Comparing the Efficacy and Safety of Ixekizumab to Placebo in Patients > 6 years of age with EB simplex generalized severe

Phase 1

• Conditions: Epidermolysis bullosa simplex; MedDRA version: 20.0Level: PTClassification code 10014989Term: Epidermolysis bullosaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

• Registration Number: EUCTR2020-001542-19-FR
• Lead Sponsor: CHU NICE

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-04-03
• Last Update Posted: 10 August 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 0

Inclusion Criteria

- Male or female patients 6 years or older
- Laboratory confirmed diagnosis of EBS-gen sev due to KRT5 or 14 mutation
- DLQI or cDLQI = 10
- Subject/caregiver agrees not to use any topical therapies other than the investigator approved

Are the trial subjects under 18? yes
Number of subjects for this age range: 25
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion Criteria

- EBS lesions requiring oral therapy to treat an infection
- Use of any diacerein containing product within 6 months prior to Visit 1
- Use of systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Visit
- Use of systemic steroidal therapy within 30 days prior to Visit 1
- Use of any systemic product that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study assessments within 30 days prior to Visit 1

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: to assess whether ixekizumab every 2 weeks (Q2W) is superior to placebo at Week 12 in the treatment of patients > 6 years of age with EBS-gen sev in improving their QoL;Secondary Objective: -To assess the safety of ixekizumab treatment in EBS-gen sev treated patients<br>-To compare the efficacy and health outcomes measures evolution between ixekizumab Q2W and placebo at Week 12 in term of severity, itch, pain and duration of dressing<br>-To compare the efficacy and health outcomes measures evolution between ixekizumab Q2W and placebo between S12 and S20<br>;Primary end point(s): proportion of patients achieving = 75% of diminution of the dermatologic QoL (DLQI and cDLQI) at week 12 compared to baseline;Timepoint(s) of evaluation of this end point: AT 12 WEEKS

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Safety and tolerability will be assessed through the description of specific events occurring during the study<br>- Secondary efficacy and health outcomes measures<br>- Severity of EB will be assessed using the validated score iscorEB, the static - Physician Global Assessment (sPGA) and the patient global assessment (PaGA)<br>- Itch will be assessed using a visual analogic scale (VAS).<br>- Pain will be assessed using a VAS<br>- The duration of dressing will be evaluated by patients/parents on the week before visit<br>- The QoL and the efficacy and health outcomes measures during the follow-up post-treatment period (S12-S20) will be defined as for the treatment period<br>;Timepoint(s) of evaluation of this end point: AT 12 WEEKS