New 2020! Research for Canadians with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP): Phase 3 clinical trial of Mitsubishi Tanabe’s investigational therapy MT-7117 (dersimelagon)
Research Goal: The study’s primary goal is to evaluate whether the therapy extends the daily sunlight exposure time before participants develop the first early symptoms — burning, tingling, or stinging — associated with sun exposure in the periods between one hour post-sunrise and one hour pre-sunset. Additional goals include assessing patients’ global impression of change and the total number of sunlight-induced pain events during the treatment period.
About the therapy: The study will evaluate (NCT04402489) MT-7117 is a man-made (synthetic) small molecule that binds to and activates the melanocortin-1 receptor (MC1R) protein. Given orally, the experimental therapy is intended to increase the production of a pigment called melanin to induce photoprotection (protection against sunlight) of the skin.
Participants: Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical history, aged 12 years to 75 years. Detailed Eligibility Criteria can be found here.
How will it work: In the double-blind Phase 3 study, participants with either EPP — the most common porphyria of childhood — or XLP will be assigned randomly to a low or high-dose of MT-7117, or a placebo, given once daily in the morning with or without food for 26 weeks. Enrolled patients may join an optional 26-week extension phase.
Researcher in Canada: Dr. Bruce Ritchie, University of Alberta, Edmonton, AB
Who to Contact for more information:
Porphyria Research in Canada - Articles
Child and family experiences with inborn errors of metabolism: a qualitative interview study with representatives of patient groups
Authors: Sara D. Khangura, Kylie Tingley, Pranesh Chakraborty, Doug Coyle, Jonathan B. Kronick, Anne-Marie Laberge, Julian Little, Fiona A Miller, John J. Mitchell, Chitra Prasad, Shabnaz Siddiq, Komudi Siriwardena, Rebecca Sparkes, Kathy N. Speechley, Sylvia Stockler, Yannis Trakadis, Brenda J. Wilson, Kumanan Wilson, Beth K. Potter on behalf of the Canadian Inherited Metabolic Diseases Research Network (CIMDRN)
This article is published with open access at Springerlink.com
Patient-centered health care for children with inborn errors of metabolism (IEM) and their families is important and requires an understanding of patient experiences, needs, and priorities. IEM-specific patient groups have emerged as important voices within these rare disease communities and are uniquely positioned to contribute to this understanding. We conducted qualitative interviews with IEM patient group representatives to increase understanding of patient and family experiences, needs, and priorities and inform patient-centered research and care.