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Epidermolysis Bullosa SimplexPhase 2/3EBToxNCT03453632

Injections of Botulinic Toxin in Plantar Lesions of Localized Epidermolysis Bullosa Simplex

A Phase 2/3 study testing Botulinic toxin in people with Epidermolysis Bullosa Simplex, run by University Hospital, Toulouse at 4 sites in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The investigators hypothesize that palmar injections of botulinic toxin, via an inhibition of the sudation, would limit the occurrence of blisters in localized epidermolysis bullosa simplex (LEBS). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Epidermolysis Bullosa Simplex
What is being tested?
Botulinic toxin
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
18 participants
Where
4 sitesFrance
Study dates
14 Jun 2018to 21 Jan 2025
Phase
Phase 2/3
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Diagnosis of LEBS based on clinical symptoms and in some cases histological or molecular findings
  • Palmar skin lesions: blisters and/or : erosions, edematous and erythematous lesions, crusts. 3 lesions per foot, as a minimum
  • Similar clinical severity of skin lesions on both feet
  • Patient with social security
  • Written consent of the patient
  • Patient able to understand the study's questionnaires

Who cannot join

  • Patients with only one leg and a different number of toes on each foot.
  • Known hypersensitivity to botulinic toxin or its excipients
  • Current treatment with aminosides
  • Myasthenia
  • Swallowing difficulties
  • Respiratory disorders
Show all 16Show fewer
  • Past medical history of dysphagia or pneumopathy of inhalation
  • Known allergy or contraindications to lidocaine, prilocaine, paracetamol or nitrous oxide
  • Pregnancy (positive pregnancy test (β-HCG) for women of childbearing age, performed within the 2 days prior to the study. Breastfeeding.
  • Contraception during 6 months from inclusion
  • Mental or physical or judicial incapacity to fill the questionnaires
  • Guardianship patients
  • Skin infection on the soles at the time of the inclusion
  • Skin lesions located on the soles, not related to LEBS (ie. post traumatic wound, wart)
  • Patient suffering from dishydrosis
  • Botulinic toxin injections in the previous 6 months

Trial locations and contacts

4 sites in 1 country.

France4 sites

  • University Hospital Bordeaux

    Bordeaux, 33000

  • University Hospital Nice

    Nice, 06000

  • Saint-Louis Hospital - APHP

    Paris, 75000

  • Hôpital Larrey - CHU Toulouse

    Toulouse, 31059

Other trials in France

About this study

Official title: Evaluation of the Efficacy of Injections of Botulinic Toxin in Plantar Lesions of Patients Suffering From Localized Epidermolysis Bullosa Simplex : Double Blind Randomized Controlled Study.

The investigators hypothesize that palmar injections of botulinic toxin, via an inhibition of the sudation, would limit the occurrence of blisters in localized epidermolysis bullosa simplex (LEBS).

Read the detailed description

Epidermolysis bullosa is a group of rare genetic diseases characterized by the occurrence of blisters and erosions due to skin fragility. There are 4 different subgroups, based on the location of the skin cleavage area. The most frequent subgroup is the simplex form, consisting predominantly of the localized form (localized epidermolysis bullosa simplex: LEBS). The incidence of LEBS was estimated at between 1/318.000 and 1/35.000. The disease starts early in infancy by the occurrence of blisters and erosions located on soles, secondary to frictions during the walk. The phenomenon is worsened by heat and sudation. LEBS is due to mutations in keratin genes. Life expectancy in LEBS is normal but the quality of life is significantly impaired due to permanent skin pain and limitation of everyday activities (walking, sports). There is no effective or curative treatment. Patients must limit the frictions, protect the skin and use plasters in case of skin lesions.

Botulinic toxin has an agreement for the treatment of axillary hyperhidrosis and has been shown to be also effective on palms and soles. The efficacy of botulinic toxin in plantar lesions of LEBS has been reported in the literature (one case report and a short retrospective series of 6 patients) but there is no proper study.

Being tested
Botulinic toxin, Placebo
Keywords
Dermatology, Rare skin diseases, epidermolysis bullosa simplex, botulinic toxin, blisters
Medical subject headings
Epidermolysis Bullosa Simplex, Blister

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Botulinic toxinDrug

    Also called Dysport® (experimental drug)

    Clinical photographs of the soles Standardized anesthesia protocol (lidocaine, prilocaine on soles, cryo-spray, oral administration of paracetamol and only if necessary oxycodone and hydroxyzine).

    Cleaning and antiseptic on the soles Injections of botulinic toxin (200 UI) on right or left foot

  • PlaceboDrug

    Also called physiological serum

    Clinical photographs of the soles Standardized anesthesia protocol (lidocaine, prilocaine on soles, cryo-spray, oral administration of paracetamol and only if necessary oxycodone and hydroxyzine).

    Cleaning and antiseptic on the soles Injections of physiological serum (200 UI) on right or left foot

Study groups in NCT03453632
Study groupRoleWhat they receive
Botulinic toxinExperimentalInjections of botulinic toxin (Dysport®, Allergan) 200 UI
Receives: Drug: Botulinic toxin
PlaceboPlacebo comparatorInjections of physiological serum
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Global clinical improvement on each foot assessed by a blinded centralized independent reviewer using photographs, at M3 vs.baseline: IGA score (Improvement Global Assessment) assessed for each foot

    IGA score is a 5-point scale (range from 0 to 4): 0=no improvement or worsening / 1=minimal improvement / 2=moderate improvement / 3=significant improvement / 4=total disappearance

    Measured: at baseline vs month 3

Secondary outcomes (8)

  1. Global clinical improvement on each foot assessed by a blinded centralized independent reviewer using photographs: IGA score (Improvement Global Assessment) for each foot

    IGA score is a 5-point scale (range from 0 to 4): 0=no improvement or worsening / 1=minimal improvement / 2=moderate improvement / 3=significant improvement / 4=total disappearance

    Measured: at baseline vs month 6

  2. Global clinical improvement on each foot assessed by the investigator: IGA score (Improvement Global Assessment) assessed for each foot.

    IGA score is a 5-point scale (range from 0 to 4): 0=no improvement or worsening / 1=minimal improvement / 2=moderate improvement / 3=significant improvement / 4=total disappearance

    Measured: at month1, month 3 and month 6 respectively vs baseline

  3. Efficacy assessment concerning the number of plantar lesions clinically observed by the investigator

    on each foot (erythematous and edematous areas, blisters, skin erosion, crusts)

    Measured: at baseline, month 1, month 3 and month 6

  4. Efficacy assessment on each foot of the affected plantar skin surface (blisters, erosions, erythematous and edematous areas, crusts)

    calculation of this affected surface by delimiting its contours on a standardized photography using "Image J" software = computerized assessment by a blinded centralized independent reviewer.

    Measured: at baseline, month 1, month 3 and month 6

  5. Efficacy assessment by the patient himself, for each foot

    Global improvement assessed with a 5-point score (0=no improvement or worsening / 1=minimal improvement / 2=moderate improvement / 3=significant improvement / 4=total disappearance)

    Measured: at month 1, month 3, month 6 and month 9

  6. Plantar pain assessment by the patient himself, for each foot

    Plantar pain assessment , using a 0 to 10 pain EVA scale. To perform the assessment, patient is requested to be standing on one foot (assessment of pain felt for this foot using EVA scale) then, standing on the other foot (assessment of pain felt for this other foot using EVA scale).

    Measured: at baseline, month 1, month 3 and month 6

  7. Immediate tolerance during injection :

    Assessment performed by the patient of the pain felt during the act for each foot, using a 0 to 10 pain EVA scale (patient interview). Assessment performed by the investigator of local adverse events (for each foot) or general adverse events, during the injections

    Measured: Day 0

  8. Mid-term and long-term tolerance

    Assessment performed by the patient of local adverse events (for each foot) or general adverse events reported in the home patient's diary between the protocol visits (collected at month 1, month 3, month 6 and month 9). • Assessment performed by investigator of local adverse events (for each foot) or general adverse events, at D0 (at the end of/after the injections), M3 and M6.

    Measured: Day 0, month 1, month 3 and month 6 and month 9

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Mar 2018
  2. Study start14 Jun 2018
  3. Primary completion21 Jan 2025
  4. Study completion21 Jan 2025

Resources

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 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Model description
Each subject is his own witness, receiving the 2 treatments : left foot vs. right foot
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
18 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03453632
Sponsor's protocol ID
RC31/16/8917
Other IDs
2017-002332-16, 2017
Acronym
EBTox
Status last verified
Sep 2026
First submitted
6 Feb 2018
Last updated
21 Sep 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Fine JD, Bruckner-Tuderman L, Eady RA, Bauer EA, Bauer JW, Has C, Heagerty A, Hintner H, Hovnanian A, Jonkman MF, Leigh I, Marinkovich MP, Martinez AE, McGrath JA, Mellerio JE, Moss C, Murrell DF, Shimizu H, Uitto J, Woodley D, Zambruno G. Inherited epidermolysis bullosa: updated recommendations on diagnosis and classification. J Am Acad Dermatol. 2014 Jun;70(6):1103-26. doi: 10.1016/j.jaad.2014.01.903. Epub 2014 Mar 29.

    PubMed 24690439

  • Sprecher E. Epidermolysis bullosa simplex. Dermatol Clin. 2010 Jan;28(1):23-32. doi: 10.1016/j.det.2009.10.003.

    PubMed 19945613

  • Langan SM, Williams HC. A systematic review of randomized controlled trials of treatments for inherited forms of epidermolysis bullosa. Clin Exp Dermatol. 2009 Jan;34(1):20-5. doi: 10.1111/j.1365-2230.2008.02789.x. Epub 2008 Sep 25.

    PubMed 18828848

  • Abitbol RJ, Zhou LH. Treatment of epidermolysis bullosa simplex, Weber-Cockayne type, with botulinum toxin type A. Arch Dermatol. 2009 Jan;145(1):13-5. doi: 10.1001/archdermatol.2008.546. No abstract available.

    PubMed 19153338

  • Schwieger-Briel A, Chakkittakandiyil A, Lara-Corrales I, Aujla N, Lane AT, Lucky AW, Bruckner AL, Pope E. Instrument for scoring clinical outcome of research for epidermolysis bullosa: a consensus-generated clinical research tool. Pediatr Dermatol. 2015 Jan-Feb;32(1):41-52. doi: 10.1111/pde.12317. Epub 2014 Mar 20.

    PubMed 24650374

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Botulinic toxin in people with Epidermolysis Bullosa Simplex, run by University Hospital, Toulouse at 4 sites in France.

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 16 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: finished, as of the sponsor's last update on 21 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in France. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined 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.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Global clinical improvement on each foot assessed by a blinded centralized independent reviewer using photographs, at M3 vs.baseline: IGA score (Improvement Global Assessment) assessed for each foot (time frame: at baseline vs month 3). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03453632, which also lists 5 linked publications.

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 21 Sep 2026.

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