Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita

NCT ID: NCT00936533

Last Updated: 2011-03-09

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-05-31
• Study Completion Date: 2012-12-31

Brief Summary

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).

Detailed Description

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.

Conditions

Epidermolysis Bullosa Simplex

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Interventions

Dysport® (Botulinumtoxin A (Btx A))

Single injection in patients´ soles with Btx A in one foot and sodium chloride in the other, with the possibility of a second injection after 12 weeks. The drug will be given in general anaesthesia after curettage of the focal hyperkeratoses. Only the areas with focal hyperkeratoses will be injected with 50 ul per injection.The active drug Btx A (Dysport®, solution for injection) has a concentration of 100 U/ml, and the maximum dose is 300 U per foot.

Intervention Type: DRUG

Placebo

Placebo preparation without active compound, administered as the active drug.

Intervention Type: DRUG

Other Intervention Names

Dysport®; Dysport Placebo

Eligibility 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

Exclusion Criteria

• 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

• Minimum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Uppsala University

OTHER

Sponsor Role: lead

Responsible Party

Sophiahemmet

Principal Investigators

Carl Swartling

Role: PRINCIPAL_INVESTIGATOR

Uppsala University

Locations

Svettmottagningen, Sofiahemmet

Stockholm, , Sweden

Site Status: RECRUITING

Countries

Sweden

Central Contacts

Carl Swartling

Role: CONTACT

+46 18 611 50 97

Facility Contacts

Carl Swartling

Role: primary

+46 8 406 24 31

Other Identifiers

EudraCT number 2009-010763-17

Identifier Type: -

Identifier Source: secondary_id

EBSBTXA09

Identifier Type: -

Identifier Source: org_study_id