In addition to Ongoing Research, here are current studies that need patient support!
Participant recruitment is now underway for a Phase 2 clinical trial on Bitopertin, an investigational treatment for Erythropoietic Protoporphyria.
This is 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).
Please click here to submit a confidential interest form.
It will be used to contact potential participants and manage referrals. Interested patients can also email email@example.com or contact UPA on 800-868-1292.
Patients with Erythropoietic Protoporphyria (EPP) or X-Linked Porphyria (XLP) are needed for an FDA-sponsored research study on cimetidine. The study will assess whether cimetidine (a medication normally used to treat gastrointestinal issues such as ulcers or acid reflux) can reduce protoporphyrin IX (PPIX) levels which may impact photosensitivity.
Participating Study Sites:
Dr. Amy Dickey, Massachusetts General Hospital
Dr. Karl Anderson, University of Texas Medical Branch
Dr. Herbert Bonkovsky, Atrium Wake Forest Baptist Health
Of interest, a recent study in Denmark indicated the potential for cimetidine to lower protoporphyrin IX (PPIX) in patients with erythropoietic protoporphyria. Click here to read about it.
To learn more about study details, contact UPA on 1-800-868-1292 or firstname.lastname@example.org to learn more.
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 email@example.com or call 800-868-1292.
* 2 slots remaining
Stay tuned for several studies related to:
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