254. Porphyria Clinical trials / Disease details
Clinical trials : 72 / Drugs : 53 - (DrugBank : 16) / Drug target genes : 19 - Drug target pathways : 35
| 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 | NCT02082860 (ClinicalTrials.gov) | November 2012 | 28/2/2014 | Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria | Phase I, Multicentre, Open Label, Single Dose, Dose-ranging Clinical Trial to Investigate the Safety and Tolerability of a Gene Therapy rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria | Acute Intermittent Porphyria | Genetic: rAAV2/5-PBGD vector dosage 1;Genetic: rAAV2/5-PBGD vector dosage 2;Genetic: rAAV2/5-PBGD vector dosage 3;Genetic: rAAV2/5-PBGD vector dosage 4 | Digna Biotech S.L. | Porphyria Centre Sweden;Centro de Investigacion Medica Aplicada de Navarra;UniQure N. V.;Nationales Centrum für Tumorerkrankungen | Completed | 18 Years | 64 Years | Both | 8 | Phase 1 | Spain |