Assessing Apremilast for Epidermolysis Bullosa Simplex in Children

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial evaluates the efficacy and safety of Apremilast in patients over 6 years old diagnosed with Epidermolysis Bullosa Simplex Generalized. Participants will undergo a structured treatment regimen over 20 weeks, which includes an initial treatment phase, a dechallenge period where treatment is paused, and a rechallenge phase. Throughout the study, patients will have regular visits to monitor compliance, adverse events, and overall health through clinical examinations and questionnaires.

Why it matters

Who can join: the full rules

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

Where it's running

• CHU de Nice
  Nice, France, France Recruiting
• APHP Necker-Enfants-Malades
  Paris, France, France Not yet recruiting
• APHP Hôpital Saint-Louis
  Paris, France, France Not yet recruiting
• chu de Toulouse
  Toulouse, France Not yet recruiting

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.