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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-002332-16 | EudraCT Number |
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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Botulinic toxin | Experimental | Injections of botulinic toxin (Dysport®, Allergan) 200 UI |
|
| Placebo | Placebo Comparator | Injections of physiological serum |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Botulinic toxin | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | at baseline vs month 3 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | at baseline vs month 6 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Juliette Mazereeuw-Hautier, MD | Contact | 5 67 77 81 41 | 33 | mazereeuw-hautier.j@chu-toulouse.fr |
| Isabelle DREYFUS, PharmD | Contact | 5 67 77 81 10 | 33 | dreyfus.i@chu-toulouse.fr |
| Name | Affiliation | Role |
|---|---|---|
| Juliette Mazereeuw-Hautier, MD | University Hospital, Toulouse | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Hospital Bordeaux | Recruiting | Bordeaux | 33000 | France |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 24690439 | Background | 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. | |
| 19945613 |
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| ID | Term |
|---|---|
| D016110 | Epidermolysis Bullosa Simplex |
| D001768 | Blister |
| ID | Term |
|---|---|
| D004820 | Epidermolysis Bullosa |
| D012868 | Skin Abnormalities |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C542869 | abobotulinumtoxinA |
| D015507 | Drugs, Investigational |
| ID | Term |
|---|---|
| D004364 | Pharmaceutical Preparations |
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Each subject is his own witness, receiving the 2 treatments : left foot vs. right foot
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|
|
| Placebo | 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 physiological serum (200 UI) on right or left foot |
|
|
| 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 | at month1, month 3 and month 6 respectively vs baseline |
| Efficacy assessment concerning the number of plantar lesions clinically observed by the investigator | on each foot (erythematous and edematous areas, blisters, skin erosion, crusts) | at baseline, month 1, month 3 and month 6 |
| 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. | at baseline, month 1, month 3 and month 6 |
| 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) | at month 1, month 3, month 6 and month 9 |
| 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). | at baseline, month 1, month 3 and month 6 |
| 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 | Day 0 |
| 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. | Day 0, month 1, month 3 and month 6 and month 9 |
| University Hospital Nice | Recruiting | Nice | 06000 | France |
|
| Saint-Louis Hospital - APHP | Recruiting | Paris | 75000 | France |
|
| Hôpital Larrey - CHU Toulouse | Recruiting | Toulouse | 31059 | France |
|
| Background |
| Sprecher E. Epidermolysis bullosa simplex. Dermatol Clin. 2010 Jan;28(1):23-32. doi: 10.1016/j.det.2009.10.003. |
| 18828848 | Background | 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. |
| 19153338 | Background | 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. |
| 24650374 | Background | 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. |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012872 | Skin Diseases, Vesiculobullous |
| D020763 | Pathological Conditions, Anatomical |
| D013568 | Pathological Conditions, Signs and Symptoms |