Trials / Active Not Recruiting
Active Not RecruitingNCT02935400
Acute Porphyria Biomarkers for Disease Activity
Acute Porphyrias: Biomarkers for Disease Activity and Response to Treatment
- Status
- Active Not Recruiting
- Phase
- —
- Study type
- Observational
- Enrollment
- 50 (estimated)
- Sponsor
- The University of Texas Medical Branch, Galveston · Academic / Other
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Hemin | Hemin will be administered under a separate protocol or for clinical treatment, and samples will be collected under this protocol before and after treatment. |
Timeline
- Start date
- 2014-04-28
- Primary completion
- 2026-12-01
- Completion
- 2026-12-31
- First posted
- 2016-10-17
- Last updated
- 2025-04-18
Locations
1 site across 1 country: United States
Source: ClinicalTrials.gov record NCT02935400. Inclusion in this directory is not an endorsement.