Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita

The recruitment status of this study is unknown because the information has not been verified recently.

Verified March 2011 by Uppsala University.
Recruitment status was Recruiting

Sponsor:

Information provided by:

Uppsala University

ClinicalTrials.gov Identifier:

NCT00936533

First received: July 8, 2009

Last updated: March 8, 2011

Last verified: March 2011

History of Changes

Purpose

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).

Condition	Intervention	Phase
Epidermolysis Bullosa Simplex	Drug: Dysport® (Botulinumtoxin A (Btx A)) Drug: Placebo	Phase 2


Study Type:	Interventional
Study Design:	Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Official Title:	Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita - a Double-blind Placebo-controlled Phase II Proof of Concept Study

Resource links provided by NLM:

Genetics Home Reference related topics: ankyloblepharon-ectodermal defects-cleft lip/palate syndrome epidermolysis bullosa simplex epidermolysis bullosa with pyloric atresia hypohidrotic ectodermal dysplasia junctional epidermolysis bullosa pachyonychia congenita

MedlinePlus related topics: Botox Foot Health

Drug Information available for: OnabotulinumtoxinA

Genetic and Rare Diseases Information Center resources: Epidermolysis Bullosa Epidermolysis Bullosa Simplex Pachyonychia Congenita Ectodermal Dysplasia

U.S. FDA Resources

Further study details as provided by Uppsala University:

Primary Outcome Measures:

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. [ Time Frame: 5 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

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


Estimated Enrollment:	40
Study Start Date:	May 2009
Estimated Study Completion Date:	December 2012
Estimated Primary Completion Date:	December 2012 (Final data collection date for primary outcome measure)

Intervention Details:

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.

Other Name: Dysport®

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

Other Name: Dysport Placebo

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.

Eligibility


Ages Eligible for Study:	16 Years and older (Child, Adult, Senior)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00936533

Contacts


Contact: Carl Swartling	+46 18 611 50 97

Locations


Sweden
Svettmottagningen, Sofiahemmet	Recruiting
Stockholm, Sweden, SE - 114 86
Contact: Carl Swartling +46 8 406 24 31
Principal Investigator: Carl Swartling

Sponsors and Collaborators

Uppsala University

Investigators


Principal Investigator:	Carl Swartling	Uppsala University

More Information


Responsible Party:	Dr Carl Swartling, Sophiahemmet
ClinicalTrials.gov Identifier:	NCT00936533 History of Changes
Other Study ID Numbers:	EBSBTXA09 EudraCT number 2009-010763-17
Study First Received:	July 8, 2009
Last Updated:	March 8, 2011
Health Authority:	Sweden: Medical Products Agency

Additional relevant MeSH terms:


Epidermolysis Bullosa Epidermolysis Bullosa Simplex Nails, Malformed Pachyonychia Congenita Skin Abnormalities Congenital Abnormalities Skin Diseases, Genetic Genetic Diseases, Inborn Skin Diseases Skin Diseases, Vesiculobullous Pathological Conditions, Anatomical Ectodermal Dysplasia Nail Diseases	AbobotulinumtoxinA Botulinum Toxins, Type A OnabotulinumtoxinA IncobotulinumtoxinA Botulinum Toxins Acetylcholine Release Inhibitors Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action Cholinergic Agents Neurotransmitter Agents Physiological Effects of Drugs Neuromuscular Agents Peripheral Nervous System Agents

ClinicalTrials.gov processed this record on September 23, 2016

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