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Clinical trial

Acute Porphyria Biomarkers for Disease Activity

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Last updated:30th Mar 2023
Identifier: NCT02935400
Acute Porphyria Biomarkers for Disease Activity ID: NCT02935400

Sponsor: The University of Texas Medical Branch, Galveston
Information provided by: The University of Texas Medical Branch, Galveston (Responsible Party)
Last Update Posted: 2023-03-31

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 Description:
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.

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

Intervention / Treatment:
- Drug: Hemin

Category Value
Study Start (Actual) 2014-04-28
Primary Completion (Estimated)
Study Completion (Estimated) 2023-12-31 
Enrollment (Estimated) 50
Study Type Observational
Other Study ID Numbers


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