Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita - a Double-blind Placebo-controlled Phase II Proof of Concept Study
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 40
- 试验地点
- 2
- 主要终点
- Determination of the difference between treated and untreated foot in change of pressure (measured by manometry) to reach unbearable pain, VAS=8, from baseline to 5 week after injection.
研究概览
简要总结
This study evaluates the clinical effect of foot injection of the bacteria protein Botulinum toxin A on plantar pain in patients with EBS (epidermolysis bullosa simplex) or PC (pachyonychia congenita).
详细描述
Epidermolysis bullosa simplex (EBS) is the most common subtype of epidermolysis bullosa and mutations in keratin genes (KRT 5/14) are responsible for this condition. Painful blisters and keratoderma of the feet are common and the blisters in EBS typically get worse in the summer due to sweating and increased environmental heat. Since 2010, also the related condition pachyonychia congenita (PC) is included in the study. The primary objective of the study is to evaluate the clinical effect of Botulinum toxin A (Dysport®), injected in the foot, on plantar pain in patients with EBS or PC. Quality of life, pain in feet, effect duration, plantar sweating and safety of the treatment will also be studied.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Informed consent received from patient
- •Informed consent received from patient´s parents (when patient < 18 years)
- •A solid EBS-diagnosis based on genetic analysis and/or phenotype. The EBS-diagnosis will be performed by Professor Anders Vahlquist, Swedish Centre of Genetic skin disorders, Uppsala.
- •Age > 16 years
- •Patients must be previously untreated with Btx A
- •If female, patient is post-menopausal, surgically sterilized, or willing to use an acceptable method of birth control
排除标准
- •Contraindication to Btx A
- •Contraindication to general anaesthesia
- •Use of aminoglycosides, tetracyclines , spectinomycin, lincomycin, polymyxin or muscle relaxants
- •Pregnancy or lactation
- •Patients unwilling to meet the requirements of the protocol
- •Other medical or social reasons for exclusion at the discretion of the Investigator
结局指标
主要结局
Determination of the difference between treated and untreated foot in change of pressure (measured by manometry) to reach unbearable pain, VAS=8, from baseline to 5 week after injection.
时间窗: 5 weeks
次要结局
- Pain in feet using a questionnaire (VAS)(5, 12 (and 17) weeks)
- Quality of life using a questionnaire (DLQI)(12 or 17 weeks)
- Effect duration measured with manometry (same as primary end-point but measured at week 12)(12 weeks)
- Safety by recording of reported adverse events (AE) by the patient at the clinic visits(5, 12 (and 17) weeks)
- Foot-sweating using iodine-starch imprints before and after injections(0 weeks)