localized epidermolysis bullosa simplex MedDRA version: 20.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Botox
Pharmaceutical Form: Powder and solution for solution for injection
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Intradermal use
Sponsors
Toulouse Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Diagnosis of LEBS based on clinical symptoms and in some cases histological or molecular findings • Palmar skin lesions: blisters and/or : erosions, edematous and erythematous lesions, crusts. 3 lesions per foot, as a minimum • Similar clinical severity of skin lesions on both feet • Patient aged 18 or older • Patient with social security • Written consent of the patient • Patient able to understand the study’s questionnaires Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 20 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: • Patients with only one leg and a different number of toes on each foot. • Known hypersensitivity to botulinic toxin or its excipients • Current treatment with aminosides • Myasthenia • Swallowing difficulties • Past medical history of dysphagia or pneumopathy of inhalation • Known allergy or contraindications to lidocaine, prilocaine, paracetamol or nitrous oxide • Pregnancy (positive pregnancy test (ß-HCG) for women of childbearing age, performed within the 2 days prior to the study. Breastfeeding. • Mental or physical or judicial incapacity to fill the questionnaires • Guardianship patients • Skin infection on the soles at the time of the inclusion • Skin lesions located on the soles, not related to LEBS (ie. post traumatic wound, wart) • Patient suffering from dishydrosis • Botulinic toxin injections in the previous 6 months • Inclusion in another study in the previous 2 months
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluation by the independent centralized assessor, to M3 versus baseline, of the global clinical improvement at the level of the sole of the foot handled by botulinique toxin vs. Untreated sole of the foot at grown-up patients affected by EBSL.;Secondary Objective: Evaluation by the independent centralized assessor, to M6 versus baseline, the global clinical improvement Evaluation to M3 then to M6, respectively versus baseline, of the global clinical improvement Evaluation to M3 then to M6, respectively versus baseline, the efficiency of the treatment(processing) by toxin botulinique on the reduction of the number of hurts Evaluation to M3 then to M6, respectively versus baseline, the efficiency of the treatment(processing) by toxin botulinique on the reduction of the use of bandages Evaluation to M3 then to M6, respectively versus baseline, the efficiency by toxin botulinique of the treatment(processing) on the reduction of the cutaneous surface;Primary end point(s): Global clinical improvement on each foot assessed by a blinded centralized independent reviewer using photographs, at M3 vs.baseline: IGA score (Improvement Global Assessment) assessed for each foot. IGA score is a 5-point scale 0=no improvement or worsening / 1=minimal improvement / 2=moderate improvement / 3=significant improvement / 4=total disappearance.;Timepoint(s) of evaluation of this end point: Day 1 (Baseline), Month 3 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Global clinical improvement on each foot assessed by a blinded centralized independent reviewer using photographs, at M6 vs. baseline: IGA score (Improvement Global Assessment) for each foot. IGA score is a 5-point scale : 0=no improvement or worsening / 1=minimal improvement / 2=moderate improvement / 3=significant improvement / 4=total disappearance.;Timepoint(s) of evaluation of this end point: day 1 (Baseline), Month 6 | — |
Countries
France
Contacts
Public ContactDREYFUS
Toulouse hospital
Outcome results
None listed