A pilot multicentric open study assessing the efficacy and safety of NEMOlizumab in patients ≥12yo with Dystrophic Epidermolysis bullosa; NEMODE

Condition studied

dystrophic epidermolysis bullosa

Investigational medicinal product(s)

Every trial of Nemolizumab across our registers →

Eligibility

Primary endpoint

Evolution of the Peak Pruritus Numerical Rating Scale (PP-NRS) score (scores range from 0 [no itch] to 10 [worst itch imaginable]) between week 0 and week 4 of treatment..

Endpoint detail

Score of a five point Likert scale ranging from 1 to 5 (1 – Very dissatisfied. 2 – dissatisfied. 3 – unsure. 4 – satisfied. 5 – Very satisfied) to assess global satisfaction of patientsat Week 24., Evolution of the Pain Numeric Rating Scale (PNRS) score between W0 and W4 and W24 will be studied., Evolution of the DLQI (Dermatology Life Quality Index) score between weeks 0, 4 and 24 of treatment., Evolution of fibrosis assessed by measuring maximal mouth opening, joint range of motion for the right elbow and right knee, finger extension and quantifying dysphagia symptoms through the Dysphagia Numerical Rating Scale at weeks 0, 4 and 24 of treatment., Evolution of skin aspect assessed based on a picture of the most involved leg taken at baseline and 6 months of treatment., Occurrence of predefined adverse events (type 1 hypersensitivity reactions, including mild urticaria and mild facial angioedema, headache, asthma, eczematous reactions), their severity grade and potential relationship to the treatment to assess the safety of Nemolizumab., Evolution of the EB-specific severity score (PP-NRS - Peak Pruritus Numerical Rating Scale) between baseline (W0) and Week 24 (W24) of treatment.