Study Status: Completed
Recruit Status: Completed
Study Type: Observational
Official Title: Observational Study of Acute Intermittent Porphyria Patients
Brief Summary:
This is an observational prospective study that will allow evaluating the clinical and laboratory parameters evolution of at least eight patients with AIP.
This study will allow establishing a baseline for the evaluation of the eight patients that are planned to be included in a gene therapy clinical trial (AAVPBGD-AIP-001) for the AIP treatment using a rAAV5-AAT-cohPBGD expression.
Patients fulfilling the study inclusion criteria will undergo a clinical and laboratory evaluation for a minimum of 6 months (with one inclusion visit, one final visit and at least two visits of follow up) up to a maximum of 24 months until their inclusion in the subsequent clinical trial.
A complete evaluation of the clinical (symptoms and quality of life assessment) and laboratory (blood and urine) data will be collected.