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

Last updated: February 21, 2025
Sponsor: Centre Hospitalier Universitaire de Nice
Overall Status: Active - Recruiting

Phase

2

Condition

Epidermolysis Bullosa

Pemphigus Vulgaris (Pv)

Treatment

Apremilast treatment

Clinical Study ID

NCT06509984
23-PP-10
2023-508794-83-00
  • Ages 6-99
  • All Genders

Study Summary

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

Inclusion

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 theinvestigator approved

Exclusion

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 toVisit 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 thesubject at undue risk by study participation or interferes with the studyassessments within 30 days prior to Visit 1

Study Design

Total Participants: 20
Treatment Group(s): 1
Primary Treatment: Apremilast treatment
Phase: 2
Study Start date:
August 06, 2024
Estimated Completion Date:
April 01, 2026

Study 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

Connect with a study center

  • CHU de Nice

    Nice, 06000
    France

    Active - Recruiting

  • APHP Hôpital Saint-Louis

    Paris, 75018
    France

    Site Not Available

  • APHP Necker-Enfants-Malades

    Paris, 75015
    France

    Site Not Available

  • chu de Toulouse

    Toulouse, 31059
    France

    Site Not Available

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