Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita

Epidermolysis Bullosa Simplex Clinical Trial

Official title:

Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita - a Double-blind Placebo-controlled Phase II Proof of Concept Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00936533
• Other study ID #: EBSBTXA09
• Secondary ID: EudraCT number 2
• Status: Recruiting
• Phase: Phase 2
• First received: July 8, 2009
• Last updated: March 8, 2011
• Start date: May 2009
• Est. completion date: December 2012

Study information

• Verified date: March 2011
• Source: Uppsala University
• Contact: Carl Swartling
• Phone: +46 18 611 50 97
• Is FDA regulated: No
• Health authority: Sweden: Medical Products Agency
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: December 2012
• Est. primary completion date: December 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 16 Years and older

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Epidermolysis Bullosa
• Epidermolysis Bullosa Simplex
• Pachyonychia Congenita

Intervention

Drug:
• 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.
• Placebo
Placebo preparation without active compound, administered as the active drug.

Locations

Country	Name	City	State
Sweden	Svettmottagningen, Sofiahemmet	Stockholm

Sponsors (1)

Lead Sponsor	Collaborator
Uppsala University

Country where clinical trial is conducted

Sweden, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Secondary	Quality of life using a questionnaire (DLQI)		12 or 17 weeks	No
Secondary	Pain in feet using a questionnaire (VAS)		5, 12 (and 17) weeks	No
Secondary	Effect duration measured with manometry (same as primary end-point but measured at week 12)		12 weeks	No
Secondary	Safety by recording of reported adverse events (AE) by the patient at the clinic visits		5, 12 (and 17) weeks	Yes
Secondary	Foot-sweating using iodine-starch imprints before and after injections		0 weeks	No