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Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita

Primary Purpose

Epidermolysis Bullosa Simplex

Status

Unknown status

Phase

Phase 2

Locations

Sweden

Study Type

Interventional

Intervention

Dysport® (Botulinumtoxin A (Btx A))

Placebo

About this trial

This is an interventional treatment trial for Epidermolysis Bullosa Simplex

Eligibility Criteria

16 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Svettmottagningen, SofiahemmetRecruiting

Outcomes

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.

Secondary Outcome Measures

Quality of life using a questionnaire (DLQI)

Pain in feet using a questionnaire (VAS)

Effect duration measured with manometry (same as primary end-point but measured at week 12)

Safety by recording of reported adverse events (AE) by the patient at the clinic visits

Foot-sweating using iodine-starch imprints before and after injections

Full Information

NCT ID

NCT00936533

First Posted

July 8, 2009

Last Updated

March 8, 2011

Sponsor

Uppsala University

1. Study Identification

Unique Protocol Identification Number

NCT00936533

Brief Title

Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2011

Overall Recruitment Status

Unknown status

Study Start Date

May 2009 (undefined)

Primary Completion Date

December 2012 (Anticipated)

Study Completion Date

December 2012 (Anticipated)

3. Sponsor/Collaborators

Name of the Sponsor

Uppsala University

4. Oversight

Data Monitoring Committee

5. 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).

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Epidermolysis Bullosa Simplex

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

N/A

Enrollment

40 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

Dysport® (Botulinumtoxin A (Btx A))

Other Intervention Name(s)

Dysport®

Intervention Description

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.

Intervention Type

Drug

Intervention Name(s)

Placebo

Other Intervention Name(s)

Dysport Placebo

Intervention Description

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

Primary Outcome Measure Information:

Title

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 Measure Information:

Title

Quality of life using a questionnaire (DLQI)

Time Frame

12 or 17 weeks

Title

Pain in feet using a questionnaire (VAS)

Time Frame

5, 12 (and 17) weeks

Title

Effect duration measured with manometry (same as primary end-point but measured at week 12)

Time Frame

12 weeks

Title

Safety by recording of reported adverse events (AE) by the patient at the clinic visits

Time Frame

5, 12 (and 17) weeks

Title

Foot-sweating using iodine-starch imprints before and after injections

Time Frame

0 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

16 Years

Accepts Healthy Volunteers

Eligibility 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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Carl Swartling

Phone

+46 18 611 50 97

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Carl Swartling

Organizational Affiliation

Uppsala University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Svettmottagningen, Sofiahemmet

City

Stockholm

ZIP/Postal Code

SE - 114 86

Country

Sweden

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Carl Swartling

Phone

+46 8 406 24 31

First Name & Middle Initial & Last Name & Degree

Carl Swartling

12. IPD Sharing Statement

Learn more about this trial

Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita

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