Epidermolysis Bullosa Simplex and Pachyonychia Congenita
Conditions
Interventions
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 us
Sponsors
Sophiahemmet
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Informed consent received from patient • Informed consent received from patient´s parents (when patient 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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 ;Primary end point(s): The primary endpoint is to measure pain using a VAS instrument and a quality of life questionnaire. | — |
Countries
Sweden
Outcome results
None listed