research

Latest News

In addition to Ongoing Research, here are current studies that need patient support!

Erythropoietic Protoporphyria (EPP)

Disc Medicine has announced a Phase 2 clinical trial for participants with Erythropoietic Protoporphyria (EPP).

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).

  • Ten study sites are anticipated
  • 62 participants (age 18+)
  • Potential enrollment for adolescents ages 12-17
  • 24-week trial
  • Oral medication (Bitopertin)
  • Inclusion/Exclusion criteria will apply
  • Must reside in the US

For detailed information visit www.clinicaltrials.gov or click here. 

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 kristen@porphyria.org. UPA will connect you with a study site when recruitment begins. 

 

Acute Hepatic Porphyria

Exit Interview for Givosiran/Givlaari

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…

  • Participated and completed an Alnylam Pharmaceuticals clinical trial for givosiran (the Phase 1/Phase 2 Open Label Extension or “ENVISION” trial), and
  • Continued to take, and currently take givosiran (GIVLAARI®)

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

 

Medication Study to Inform Drug Safety

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 info@porphyria.org or call 800-868-1292.

 

Cannabis and Complementary and Alternative Medicine

Patients with Acute Intermittent Porphyria (AIP) anecdotally report using Complementary and Alternative Medicines (CAMs) and cannabis to alleviate their symptoms, but this has never been formally assessed. Click the link below to participate in a de-identified survey so we can learn more!

Anyone with AIP who is 18 years or older can participate. If you have never used CAMs or cannabis, you can participate as well.

Click Here

 

EPP/XLP

Erythropoietic Protoporphyria Interview Research Study

* 2 slots remaining

  • We are looking for ~ 20 adults and adolescents with Erythropoietic Protoporphyria (EPP) or X-linked protoporphyria (XLP) in the US and Canada who are at least 12 years of age and speak English.
  • If you qualify for the study, you will be asked to participate in one Zoom interview that will last no more than one hour.
  • During the interview, you will be asked questions about symptoms and impacts you may have experienced as a result of having EPP or XLP. You will also be asked to complete and provide feedback on several questionnaires during the interview.
  • The interview will be conducted by a member of the project team from Health Outcomes Solutions, a consulting group specializing in this type of research.
  • Each interview will be scheduled at a time that is convenient for you, including during the day, during the evening, or on weekends.
  • After you complete the interview, you will receive a US$150 Amazon gift card for your time.

 

Cannabis and Complementary and Alternative Medicine

Patients with Erythropoietic Protoporphyria (EPP) anecdotally report using Complementary and Alternative Medicines (CAMs) and cannabis to alleviate their symptoms, but this has never been formally assessed. Click the links below to participate in a de-identified survey so we can learn more!

Anyone with EPP who is 18 years or older can participate. If you have never used CAMs or cannabis, you can still participate!

Click Here

 

Upcoming

Stay tuned for several studies related to:

  • CEP
  • EPP pediatric/caregiver interviews
  • EPP clinical trials, and
  • AHP Patient-Reported Outcomes

DON’T MISS A THING! SIGN UP

The excitement in porphyria is building as research projects grow – don’t miss an opportunity to participate!

 

Sign UP at Sign Up - United Porphyrias Association.