Menu
Recruiting NCT06509984

A 20-Week Study Assessing the Efficacy of Apremilast in Patients with EB Simplex Generalized

Phase II Interventional Epidermolysis Bullosa Simplex Genodermatosis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Apremilast treatment.
Who it may be relevant to
Registry conditions: Epidermolysis Bullosa Simplex, Genodermatosis. Basic parameters: 6 years — 99 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
France
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

EBULO. a 20-Week Multicentre, Open Study Assessing the Efficacy and Safety of Apremilast in Patients > 6 Years of Age with EB Simplex Generalized

Overview

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.

Detailed description

The patient will have 7 visits. After Confirmation of inclusion criteria and no exclusion criteria, the study will be explain to the patient after signing the inform consent and be included in the study Treatment Period (Period 1); challenge period This period will be the first treatment period that will occur from 8 weeks. During this period the patient will see the doctor at the hospital, he will perform the study procedures The second period : dechallenge (period 2): All patients stop their treatment for 4 weeks.

The second treatment period - rechallenge (period 3) the patient will take the treatment for a second 8 weeks period At week 20 - End of study

At each visit:

The investigator at each visit compliance to the treatment and adverse events, Measure vital signs and perform clinical examination.

The patient will assess the different questionnaires, for the study

Interventions

  • Drug Apremilast treatment
    treatment of apremilast

Primary outcome measures

  • To describe efficacy of apremilast [Time frame: weeks 20]
Secondary outcome measures (1)
  • Safety of apremilast treatment [Time frame: weeks 20]

Eligibility criteria

Inclusion criteria

  • 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

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 1
  • 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

France · 4 centers
  • CHU de Nice — Nice
  • APHP Necker-Enfants-Malades — Paris
  • APHP Hôpital Saint-Louis — Paris
  • chu de Toulouse — Toulouse

Publications

  • Fontas E, Hubiche T, Maruani A, Moschoi R, Hussain S, Chiaverini C. Multicentre phase II open study assessing the efficacy and safety of apremilast in children and adults with epidermolysis bullosa simplex generalised: study protocol for the EBULO study. BMJ Open. 2025 Nov 12;15(11):e103194. doi: 10.1136/bmjopen-2025-103194. PMID 41224305

Identifiers

NCT: NCT06509984 · 23-PP-10 · 2023-508794-83-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗