Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita

NCT00936533 | Unknown Phase 2

• 2 other identifiers: EBSBTXA09EudraCT number 2009-010763-17
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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).

Conditions

Epidermolysis Bullosa Simplex

Outcome Measures

Primary Outcomes (1)

• 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.

  5 weeks

Secondary Outcomes (5)

• Quality of life using a questionnaire (DLQI)

  12 or 17 weeks

• Pain in feet using a questionnaire (VAS)

  5, 12 (and 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

Interventions

Dysport® (Botulinumtoxin A (Btx A)) DRUG

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.

Also known as: Dysport®

Placebo DRUG

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

Also known as: Dysport Placebo

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Uppsala University: lead

Study Sites (1)

Svettmottagningen, Sofiahemmet

Stockholm, SE - 114 86, Sweden

RECRUITING

MeSH Terms

Conditions

Epidermolysis Bullosa Simplex

Interventions

abobotulinumtoxinA

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous

Study Officials

• Carl Swartling

  Uppsala University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Carl Swartling

CONTACT

+46 18 611 50 97

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: July 8, 2009
• First Posted: July 10, 2009
• Study Start: May 1, 2009
• Primary Completion: December 1, 2012
• Study Completion: December 1, 2012
• Last Updated: March 9, 2011

Record last verified: 2011-03

Locations

SE (1)