Recruiting more participants to this clinical trial could provide the evidence needed to repurpose a current eczema treatment, dupilumab, to treat itch in all types of EB.
Prof Amy Paller works at Northwestern University, USA, on this project to provide evidence that dupilumab can treat EB symptoms. Dupilumab is currently used as an injection to treat adults and children with eczema. It has been shown to reduce itch in EB and can also reduce EB blistering and pain for some people who have tried it. This funding is to increase the number of people taking part in a clinical trial to provide the evidence needed to repurpose dupilumab as a treatment for all types of EB.
Prof Amy Paller, MD, is a paediatric dermatologist and clinical researcher. She is Chair of the Department of Dermatology and Director of the Skin Biology and Disease Resource-Based Center, Northwestern University, Chicago, USA.
This application covers costs for EB patients within the United States to travel to Chicago for participation in a clinical trial to investigate the effect of dupilumab on itch. The study involves: A) an 8-week observation period during which itch responses and sensor-generated objective scratch/sleep data are collected (mean is baseline); B) 16-week treatment with dupilumab during which itch is measured; and C) a long-term extension period for those who respond by at least 2 points in the peak itch score (minimal clinical response).
The primary endpoint is >50% of patients achieving reduction by >2 points in Worst Itch NRS (minimal meaningful reduction). We test if the reduced itch improves sleep and quality of life and if impact is correlated with eosinophil and IgE levels. Natural history of disease itch is captured during the observation period. At interim with 9 with EB completing the 16 weeks on dupilumab, most patients achieved itch reduction of >2 at 16 wks and 45% >4 points. EB activity score was reduced by 20% and in a few was dramatic. Improvement was seen across all subtypes of EB. Higher baseline IgE was associated with faster but not end reduction in itch. With these encouraging results, we hope to add 15 more EB patients and seek funds to invite patients outside of Chicago to participate. No travel budget was approved in the initial investigator-initiated study funded by Regeneron (providing drug and other study costs but not travel or mechanistic assessments).
Due in 2026
Prof Amy Paller