Clinical trial • Phase IV • Dermatology|Rare Disease

APREMILAST for Epidermolysis bullosa simplex

Phase IV trial of APREMILAST for Epidermolysis bullosa simplex. open-label, none/not specified-controlled. 20 participants.

Overview

open-label, none/not specified-controlled Phase IV trial across 5 sites in France.

Recruits 20 paediatric patients.

Vulnerable Population: Vulnerable population not selected in the population selection (isVulnerablePopulationSelected:false). Age-specific subject information and informed consent/assent forms are provided (documents: L1_SIS and ICF_6 12 ans; L1_SIS and ICF_13-15; L1_SIS and ICF_16 17 ans; L1_SIS and ICF_parent; L1_SIS and ICF_Adulte), indicating parental/guardian consent for minors and age-appropriate assent/ICF for children/adolescents.

{"criterion_text":"- Male or female patients 6 years or older\n- Laboratory confirmed diagnosis of EBS-sev due to KRT5 or 14 mutation (autosomal)\n- Mean daily number of new blisters >4.\n- Subject/caregiver agrees not to use any topical therapies other than the investigator approved"}

{"criterion_text":"- EBS lesions requiring oral therapy to treat an infection\n- Use of any diacerein containing product within 6 months prior to Visit 1\n- Use of systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Visit 1\n- Use of systemic steroidal therapy within 30 days prior to Visit 1\n- 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"}

{"endpoint_text":"- The aim of this study is to assess the efficacy of apremilast in the treatment of patients with EBS-sev.","definition_or_measurement_approach":""}

{"endpoint_text":"- Safety and tolerability will be assessed through the description of the following specific events occurring during the treatment periods of the study plus 1 week to be conservative","definition_or_measurement_approach":""}
{"endpoint_text":"- Secondary efficacy and health outcomes measures:Severity, itch, pain, Duration of dressing, Quality of life","definition_or_measurement_approach":""}
{"endpoint_text":"- compliance","definition_or_measurement_approach":""}
{"endpoint_text":"- Validation of a new severity scale for EBS-sev patients","definition_or_measurement_approach":""}

Planned Sample Size: 20
Recruitment Window Months: 30
Consent Approach: Age-specific informed consent and assent documents are provided. Documents available include adult ICF (L1_SIS and ICF_Adulte), parent/guardian ICF (L1_SIS and ICF_parent), and child/adolescent ICFs for ages 6-12, 13-15 and 16-17 (L1_SIS and ICF 6 12 ans; L1_SIS and ICF_13-15; L1_SIS and ICF_16 17 ans), indicating parental/guardian consent for minors and age-appropriate assent/ICF for children/adolescents.

Investigational Product Name: Otezla 10mg, 20mg, 30 mg film-coated tablets
Active Substance: APREMILAST
Modality: Small molecule
Routes Of Administration: ORAL
Route: ORAL
Authorisation Status: Marketing authorisation number: EU/1/14/981/001 (prodAuthStatus:2)
Dose Levels: 10 mg, 20 mg, 30 mg
Maximum Dose: 60 mg

Investigational Product Name: Otezla 30 mg film-coated tablets
Active Substance: APREMILAST
Modality: Small molecule
Routes Of Administration: ORAL USE
Route: ORAL
Authorisation Status: Marketing authorisation number: EU/1/14/981/002 (prodAuthStatus:2)
Dose Levels: 30 mg
Maximum Dose: 60 mg

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