Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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
  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:


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:
    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
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
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

No publications provided

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:
Nail Diseases
Epidermolysis Bullosa
Epidermolysis Bullosa Simplex
Pachyonychia Congenita
Congenital Abnormalities
Ectodermal Dysplasia
Genetic Diseases, Inborn
Skin Abnormalities
Skin Diseases
Skin Diseases, Genetic
Skin Diseases, Vesiculobullous
Botulinum Toxins
Botulinum Toxins, Type A
Anti-Dyskinesia Agents
Central Nervous System Agents
Neuromuscular Agents
Peripheral Nervous System Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on November 20, 2014