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)

NCT07282847 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: ASK-POM9-CS101
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 9

Brief 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

LOPD; Pompe Disease Late-Onset

Keywords

Pompe Disease; Glycogen Storage Disease; Lysosomal Storage Diseases; Acid Maltase Deficiency; Acid Maltase Deficiency Disease; Gene Therapy; AB-1009; Neuromuscular Disease; LOPD; GAA gene; Adeno-Associated Virus (AAV); Late-Onset Pompe Disease; Acid Alpha-Glucosidase (GAA)

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) during the primary observation period

  Day 1 (Dosing) through Week 52 (the end of the primary observation period)

Secondary Outcomes (13)

• Area under the curve (AUC) of GAA activity in serum

  Baseline through Week 52

• Viral shedding (whole blood, saliva, urine)

  Day 1 through Week 24 (or until 3 consecutive data points are at or below the limit of detection)

• AUC of urinary Glc4

  Baseline during the primary observation period (through Week 52)

• Change from baseline in GAA activity in muscle biopsy tissue at Week 52

  Baseline and Week 52

• Change from baseline in muscle biopsy glycogen content at Week 52

  Baseline and Week 52

Study Arms (2)

Cohort 1

EXPERIMENTAL

1.0E13 vg/kg

Genetic: AB-1009 (GAA Gene)

Cohort 2

EXPERIMENTAL

1.5E13 vg/kg

Genetic: AB-1009 (GAA Gene)

Interventions

AB-1009 (GAA Gene) GENETIC

A single intravenous infusion of AB-1009

Cohort 1; Cohort 2

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant must be ≥18 to ≤65 years of age at the time of signing the informed consent form.
• Confirmed GAA enzyme deficiency from any tissue source and/or confirmed biallelic GAA gene mutations.
• Undergone enzyme replacement treatment (ERT) (either alglucosidase alfa (Lumizyme®), avalglucosidase alfa-ngpt (Nexviazyme®)), or cipaglucosidase alfa (Pombiliti®) 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;
• FVC in the upright position ≥30% and ≤80% of predicted;
• Capable of walking at least 100 meters in the 6MWT (use of a cane, quad cane, or standard walker is permitted);
• Male or female. Contraceptive/barrier use by men and women requirements as per protocol.
• Capable of giving informed consent.
• Able to understand and comply with all study procedures.

You may not qualify if:

• Severe cardiomyopathy, defined as left ventricular ejection fraction (LVEF) \<40% or New York Heart Association (NYHA) functional class 3 or above;
• Require invasive mechanical ventilation, or rely on noninvasive ventilation during the day;
• Intolerance to ERT or investigator-assessed intolerance to ERT, prior experience of serious ERT-related infusion-associated reactions (IARs);
• 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;
• Prior or ongoing medical condition(s), including any active infection, malignancy within 5 years of screening (except basal or squamous cell skin cancer), 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.
• Have received gene therapy prior to screening;
• Have received any systemic immunosuppressants (except inhalation or topical use) other than glucocorticoids 30 days prior to screening through completion of screening through completion of screening, and/or known intolerance to immunosuppressants such as glucocorticoids; other concomitant immunosuppression would require sponsor approval.
• 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);
• Have received any vaccine within 30 days prior to dosing;
• Other conditions that make the participant not eligible for the study according to the investigator.
• Contraindication to MRI, hypersensitivity to contrast dyes, shellfish, or iodine, or implanted spinal rods, cardiac pacemaker, or other implantation that would distort cMRI images.

Sponsors & Collaborators

• Bayer: collaborator
• AskBio Inc: lead

Study Sites (9)

Barrow Neurological Institute

Phoenix, Arizona, 85013, United States

NOT YET RECRUITING

University of California, Irvine (UCI)

Irvine, California, 92697, United States

RECRUITING

Stanford Neuroscience Health Center

Palo Alto, California, 94304, United States

NOT YET RECRUITING

NYU Langone

New York, New York, 10016, United States

NOT YET RECRUITING

Duke University

Durham, North Carolina, 27705, United States

RECRUITING

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

University of Pittsburgh Medical Center (UPMC)

Pittsburgh, Pennsylvania, 15213, United States

NOT YET RECRUITING

University of Texas Southwest Medical Center

Dallas, Texas, 75235, United States

RECRUITING

Virginia Commonwealth University (VCU)

Richmond, Virginia, 23298, United States

NOT YET RECRUITING

MeSH Terms

Conditions

Glycogen Storage Disease Type II; Glycogen Storage Disease; Lysosomal Storage Diseases; Neuromuscular Diseases

Interventions

alpha-Glucosidases

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Carbohydrate Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Glucosidases; Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Central Study Contacts

AskFirst Patient Engagement

CONTACT

919-561-6210; AskFirst@AskBio.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 26, 2025
• First Posted: December 15, 2025
• Study Start: April 15, 2026
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): September 1, 2032
• Last Updated: July 20, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (9)