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

NCT06509984 | Recruiting Phase 2

• 2 other identifiers: 23-PP-102023-508794-83-00
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 4

Brief 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.

Conditions

Epidermolysis Bullosa Simplex; Genodermatosis

Keywords

Apremilast; Children; Pediatric

Outcome Measures

Primary Outcomes (1)

• To describe efficacy of apremilast

  number of new blister conting every day

  weeks 20

Secondary Outcomes (1)

• Safety of apremilast treatment

  weeks 20

Study Arms (1)

apremilast treatment

EXPERIMENTAL

treatment by apremilast in two phases of challenge

Drug: Apremilast treatment

Interventions

Apremilast treatment DRUG

treatment of apremilast

apremilast treatment

Eligibility Criteria

• Age: 6 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• \- 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

Sponsors & Collaborators

• Centre Hospitalier Universitaire de Nice: lead

Study Sites (4)

CHU de Nice

Nice, France, 06000, France

RECRUITING

APHP Necker-Enfants-Malades

Paris, France, 75015, France

NOT YET RECRUITING

APHP Hôpital Saint-Louis

Paris, France, 75018, France

NOT YET RECRUITING

chu de Toulouse

Toulouse, 31059, France

NOT YET RECRUITING

Related Publications (1)

• Fontas E, Hubiche T, Maruani A, Moschoi R, Hussain S, Chiaverini C. Multicentre phase II open study assessing the efficacy and safety of apremilast in children and adults with epidermolysis bullosa simplex generalised: study protocol for the EBULO study. BMJ Open. 2025 Nov 12;15(11):e103194. doi: 10.1136/bmjopen-2025-103194.

  PMID: 41224305 DERIVED

MeSH Terms

Conditions

Epidermolysis Bullosa Simplex; Skin Diseases, Genetic

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 15, 2024
• First Posted: July 19, 2024
• Study Start: August 6, 2024
• Primary Completion: October 1, 2025
• Study Completion: April 1, 2026
• Last Updated: February 24, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

FR (4)