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Gene Therapy Trial Report

Summary

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)


NCTID NCT07282847 (View at clinicaltrials.gov)
Description
Development Status 🔄 Active
Indication Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Disease Ontology Term 🔄 DOID:2752
Compound Name 🔄 AB-1009
Sponsor AskBio Inc
Funder Type Industry
Recruitment Status
Enrollment Count 12 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant 🔄 GAA
Therapy Type 🔄 Gene transfer
Therapy Route 🔄 In-vivo
Mechanism of Action 🔄 Functional gene replacement
Route of Administration 🔄 Intravenous
Drug Product Type 🔄 Viral vector
Delivery System 🔄 Viral transduction
Vector Type 🔄 AAV
Dose 1 🔄 1E13 vg/kg
Dose 2 🔄 1.5E13 vg/kg

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2025-11-26
Completion Date 2032-09
Last Update 🔄 2026-03-31

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 10
Locations United States

Regulatory Information


Has US IND True
FDA Designations 🔄 Fast Track, Orphan Drug Designation
Recent Updates FDA accepted IND in January 2026

Resources/Links