Clinical Trial: Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita

Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional

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

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).

Detailed Summary: 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.
Sponsor: Uppsala University

Current Primary Outcome: 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 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

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


Original Secondary Outcome:

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


Information By: Uppsala University

Dates:
Date Received: July 8, 2009
Date Started: May 2009
Date Completion: December 2012
Last Updated: March 8, 2011
Last Verified: March 2011