research

Ongoing Research

If you are interested in learning more or participating in any of these studies contact UPA at info@porphyria.org or 800-868-1292.

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. 

Study Short Name Summary Who can participate? Sponsor (with link)
Cimetidine for EPP/XLP This trial will look at cimetidine as a possible treatment for EPP/XLP People 15 years or older with EPP/XLP who experiences symptoms within 30 minutes of sun exposure The Porphyrias Consortium
Panhematin for the Prevention of Acute Attacks To determine if Panhematin is safe and effective for prevention of acute attacks of porphyria People with AHP who have frequent attacks The University of Texas Medical Branch, Galveston

 

 

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.

Study Short Name Summary Who can participate? Sponsor 
Longitudinal Study of the Porphyrias A study looking to learn more about the disease course and quality of life for all the porphyrias Anyone (adults and children) with a confirmed diagnosis of a porphyria The Porphyrias Consortium
ELEVATE A registry of patients with AHPs People with a diagnosis of AHP Alnylam Pharmaceuticals
AHP Drug Database To collect information on acute attacks that may have been caused by a medication. Anyone with a confirmed diagnosis of an AHP The Porphyrias Consortium

Other studies may be available at clinicaltrials.gov

 

No Longer Enrolling

Therapeutic Studies

Study Short Name Summary Sponsor (with link)
Harvoni for PCT Giving PCT patients with hepatitis C a standard dose of Harvoni to see if it also resolves the PCT symptoms The Porphyrias Consortium
Panhematin for Acute Attacks Comparing Panhematin to glucose to determine the effectiveness of Panhematin as treatments for acute attacks University of Texas Medical Branch, Galveston
MT-7117 for EPP A placebo-controlled trial of MT-7117 at treatment for EPP/XLP Mitsubishi Tanabe Pharma

Observational Studies

Study Short Name Summary Sponsor (with link)
Clinical Diagnosis of Acute Porphyrias This study will look at the number of abnormal lab tests and AHP-like symptoms in family members of patients with AHPs University of California at San Francisco
AHP Biomarkers To identify new biomarkers of disease activity in people with AHPs University of Texas Medical Branch, Galveston