Participate in Research

The UPA supports research which is focused on improving diagnosis, developing therapies, understanding of the disease burden, and advocates for increased research funding that can benefit patients — with an ultimate goal of helping researchers find a cure for all porphyrias.

Currently Recruiting- Clinical Trials

Clinical trials are studies testing new or experimental potential treatments for the porphyrias. These studies are important because they playing a key role in the development and FDA (Food and Drug Administration) approval of new therapies. Other studies may be available at clinicaltrials.gov.

Currently there are no clinical trials recruiting. Join our mailing list to learn about upcoming opportunities.

Currently Recruiting- Observational Studies

Observational studies look to understand more about the complications and issues people with the porphyrias experience. These studies are important because they contribute to the overall knowledge and progress of the understanding of the porphyrias.

Featured

EPP, XLP, Caregivers

Survey: Understanding EPP/XLP Pain

EPP, XLP, Caregivers

Researchers at Stanford University are conducting a research study to better understand the pain experienced by people with EPP and its impact on quality of life and daily behaviors. Participation involves completing a ~10 minute online survey.

EPP, XLP, Caregivers

EPP, XLP

stEPP Study for EPP & XLP

EPP, XLP

Sponsored by Portal Therapeutics. This study tracks the disease and its impact on individuals living with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP).

EPP, XLP

AIP, HCP, VP, ADP, EPP, XLP, PCT, CEP, HEP, Caregivers

Support research as a control participant

AIP, HCP, VP, ADP, EPP, XLP, PCT, CEP, HEP, Caregivers

You do not need to have porphyria to participate in porphyria research. Learn how you can get involved!

AIP, HCP, VP, ADP, EPP, XLP, PCT, CEP, HEP, Caregivers

AIP, HCP, VP, ADP

ELEVATE, a Registry of Patients With AHP

AIP, HCP, VP, ADP

This global patient registry is being conducted to characterize the natural history and real-world clinical management of patients with AHP, and to further characterize the real-world safety and effectiveness of givosiran and other approved AHP therapies.

AIP, HCP, VP, ADP

AIP, HCP, VP, ADP, EPP, XLP, HEP, PCT, CEP

Longitudinal Study of the Porphyrias

AIP, HCP, VP, ADP, EPP, XLP, HEP, PCT, CEP

Long-term study to learn more about the disease course and quality of life for all the porphyrias. Open to US residents with a confirmed diagnosis of porphyria.

AIP, HCP, VP, ADP, EPP, XLP, HEP, PCT, CEP

AIP, ADP, VP, HCP

Online AHP Drug Database

AIP, ADP, VP, HCP

This study collects information on acute attacks that may have been caused by a medication.

AIP, ADP, VP, HCP

EPP, XLP

EPP/XLP Gene Modifier Study

EPP, XLP

Study to better understand why some patients with EPP and XLP experience more severe symptoms than others.

EPP, XLP

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Past Research Opportunities

Research Opportunities

EPP, XLP

EPP/XLP Parents Questionnaire

EPP, XLP

Help develop a handbook for EPP parents by sharing your experiences in this brief survey.

EPP, XLP

EPP/XLP Questionnaire

EPP, XLP

The goal of this questionnaire is to better understand how EPP/XLP impacts the lives of individuals with this condition.

EPP, XLP

EPP/XLP Access to Care Interviews

EPP, XLP

The goal of this research is to better understand the drivers and barriers to accessing care and treatment in EPP/XLP.

EPP, XLP

AIP, ADP, HCP, VP

Mental Health Focus Group- Acute Porphyrias

AIP, ADP, HCP, VP

This focus group with focus on mental health and mental health resources for the acute hepatic porphyrias.

AIP, ADP, HCP, VP

EPP, XLP, PCT, CEP, HEP

Mental Health Focus Group- Cutaneous Porphryias

EPP, XLP, PCT, CEP, HEP

This focus group with focus on mental health and mental health resources for cutaneous porphyrias.

EPP, XLP, PCT, CEP, HEP

EPP, XLP

Cimetidine Research Study in Patients with EPP and XLP

EPP, XLP

An FDA-sponsored study to assess whether cimetidine a medication normally used to treat gastrointestinal issues such as ulcers or acid reflux) reduces protoporphyrin IX (PPIX) levels which may impact photosensitivity.

EPP, XLP

Phase 3 INSPIRE Study for EPP & XLP – Filled!

EPP, XLP

MT-7117 is a potential treatment for EPP and XLP. Recruiting patients ages 12+. International and US sites.

EPP, XLP

Participate in Research

Currently Recruiting- Clinical Trials

Currently Recruiting- Observational Studies

Past Research Opportunities

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