Current Research Needs – Observational Studies, Clinical Trials, and surveys

 

Acute Porphyrias (AIP, VP, HCP, ADP)

Panhematin Prevention Study

Are you currently using Panhematin® to prevent acute porphyria attacks? Dr. Karl Anderson, esteemed Porphyria Expert at The University of Texas Medical Branch in Galveston, Texas is currently seeking patients for a Panhematin® Prevention study. The purpose of this study is to determine if Panhematin® is effective for prevention of acute attacks of porphyria.  We are seeking 20-30 patients who currently receive prophylactic preventative heme treatment. You may be receiving heme weekly, bi-weekly or once a month and have successfully prevented attacks. Participation in this study will involve traveling to the study site for participation. Travel to the study site will be included.

 

RESEARCH STUDY:

STUDY DESCRIPTION:

WHO CAN PARTICIPATE:

PARTICIPATING SITES:

Panhematin Prevention Study

The purpose of this study is to determine if Panhematin® is effective for prevention of acute attacks of porphyria.

Patients diagnosed with AIP, VP or HCP

  1. UTMB – Galveston, Texas

Contact Edrin Williams, Director of Patient Services to learn more about this study and be placed in contact with the research coordinator. (edrinw@porphyriafoundation.org ; 301-347-7166)

Acute Porphyria Survey – Must have 1-2 Annual Attacks

Have you experienced at least one porphyria attack in the past 2 years, but no more than 2 attacks-per-year in the past 2 years?

We are looking for individuals with acute porphyria who do not currently receive prophylactic treatment to participate in a 90-minute in-person or telephone interview. Eligible study participants will be compensated. Contact the APF today for more information at info@porphyriafoundation.org or 301-347-7166.

 


 

Erythropoietic Protoporphyria (EPP)

Mitsubishi Tanabe MT-7117 Clinical Trial

Phase 3 Clinical Trial
Participants with EPP or XLP are needed!
FIRST SITES ACTIVE NOW – Boston, MA and Columbus, OH

Trial Description: A Phase 3, Global, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, And Tolerability of MT-7117 in Subjects with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

Treatment: Oral medication (pills), once daily in the morning with or without food Duration: 26 weeks plus optional 26 week double-blinded extension.

Endpoint: Increased pain free light exposure in adults and adolescents with a history of phototoxic reactions from EPP or XLP.

Study Sites: There will be 12 study sites in the US, followed by global study sites across multiple countries. COLUMBUS, OHIO and BOSTON, MA NOW OPEN!

Age Range: Patients age 12 - 75

Travel: Will be included and will be arranged by a concierge service trained on EPP/XLP.

Please contact the American Porphyria Foundation for more information and we will connect you with a study site nearest you. Email info@porphyriafoundation.org OR call 866-APF-3635 (301-347-7166).

SUNSENSE Short-Duration study for Erythropoietic Protoporphyria (EPP)

Announcement: Recruiting volunteers for EPP research study

Dr. Amy Dickey, Massachusetts General Hospital, is currently recruiting adult EPP patients for a study investigating methods for measuring light exposure and symptoms in EPP. Her study may eventually help patients to predict symptoms using a wearable smartphone-based device. This could help patients manage the disease better and with less anxiety. These accurate measurements are important in order for researchers and the food and drug administration (FDA) to know whether or not a new therapy is effective. Consequently, the information gained from this study may help new therapies be approved more easily.

The time commitment includes answering a very brief symptom and light exposure survey every day for two months. For one month, you will also be asked to wear a small light sensor. To learn more, Email info@porphyriafoundation.org OR call 866-APF-3635 (301-347-7166).

 


 

Porphyria Cutanea Tarda (PCT)

Harvoni Study for PCT

This research aims to:

  • Assess whether Harvoni alone is an effective and durable treatment for PCT patients with both Hepatitis C and PCT.
  • Determine whether treating patients with PCT and Hepatitis C with Harvoni is an effective as treating PCT with the standard therapies, phlebotomy or hyrdrochloroquine.

 

RESEARCH STUDY:

STUDY DESCRIPTION:

WHO CAN PARTICIPATE:

PARTICIPATING SITES:

HARVONI STUDY (PCT)

This is a clinical trial giving PCT patients with hepatitis C a standard dose of Harvoni to see if it also resolves the PCT symptoms.

Adult PCT patients who also have Hepatitis C.

  1. UTMB – Galveston, TX
  2. Wake Forest, NC

Contact Edrin Williams, Director of Patient Services to learn more about this study and be placed in contact with the research coordinator. (edrinw@porphyriafoundation.org ; 301-347-7166)