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

February 21, 2025 updated by: Centre Hospitalier Universitaire de Nice

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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

Study Type

Interventional

Enrollment (Estimated)

20

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Nice, France, 06000
      • Paris, France, 75015
        • Not yet recruiting
        • APHP Necker-Enfants-Malades
        • Contact:
          • Christine BODEMER
      • Paris, France, 75018
        • Not yet recruiting
        • APHP Hôpital Saint-Louis
        • Contact:
          • Emmanuelle BOURRAT
      • Toulouse, France, 31059
        • Not yet recruiting
        • CHU de Toulouse
        • Contact:
          • Juliette MAZEREEUW -HAUTIER

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: apremilast treatment
treatment by apremilast in two phases of challenge
Drug: Apremilast treatment
treatment of apremilast

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To describe efficacy of apremilast
Time Frame: weeks 20
number of new blister conting every day
weeks 20

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety of apremilast treatment
Time Frame: weeks 20
number of SAE
weeks 20

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Centre Hospitalier Universitaire de Nice

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 6, 2024

Primary Completion (Estimated)

October 1, 2025

Study Completion (Estimated)

April 1, 2026

Study Registration Dates

First Submitted

July 15, 2024

First Submitted That Met QC Criteria

July 18, 2024

First Posted (Actual)

July 19, 2024

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

February 21, 2025

Last Verified

February 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 23-PP-10
  • 2023-508794-83-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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