Evaluation of the Efficacy of Injections of Botulinic Toxin in Plantar Lesions of Patients Suffering From Localized Epidermolysis Bullosa Simplex : Double Blind Randomized Controlled Study.
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 25
- 试验地点
- 4
- 主要终点
- 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
研究概览
简要总结
The investigators hypothesize that palmar injections of botulinic toxin, via an inhibition of the sudation, would limit the occurrence of blisters in localized epidermolysis bullosa simplex (LEBS).
详细描述
Epidermolysis bullosa is a group of rare genetic diseases characterized by the occurrence of blisters and erosions due to skin fragility. There are 4 different subgroups, based on the location of the skin cleavage area. The most frequent subgroup is the simplex form, consisting predominantly of the localized form (localized epidermolysis bullosa simplex: LEBS). The incidence of LEBS was estimated at between 1/318.000 and 1/35.000. The disease starts early in infancy by the occurrence of blisters and erosions located on soles, secondary to frictions during the walk. The phenomenon is worsened by heat and sudation. LEBS is due to mutations in keratin genes. Life expectancy in LEBS is normal but the quality of life is significantly impaired due to permanent skin pain and limitation of everyday activities (walking, sports). There is no effective or curative treatment. Patients must limit the frictions, protect the skin and use plasters in case of skin lesions.
Botulinic toxin has an agreement for the treatment of axillary hyperhidrosis and has been shown to be also effective on palms and soles. The efficacy of botulinic toxin in plantar lesions of LEBS has been reported in the literature (one case report and a short retrospective series of 6 patients) but there is no proper study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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 with social security
- •Written consent of the patient
- •Patient able to understand the study's questionnaires
排除标准
- •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
- •Respiratory disorders
- •Past medical history of dysphagia or pneumopathy of inhalation
- •Known allergy or contraindications to lidocaine, prilocaine, paracetamol or nitrous oxide
- •Pregnancy (positive pregnancy test (β-HCG) for women of childbearing age, performed within the 2 days prior to the study. Breastfeeding.
- •Contraception during 6 months from inclusion
- •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
研究组 & 干预措施
Botulinic toxin
Injections of botulinic toxin (Dysport®, Allergan) 200 UI
干预措施: Botulinic toxin (Drug)
Placebo
Injections of physiological serum
干预措施: Placebo (Drug)
结局指标
主要结局
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
时间窗: at baseline vs month 3
IGA score is a 5-point scale (range from 0 to 4): 0=no improvement or worsening / 1=minimal improvement / 2=moderate improvement / 3=significant improvement / 4=total disappearance
次要结局
- Efficacy assessment concerning the number of plantar lesions clinically observed by the investigator(at baseline, month 1, month 3 and month 6)
- Efficacy assessment on each foot of the affected plantar skin surface (blisters, erosions, erythematous and edematous areas, crusts)(at baseline, month 1, month 3 and month 6)
- Efficacy assessment by the patient himself, for each foot(at month 1, month 3, month 6 and month 9)
- Immediate tolerance during injection :(Day 0)
- Mid-term and long-term tolerance(Day 0, month 1, month 3 and month 6 and month 9)
- Global clinical improvement on each foot assessed by a blinded centralized independent reviewer using photographs: IGA score (Improvement Global Assessment) for each foot(at baseline vs month 6)
- Global clinical improvement on each foot assessed by the investigator: IGA score (Improvement Global Assessment) assessed for each foot.(at month1, month 3 and month 6 respectively vs baseline)
- Plantar pain assessment by the patient himself, for each foot(at baseline, month 1, month 3 and month 6)