In addition to Ongoing Research, here are current studies that need patient support!
Dr. A. Dickey at Massachusetts General Hospital, Dr. K. Anderson at the University of Texas Medical Branch, and Dr. H. Bonkovsky at Atrium Wake Forest Baptist Health are currently recruiting patients with EPP who are 15 years of age or older for a study investigating whether taking oral cimetidine pills can affect your EPP or XLP symptoms. The study will test whether Cimetidine (a medication normally used to treat gastrointestinal issues such as ulcers or acid reflux) can reduce the level of protoporphyrin in your blood, and if it can affect your symptoms.
The entire study will last 9 months. For 4 months of the study, participants will be asked to wear a wearable light sensor and respond to brief surveys send by text message. In some cases, the entire study may be able to be completed with telemedicine visits.
Please contact the United Porphyrias Association (UPA) on 1-800-868-1292 or email firstname.lastname@example.org for more information.
Note: Participants can be on Scenesse (afamelanotide) for this study if symptoms still occur within approximately 30 minutes of bright light exposure.
A Randomized, Double-blind, Placebo-Controlled Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and Protoporphyrin IX (PPIX) Concentrations in Participants With Erythropoietic Protoporphyria (EPP).
Disc Medicine is a Massachusetts-based pharmaceutical company focused on Hematology.
To read the press release, click here.
To learn more, contact United Porphyrias Association at 800-868-1292 or email email@example.com. UPA will connect you with a study site when recruitment begins.
The United Porphyrias Association (UPA) is collaborating with Alnylam Pharmaceuticals and RTI Health Solutions (RTI-HS) to identify individuals with acute hepatic porphyria (AHP) who are interested in participating in an interview study conducted by RTI-HS (on behalf of Alnylam Pharmaceuticals).
You qualify for this one-time 60-minute telephone interview study if you…
The objective of these interviews is to capture, in the patients’ own words, the symptoms and impacts of AHP and their experiences over the time they have received givosiran.
Contact Kristen Wheeden by email or telephone: Kristen@porphyria.org or 800-868-1292
The NIH-Supported Porphyrias Consortium has launched an online observational study to collect information on acute porphyria attacks that may have been caused by a medication. The “Evidence-Based Assessment of Medication Sensitivity in Acute Hepatic Porphyria” requests patient and caregiver reports on suspected medication/drug-induced acute porphyria attacks, as well as safe use of drugs previously labeled "unsafe" or with unknown risk.
The goal of this project is to create an updated list of medications that are risky or safe for people with one of the acute porphyrias.
After consenting, participants 18+ will be provided a unique web link to complete the initial survey. Participants will be encouraged to update their surveys when they have new information to report.
To participate, please contact United Porphyrias Association on firstname.lastname@example.org or call 800-868-1292.
* 2 slots remaining
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