A 20-Week Study Assessing the Efficacy of Apremilast in Patients with EB Simplex Generalized
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age6-99
SponsorCentre Hospitalier Universitaire de Nice
About this trial
The goal of this clinical trial is to Assessing the Efficacy and Safety of Apremilast in Patients \> 6 years of age with EB simplex generalized . The main question it aims to answer are: describe efficacity of this treatment.
Participants will take treatments and have to use bullets during the study period.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male or female patients 6 years or older
Laboratory confirmed diagnosis of EBS-sev due to KRT5 or 14 mutation (autosomal)
Mean daily number of new blisters >3.
Subject/caregiver agrees not to use any topical therapies other than the investigator approved
Disqualifiers
- 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 1
Use of systemic steroidal therapy within 30 days prior to Visit 1
Trial design
Design model
Single group
Treatments tested in this trial
Apremilast treatment
Drugtreatment of apremilast
Treatment groups
20 Participants
are divided into 1 treatment groupGroup A: apremilast treatmentExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
To describe efficacy of apremilast
number of new blister conting every day
Time frame
weeks 20Secondary outcomes
1
Safety of apremilast treatment
number of SAE
Time frame
weeks 20Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov