Epidermolysis Bullosa Simplex Clinical Trial
— EBToxOfficial 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).
Status | Recruiting |
Enrollment | 25 |
Est. completion date | December 2023 |
Est. primary completion date | September 2023 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - 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 Exclusion Criteria: - 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 - Past medical history of dysphagia or pneumopathy of inhalation - Known allergy or contraindications to lidocaine, prilocaine, paracetamol or nitrous oxide - Pregnancy (positive pregnancy test (ß-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 - Inclusion in another study in the previous 2 months |
Country | Name | City | State |
---|---|---|---|
France | University Hospital Bordeaux | Bordeaux | |
France | University Hospital Nice | Nice | |
France | Saint-Louis Hospital - APHP | Paris | |
France | Hôpital Larrey - CHU Toulouse | Toulouse |
Lead Sponsor | Collaborator |
---|---|
University Hospital, Toulouse |
France,
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. — View Citation
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. — View Citation
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. — View Citation
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. — View Citation
Sprecher E. Epidermolysis bullosa simplex. Dermatol Clin. 2010 Jan;28(1):23-32. doi: 10.1016/j.det.2009.10.003. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 | |
Secondary | 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 | |
Secondary | 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 | |
Secondary | 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 | |
Secondary | 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 | |
Secondary | 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 | |
Secondary | 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 | |
Secondary | 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 | |
Secondary | 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 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT02592954 -
Effect of Broccoli Sprout Extract on Keratinocyte Differentiation in Normal Skin
|
Phase 1 | |
Recruiting |
NCT03269474 -
Computational Drug Repurposing for All EBS Cases
|
||
Completed |
NCT05062070 -
Safety and Efficacy of Topical TolaSure Targeting Aggregated Mutant Keratin in Severe Epidermolysis Bullosa Simplex
|
Phase 1 | |
Recruiting |
NCT03016715 -
Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study
|
Phase 2 | |
Completed |
NCT02960997 -
Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study
|
Phase 2 | |
Recruiting |
NCT00936533 -
Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita
|
Phase 2 | |
Completed |
NCT03472287 -
To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB)
|
Phase 1 | |
Recruiting |
NCT04213703 -
A Pilot Study to Explore the Role of Gut Flora in Epidermolysis Bullosa
|
||
Not yet recruiting |
NCT02470689 -
Diacerin for the Treatment of Epidermolysis Bullosa Simplex
|
Phase 2 | |
Terminated |
NCT03154333 -
Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)
|
Phase 2 | |
Active, not recruiting |
NCT05033574 -
The State of Sexual Development in Children With Inherited Epidermolysis Bullosa
|
||
Completed |
NCT04908215 -
INM-755 (Cannabinol) Cream for Treatment of Epidermolysis Bullosa
|
Phase 2 | |
Completed |
NCT03389308 -
Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex
|
Phase 2 |