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Variegate Porphyria clinical trials

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NCT ID: NCT05854784 Completed - Variegate Porphyria Clinical Trials

Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)

Start date: March 28, 2023
Phase: Phase 2
Study type: Interventional

The primary objective is to evaluate the impact of afamelanotide on the severity of skin disease in patients with Variegate Porphyria (VP). The secondary objectives are to evaluate the safety and tolerability of afamelanotide in patients with VP and evaluate the impact of afamelanotide on the quality of life of patients with VP.

NCT ID: NCT02935400 Active, not recruiting - Clinical trials for Acute Intermittent Porphyria

Acute Porphyria Biomarkers for Disease Activity

Start date: April 28, 2014
Phase:
Study type: Observational

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.

NCT ID: NCT02922413 Terminated - Clinical trials for Acute Intermittent Porphyria

Panhematin for Prevention of Acute Attacks of Porphyria

Start date: October 30, 2015
Phase: Phase 2
Study type: Interventional

The purpose of this study is to determine if Panhematin is safe and effective for prevention of acute attacks of porphyria. The study aims to provide high quality evidence for the use on hemin for prevention of acute attacks of porphyria. High quality studies have not been done previously for treating or preventing acute attacks with hemin. The lack of strong evidence for efficacy of hemin for treatment and prevention of attacks limits its availability for patients with acute porphyrias. Funding source: FDA Office of Orphan Product Development (FDA OOPD) FD-R-03720