Assessing Apremilast for Epidermolysis Bullosa Simplex in Children
EBULO. a 20-Week Multicentre, Open Study Assessing the Efficacy and Safety of Apremilast in Patients > 6 Years of Age with EB Simplex Generalized
This study is testing if a medication called Apremilast can help children with Epidermolysis Bullosa Simplex feel better and stay safe over 20 weeks.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 6 Years to 99 Years |
| Sex | All |
| Sponsor | Centre Hospitalier Universitaire de Nice Academic / other |
| Drugs / interventions | chemotherapy, immunotherapy |
| Locations | 4 sites (Nice, France and 3 other locations) |
| Trial ID | NCT06509984 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are male or female patients aged 6 years or older with a confirmed diagnosis of Epidermolysis Bullosa Simplex due to specific genetic mutations.
Not a fit: Patients with active infections requiring oral therapy or those who have recently used certain systemic therapies may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce the frequency of blister formation in patients with Epidermolysis Bullosa Simplex.
How similar studies have performed: While this approach is novel for this specific condition, similar studies evaluating Apremilast in other dermatological conditions have shown promising results.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Nice, France and 3 other locations
- 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)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.