Actively 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)
Led by AskBio Inc · Updated on 2026-05-06
12
Participants Needed
10
Research Sites
333 weeks
Total Duration
On this page
Sponsors
A
AskBio Inc
Lead Sponsor
B
Bayer
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be 18 years or older at the time of consent.
- Confirmed GAA enzyme deficiency or biallelic GAA gene mutations.
- Have received enzyme replacement therapy (alglucosidase alfa or avalglucosidase alfa-ngpt) for at least 6 months (minimum 10 infusions).
- Forced vital capacity (FVC) in upright position between 30% and 80% of predicted.
- Able to walk at least 100 meters in 6-minute walk test; use of cane, quad cane, or walker allowed.
- Use of contraceptive or barrier methods as required by the protocol.
- Able to give informed consent and follow study procedures.
You will not qualify if you...
- Severe cardiomyopathy with left ventricular ejection fraction below 40% or NYHA functional class 3 or higher.
- Require invasive mechanical ventilation or daytime noninvasive ventilation.
- Intolerance or serious infusion-associated reactions to enzyme replacement therapy.
- Known intrinsic liver diseases including hepatitis, HIV-related liver disease, portal hypertension, splenomegaly, hepatic encephalopathy, severe fatty liver, cirrhosis, liver fibrosis stage 2 or higher, liver tumors, or abnormal liver laboratory tests.
- Any medical condition or abnormality that may affect safety or compliance as judged by the investigator.
- Prior gene therapy treatment.
- Use of systemic immunosuppressants (except inhaled or topical) other than glucocorticoids within 30 days before screening.
- Use of investigational drugs or drugs affecting this study within 30 days before screening or during the study.
- Received any vaccine within 30 days before dosing.
- Other conditions deemed exclusionary by the investigator.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 10 locations
1
Barrow Neurological Institute
Phoenix, Arizona, United States, 85013
Not Yet Recruiting
2
University of California, Irvine (UCI)
Irvine, California, United States, 92697
Actively Recruiting
3
Stanford Neuroscience Health Center
Palo Alto, California, United States, 94304
Not Yet Recruiting
4
NYU Langone
New York, New York, United States, 10016
Not Yet Recruiting
5
Duke University
Durham, North Carolina, United States, 27705
Not Yet Recruiting
6
Oregon Health and Science University (OHSU)
Portland, Oregon, United States, 97239
Not Yet Recruiting
7
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
8
University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, United States, 15213
Not Yet Recruiting
9
University of Texas Southwest Medical Center
Dallas, Texas, United States, 75235
Not Yet Recruiting
10
Virginia Commonwealth University (VCU)
Richmond, Virginia, United States, 23298
Not Yet Recruiting
Research Team
A
AskFirst Patient Engagement
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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