Epidermolysis Bullosa SimplexPhase 2NCT00936533
Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita
A Phase 2 study testing Dysport® (Botulinumtoxin A (Btx A)) in people with Epidermolysis Bullosa Simplex, run by Uppsala University at 1 site in Sweden.
Trial 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). Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Epidermolysis Bullosa Simplex
- What is being tested?
- Dysport® (Botulinumtoxin A (Btx A))
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteSweden
- Study dates
- May 2009to Dec 2012
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Carl Swartling contact
Sweden1 site, 1 recruiting
- Recruiting
Svettmottagningen, Sofiahemmet
Stockholm, SE - 114 86
Carl Swartling, +46 8 406 24 31
Carl Swartling
About this study
Official title: Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita - a Double-blind Placebo-controlled Phase II Proof of Concept Study
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).
Read the 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.
- Conditions
- Epidermolysis Bullosa Simplex
- Being tested
- Dysport® (Botulinumtoxin A (Btx A)), Placebo
- Medical subject headings
- Epidermolysis Bullosa Simplex
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dysport® (Botulinumtoxin A (Btx A))Drug
Also called Dysport®
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.
PlaceboDrug
Also called Dysport Placebo
Placebo preparation without active compound, administered as the active drug.
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 5 weeks
Secondary outcomes (5)
Quality of life using a questionnaire (DLQI)
Measured: 12 or 17 weeks
Pain in feet using a questionnaire (VAS)
Measured: 5, 12 (and 17) weeks
Effect duration measured with manometry (same as primary end-point but measured at week 12)
Measured: 12 weeks
Safety by recording of reported adverse events (AE) by the patient at the clinic visits
Measured: 5, 12 (and 17) weeks
Foot-sweating using iodine-starch imprints before and after injections
Measured: 0 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2009 (estimated)
- Study startMay 2009
- Primary completionDec 2012 (estimated)
- Study completionDec 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis Bullosa SimplexStudies of the same condition, open and under way.
- More trials run by Uppsala UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Uppsala University
- Sponsor type
- University, hospital or other
- Investigators
- Carl Swartling, Principal investigator, Uppsala University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00936533
- Sponsor's protocol ID
- EBSBTXA09
- Other IDs
- EudraCT number 2009-010763-17
- Status last verified
- Mar 2011
- First submitted
- 8 Jul 2009
- Last updated
- 9 Mar 2011
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita - a Double-blind Placebo-controlled Phase II Proof of Concept Study
- ClinicalTrials.gov ID
- NCT00936533
- Acronym / sponsor ID
- EBSBTXA09
- Conditions
- Epidermolysis Bullosa Simplex
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Uppsala UniversityUniversity, hospital or other
- Study start
- May 2009
- Primary completion
- Dec 2012 (estimated)
- Study completion
- Dec 2012 (estimated)
- First posted
- 10 Jul 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Dysport® (Botulinumtoxin A (Btx A))Also: Dysport®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. | Drug | – |
| PlaceboAlso: Dysport PlaceboPlacebo preparation without active compound, administered as the active drug. | Drug | – |
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
5 secondary outcomes
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2011.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Carl Swartling contact
Carl Swartling principal investigator
Uppsala University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Dysport® (Botulinumtoxin A (Btx A)) in people with Epidermolysis Bullosa Simplex, run by Uppsala University at 1 site in Sweden.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 9 Mar 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Carl Swartling (+46 18 611 50 97), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Mar 2011.