What is Apremilast studying for Epidermolysis Bullosa Simplex generalized?

Apremilast is being studied to understand its effectiveness and safety in treating patients aged 6 years and older with Epidermolysis Bullosa Simplex generalized. The trial aims to describe how well this medication reduces symptoms like new blisters. The research is sponsored by the Centre Hospitalier Universitaire de Nice.

Who may be eligible for this Epidermolysis Bullosa Simplex generalized clinical trial?

Patients 6 years or older with a confirmed diagnosis of EBS due to KRT5 or KRT14 mutation may be eligible. Participants must have an average of more than 3 new blisters daily and agree not to use unapproved topical treatments. This trial accepts all genders. Please review eligibility criteria for more details.

Is there any cost to participate in this Epidermolysis Bullosa Simplex generalized clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel expenses to and from study visits may vary and are not guaranteed to be covered.

Actively Recruiting

Phase 2

Age: 6Years - 99Years

All Genders

ID06509984

A 20-Week Study to Evaluate the Safety and Effectiveness of Apremilast in Patients Aged 6 Years and Older with Epidermolysis Bullosa Simplex Generalized

Led by Centre Hospitalier Universitaire de Nice · Updated on 2025-02-24

Participants Needed

Research Sites

26 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the efficacy and safety of Apremilast in patients aged 6 years and older with Epidermolysis Bullosa Simplex generalized, a genetic skin condition. This phase 2 open-label study aims to describe how well Apremilast works in reducing symptoms, particularly the occurrence of new blisters, over a 20-week period. Participants will undergo three distinct periods an initial 8-week treatment phase called challenge, followed by a 4-week period without treatment called dechallenge, and a second 8-week treatment phase called rechallenge. During these periods, patients will take Apremilast and have regular visits to the hospital where doctors will perform study procedures and monitor progress. Throughout the study, participants will have seven visits where doctors will check vital signs, perform clinical examinations, review treatment adherence, and monitor for any side effects. Patients will also complete various questionnaires about their condition. The main measure of success is the efficacy of Apremilast at 20 weeks, along with safety assessments. The total study duration for each participant is 20 weeks.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 6Years - 99Years

All Genders

Eligibility Criteria

You may qualify if you...

Male or female patients 6 years or older
Laboratory confirmed diagnosis of EBS-sev due to KRT5 or KRT14 mutation (autosomal)
Mean daily number of new blisters greater than 3
Subject or caregiver agrees not to use any topical therapies other than those approved by the investigator

You will not qualify if you...

EBS lesions requiring oral therapy to treat an infection
Use of any diacerein-containing product within 6 months prior to first visit
Use of systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to first visit
Use of systemic steroidal therapy within 30 days prior to first visit
Use of any systemic product that might put the subject at undue risk or interfere with study assessments within 30 days prior to first visit

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