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

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2009-010763-17-SE
• Date of registration: 20/03/2009
• Prospective Registration: Yes
• Primary sponsor: Sophiahemmet
• Public title: Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia congenita-a double-blind placebo-controlled phase II proof of concept study
• Scientific title: Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia congenita-a double-blind placebo-controlled phase II proof of concept study
• Date of first enrolment: 26/05/2009
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-010763-17
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no

Countries of recruitment

Sweden

Key inclusion & exclusion criteria

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




Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Epidermolysis Bullosa Simplex and Pachyonychia Congenita

Intervention(s)

Trade Name: Dysport
Pharmaceutical Form: Powder for solution for injection
Pharmaceutical form of the placebo: Powder for solution for injection
Route of administration of the placebo: Subcutaneous use

Primary Outcome(s)

Primary end point(s): The primary endpoint is to measure pain using a VAS instrument and a quality of life questionnaire.

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
Pain in feet
Effect duration
Plantar sweating before Btx A treatment
The safety of the Btx A treatment

Secondary ID(s)

EBSBTXA09