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Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita

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ClinicalTrials.gov Identifier: NCT00936533
Recruitment Status : Unknown
Verified March 2011 by Uppsala University.
Recruitment status was:  Recruiting
First Posted : July 10, 2009
Last Update Posted : March 9, 2011
Sponsor:
Information provided by:

Study Description
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).

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

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.

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Intervention Model: Single Group Assignment
Masking: Quadruple (Participant, Care Provider, 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
Study Start Date : May 2009
Estimated Primary Completion Date : December 2012
Estimated Study Completion Date : December 2012


Arms and Interventions

Intervention Details:
    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.
    Other Name: Dysport®
    Drug: Placebo
    Placebo preparation without active compound, administered as the active drug.
    Other Name: Dysport Placebo

Outcome Measures

Primary Outcome Measures :
  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. [ Time Frame: 5 weeks ]

Secondary Outcome Measures :
  1. Quality of life using a questionnaire (DLQI) [ Time Frame: 12 or 17 weeks ]
  2. Pain in feet using a questionnaire (VAS) [ Time Frame: 5, 12 (and 17) weeks ]
  3. Effect duration measured with manometry (same as primary end-point but measured at week 12) [ Time Frame: 12 weeks ]
  4. Safety by recording of reported adverse events (AE) by the patient at the clinic visits [ Time Frame: 5, 12 (and 17) weeks ]
  5. Foot-sweating using iodine-starch imprints before and after injections [ Time Frame: 0 weeks ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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
First Posted: July 10, 2009    Key Record Dates
Last Update Posted: March 9, 2011
Last Verified: March 2011

Additional relevant MeSH terms:
Pachyonychia Congenita
Epidermolysis Bullosa
Epidermolysis Bullosa Simplex
Nails, Malformed
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
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