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
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Epidermolysis Bullosa Simplex | Drug: Dysport® (Botulinumtoxin A (Btx A)) Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 40 participants |
Allocation: | N/A |
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 |

- 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
- 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 ]
- 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 ]

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Ages Eligible for Study: | 16 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

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
Contact: Carl Swartling | +46 18 611 50 97 |
Sweden | |
Svettmottagningen, Sofiahemmet | Recruiting |
Stockholm, Sweden, SE - 114 86 | |
Contact: Carl Swartling +46 8 406 24 31 | |
Principal Investigator: Carl Swartling |
Principal Investigator: | Carl Swartling | Uppsala University |
Responsible Party: | Dr Carl Swartling, Sophiahemmet |
ClinicalTrials.gov Identifier: | NCT00936533 |
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 |
Epidermolysis Bullosa Epidermolysis Bullosa Simplex Pachyonychia Congenita Nails, Malformed Skin Abnormalities Congenital Abnormalities Skin Diseases, Genetic Genetic Diseases, Inborn Skin Diseases Skin Diseases, Vesiculobullous Pathological Conditions, Anatomical Ectodermal Dysplasia Abnormalities, Multiple |
Nail Diseases abobotulinumtoxinA Botulinum Toxins, Type A 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 |