening period of up to 75 days, primary observation of 52 weeks, and a long-term follow-up period of 4 years.

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: AskFirst Patient Engagement; Phone Number: 919-561-6210; Email: AskFirst@AskBio.com

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85013
      • Not yet recruiting
      • Barrow Neurological Institute
      • Principal Investigator: Shafeeq Ladha, MD
  • California
    • Irvine, California, United States, 92697
      • Recruiting
      • University of California, Irvine (UCI)
      • Principal Investigator: Tahseen Mozaffar, MD
      • Contact: UCI Alpha Clinic; Phone Number: 949-824-3990; Email: alphaclinic@hs.uci.edu
    • Palo Alto, California, United States, 94304
      • Not yet recruiting
      • Stanford Neuroscience Health Center
      • Principal Investigator: John Day, MD, PhD
  • New York
    • New York, New York, United States, 10016
      • Not yet recruiting
      • NYU Langone
      • Principal Investigator: Nicolas Abreu, MD
  • North Carolina
    • Durham, North Carolina, United States, 27705
      • Not yet recruiting
      • Duke University
      • Principal Investigator: Natalie Katz, MD, PhD
      • Contact: Ming Xu, RN, MSN; Phone Number: 919-681-1945; Email: mingfen.xu@duke.edu
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Hospital of the University of Pennsylvania
      • Principal Investigator: Paul McIntosh, MD
      • Contact: Amanda Hicks; Phone Number: 267-496-3772; Email: Amanda.Hicks@pennmedicine.upenn.edu
      • Contact: Abigail Browngoehl; Email: Abigail.Browngoehl@Pennmedicine.upenn.edu
    • Pittsburgh, Pennsylvania, United States, 15213
      • Not yet recruiting
      • University of Pittsburgh Medical Center (UPMC)
      • Principal Investigator: Paula Clemens, MD
  • Texas
    • Dallas, Texas, United States, 75235
      • Recruiting
      • University of Texas Southwest Medical Center
      • Principal Investigator: Markey McNutt, MD, PhD
      • Contact: Juana Luevao; Phone Number: 214-645-7411; Email: geneticsresearch@utsouthwestern.edu
      • Contact: Markey McNutt, MD, PhD; Phone Number: 214-645-7411; Email: geneticsresearch@utsouthwestern.edu
  • Virginia
    • Richmond, Virginia, United States, 23298
      • Not yet recruiting
      • Virginia Commonwealth University (VCU)
      • Principal Investigator: Susan Sparks, MD, PhD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Participant must be ≥18 years of age at the time of signing the informed consent form.
2. Confirmed GAA enzyme deficiency from any tissue source and/or confirmed biallelic GAA gene mutations.
3. Undergone enzyme replacement treatment (ERT) (either alglucosidase alfa (Lumizyme®) or avalglucosidase alfa-ngpt (Nexviazyme®)), for at least 6 months (at least 10 infusions) before signing the initial informed consent form. During the screening process, participants need to remain on their current ERT until close to dosing;
4. FVC in the upright position ≥30% and ≤80% of predicted;
5. Capable of walking at least 100 meters in the 6MWT (use of a cane, quad cane, or standard walker is permitted);
6. Contraceptive/barrier use by men and women requirements as per protocol.
7. Capable of giving informed consent and able to understand and comply with all study procedures.

Exclusion Criteria:

1. Severe cardiomyopathy, defined as left ventricular ejection fraction (LVEF) <40% or New York Heart Association (NYHA) functional class 3 or above;
2. Require invasive mechanical ventilation, or rely on noninvasive ventilation during the day;
3. Intolerance to ERT or investigator-assessed intolerance to ERT, prior experience of serious ERT-related infusion-associated reactions (IARs);
4. Have known intrinsic liver diseases, including hepatitis, HIV-related liver disease, prior diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy, severe fatty liver, cirrhosis or liver fibrosis ≥stage 2, ultrasound-identified liver neoplasms, or laboratory tests suggesting elevated alpha-fetoprotein. Patients with liver function tests including ALT or AST >3× upper limit of normal (ULN) or any total bilirubin above ULN during screening will also be excluded;
5. Prior or ongoing medical condition(s), physical finding(s), assessment findings, or laboratory abnormality that, in the investigator's opinion, would impact participant's safety and compliance with the study procedures.
6. Have received gene therapy prior to screening;
7. Have received any systemic immunosuppressants (except inhalation or topical use) other than glucocorticoids or investigator-recommended immunosuppressants 30 days prior to screening through completion of screening, and/or known intolerance to immunosuppressants such as glucocorticoids;
8. Use of investigational drugs or drugs that could affect this study as evaluated by the investigator within 30 days prior to screening through completion of Week 52 or within 5 half-lives of the investigational drug (whichever is longer);
9. Have received any vaccine within 30 days prior to dosing;
10. Other conditions that make the participant not eligible for the study according to the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1; 1.0E13 vg/kg	Genetic: AB-1009 (GAA Gene); A single intravenous infusion of AB-1009
Experimental: Cohort 2; 1.5E13 vg/kg	Genetic: AB-1009 (GAA Gene); A single intravenous infusion of AB-1009

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of treatment-emergent adverse events (TEAEs)	From Day 1 (Dosing) to Week 52 (the end of the primary observation period)

Collaborators and Investigators

• Sponsor: AskBio Inc
• Collaborators: Bayer

Study record dates

Study Major Dates

• Study Start (Actual): April 15, 2026
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): September 1, 2032

Study Registration Dates

• First Submitted: November 26, 2025
• First Submitted That Met QC Criteria: December 5, 2025
• First Posted (Actual): December 15, 2025

Study Record Updates

• Last Update Posted (Actual): May 28, 2026
• Last Update Submitted That Met QC Criteria: May 27, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: Gene Therapy; Neuromuscular Disease; Pompe Disease; Acid Maltase Deficiency Disease; LOPD; Lysosomal Storage Diseases; Acid Maltase Deficiency; Glycogen Storage Disease; Adeno-Associated Virus (AAV); AB-1009; Acid-Alpha Glucoside (GAA); GAA gene; Late-Onset Pompe Disease
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Carbohydrate Metabolism, Inborn Errors; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Lysosomal Storage Diseases, Nervous System; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Glycogen Storage Disease Type II; Glycogen Storage Disease; Neuromuscular Diseases; Lysosomal Storage Diseases; Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Glucosidases; alpha-Glucosidases
• Other Study ID Numbers: ASK-POM9-CS101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No