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

• Register: EUCTR
• Last refreshed on: 10 August 2020
• Main ID: EUCTR2020-001542-19-FR
• Date of registration: 03/04/2020
• Prospective Registration: Yes
• Primary sponsor: CHU NICE
• Public title: Comparing the Efficacy and Safety of Ixekizumab to Placebo in Patients > 6 years of age with EB simplex generalized severe
• Scientific title: A 12-Week Multicentre, Randomized, Blinded, Parallel-Group Study Comparing the Efficacy and Safety of Ixekizumab to Placebo in Patients > 6 years of age with EB simplex generalized severe - EB
• Date of first enrolment: 22/07/2020
• Target sample size: 0
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2020-001542-19
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

France

Contacts

• Name: Caillon
• Affiliation: CHU de Nice

• Name: Caillon
• Affiliation: CHU de Nice

Key inclusion & exclusion criteria

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



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Epidermolysis bullosa simplex
MedDRA version: 20.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Intervention(s)

Trade Name: Ixekizumab
Product Name: Taltz 80mg
Product Code: 3400930060797
Pharmaceutical Form: Injection
Pharmaceutical form of the placebo: Injection
Route of administration of the placebo: Subcutaneous use

Primary Outcome(s)

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

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
-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
-To compare the efficacy and health outcomes measures evolution between ixekizumab Q2W and placebo between S12 and S20

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: AT 12 WEEKS

Secondary end point(s): - Safety and tolerability will be assessed through the description of specific events occurring during the study
- Secondary efficacy and health outcomes measures
- Severity of EB will be assessed using the validated score iscorEB, the static - Physician Global Assessment (sPGA) and the patient global assessment (PaGA)
- Itch will be assessed using a visual analogic scale (VAS).
- Pain will be assessed using a VAS
- The duration of dressing will be evaluated by patients/parents on the week before visit
- 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

Secondary ID(s)

20-PP-04

Source(s) of Monetary Support

CHU de Nice

Ethics review

Status: Approved
Approval date: 22/07/2020
Contact: