Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia congenita-a double-blind placebo-controlled phase II proof of concept study

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: Not specified

Inclusion Criteria

•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 (i.e. a hormonal contraceptive, intra-uterine device, diaphragm with spermicide, condom*) throughout the study. If male with a female partner of childbearing potential, willing to use an acceptable method of birth control for the study duration of the study, for both female and male, acceptable birth control must be used for at least 3 months after the last dose of study medication.* A condom alone is not considered an acceptable method for birth control. Two barrier methods only are not considered an acceptable method of birth control.

Are the trial subjects under 18? yes
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
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

•Contraindication to Btx A
•Contraindication to general anaesthesia
•Use of aminoglycosides, tetracyklines , 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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of the study is to evaluate the clinical effect of Botulinum toxin A (Dysport®) treatment of plantar pain and quality of life in patients with EBS and PC.;Secondary Objective: Quality of life<br>Pain in feet <br>Effect duration<br>Plantar sweating before Btx A treatment<br>The safety of the Btx A treatment<br>;Primary end point(s): The primary endpoint is to measure pain using a VAS instrument and a quality of life questionnaire.

Secondary Outcome Measures

Name	Time	Method

Related Trials