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 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)
Recruiting · NCT07282847 · Registry updated 2026-07-20Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| 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) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, disorder of glycogen metabolism, glycogen storage disease II, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorder, neuromuscular disease | Recruiting | Phase 1 / Phase 2 | 2026-07-20 | NCT07282847 |
2 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| AskBio Inc | 8 |
| Bayer | 178 |
9 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Barrow Neurological Institute | Phoenix | United States | 50 |
| Duke University | Durham | United States | 91 |
| Hospital of the University of Pennsylvania | Philadelphia | United States | 142 |
| NYU Langone | New York | United States | 34 |
| Stanford Neuroscience Health Center | Palo Alto | United States | 15 |
| University of California, Irvine (UCI) | Irvine | United States | 1 |
| University of Pittsburgh Medical Center (UPMC) | Pittsburgh | United States | 10 |
| University of Texas Southwest Medical Center | Dallas | United States | 1 |
| Virginia Commonwealth University (VCU) | Richmond | United States | 4 |