RAAV1-CMV-GAA (study agent) Administration ( DrugBank: - )
1 disease
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 256 | Muscle glycogenosis | 1 |
256. Muscle glycogenosis
Clinical trials : 180 / Drugs : 133 - (DrugBank : 29) / Drug target genes : 25 - Drug target pathways : 105
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT00976352 (ClinicalTrials.gov) | September 2010 | 13/7/2009 | Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease | Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients With Pompe Disease | Pompe Disease | Drug: rAAV1-CMV-GAA (study agent) Administration;Other: RMST | University of Florida | National Heart, Lung, and Blood Institute (NHLBI) | Completed | 2 Years | 18 Years | All | 9 | Phase 1/Phase 2 | United States |