Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02082860 | Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria | PHASE1 | COMPLETED | 8 | — | — | Nov 1, 2012 | Nov 1, 2014 | Dec 18, 2014 | 2 | Spain |
To assess the safety and determine the maximum therapeutic safe dose of the investigational gene therapy (GT) product rAAV2/5-PBGD for the treatment of AIP, registering and evaluating the occurrence of Adverse Events and/or Serious Adverse Events at the dose identified will be performed
| Arm | Type | Description |
|---|---|---|
| Cohort A | EXPERIMENTAL | rAAV2/5-PBGD vector dosage 1 |
| Cohort B | EXPERIMENTAL | rAAV2/5-PBGD vector dosage 2 |
| Cohort C | EXPERIMENTAL | rAAV2/5-PBGD vector dosage 3 |
| Cohort D | EXPERIMENTAL | rAAV2/5-PBGD vector dosage 4 |
| Name | Type | Description |
|---|---|---|
| rAAV2/5-PBGD vector dosage 1 | GENETIC | Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1. |
| rAAV2/5-PBGD vector dosage 2 | GENETIC | Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1. |
| rAAV2/5-PBGD vector dosage 3 | GENETIC | Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1. |
| rAAV2/5-PBGD vector dosage 4 | GENETIC | Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1. |
Inclusion Criteria: * Patient's written Informed Consent * Age between 18 and 64 years, inclusively. * Patients with confirmed diagnosis of Acute Intermittent Porphyria(AIP), as confirmed by clinical, biochemical data and genetic confirmation of porphobilinogen deaminase (PBGD) gene mutation. The p...