Clinical Trial: Acute Porphyria Biomarkers

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Observational

Official Title: Acute Porphyrias: Biomarkers for Disease Activity and Response to Treatment

Brief Summary: The long term objective of the research is to identify new biomarkers of disease activity in the human acute porphyrias. This pilot study is intended to provide pilot and feasibility data needed to plan larger and more definitive future studies.

Detailed Summary: This translational pilot research is based on preliminary results using animal models. The investigators will collect samples of blood, urine and feces from up to 50 participants with well documented acute porphyrias, at 2 expert sites that are members of the Porphyrias Consortium. Collection and analysis of these samples will be used to assess feasibility of performing such studies in humans with acute porphyrias, recognizing that these disorders are more heterogeneous than reproduced in animal models, and affect individuals who cannot all be studied simultaneously and in large groups. Therefore, we will assess the feasibility of methods for collecting, processing, storing and shipping samples at multiple study sites for later biomarker analysis. Larger and more definitive studies of biomarkers will be designed and implemented based on data and experience from this pilot-feasibility study.
Sponsor: The University of Texas Medical Branch, Galveston

Current Primary Outcome: Potential biomarkers Biomarkers [ Time Frame: 10 days ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: The University of Texas Medical Branch, Galveston

Dates:
Date Received: October 13, 2016
Date Started: January 2014
Date Completion: December 2018
Last Updated: October 17, 2016
Last Verified: October 2016