Contact UPA at 800-868-1292 or info@porphyria.org
If you are interested in learning more or participating in any of these studies contact UPA at info@porphyria.org or 800-868-1292.
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 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 |
| CAMs and Cannabis for EPP & AIP | A survey looking at Complementary and Alternative Medicines (CAM) and Cannabis use in AIP & EPP to help determine whether these may improve symptoms | Anyone with EPP or AIP who is 18 years or older. If you have never used CAMs or cannabis you can still participate | The Porphyrias Consortium |
Other studies may be available at clinicaltrials.gov
| 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 |
| 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 |
