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Observational Study of Acute Intermittent Porphyria Patients

NCT02076763 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 9

Last updated 2014-03-12

No results posted yet for this study

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.

Conditions

  • Acute Intermittent Porphyria

Sponsors & Collaborators

  • Porphyria Centre Sweden

    collaborator UNKNOWN

  • University of Navarra

    collaborator OTHER

  • UniQure N.V.

    collaborator INDUSTRY

  • Digna Biotech S.L.

    lead INDUSTRY

Principal Investigators

  • Juan Ruiz, MD · Digna Biotech S.L.

  • Jesus Prieto, MD · Clinica Universidad de Navarra

  • Rafael Enriquez de Salamanca, MD · Hospital 12 Octubre

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-08-31
Primary Completion
2013-07-31
Completion
2014-02-28

Countries

  • Spain

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02076763 on ClinicalTrials.gov