RecruitingPhase 2NCT06509984

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

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


Sponsor

Centre Hospitalier Universitaire de Nice

Enrollment

20 participants

Start Date

Aug 6, 2024

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 6 YearsMax Age: 99 Years

Inclusion Criteria4

  • 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 Criteria5

  • \- 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

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Interventions

DRUGApremilast treatment

treatment of apremilast


Locations(4)

CHU de Nice

Nice, France, France

APHP Necker-Enfants-Malades

Paris, France, France

APHP Hôpital Saint-Louis

Paris, France, France

chu de Toulouse

Toulouse, France

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NCT06509984


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