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Observational Study of Acute Intermittent Porphyria Patients

Digna Biotech S.L. · 2011
ClinicalTrials.gov · Datasets · License: Public Domain · 2011
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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.
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