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

NCT03453632 | Unknown Phase 2

• 2 other identifiers: RC31/16/89172017-002332-16
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 4

Brief 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).

Conditions

Epidermolysis Bullosa Simplex

Keywords

Dermatology; Rare skin diseases; epidermolysis bullosa simplex; botulinic toxin; blisters

Outcome Measures

Primary Outcomes (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

  at baseline vs month 3

Secondary Outcomes (8)

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

  at baseline vs month 6

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

  at month1, month 3 and month 6 respectively vs baseline

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

  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)

  at baseline, month 1, month 3 and month 6

• Efficacy assessment by the patient himself, for each foot

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

Study Arms (2)

Botulinic toxin

EXPERIMENTAL

Injections of botulinic toxin (Dysport®, Allergan) 200 UI

Drug: Botulinic toxin

Placebo

PLACEBO COMPARATOR

Injections of physiological serum

Drug: Placebo

Interventions

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

Also known as: Dysport® (experimental drug)

Botulinic toxin

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

Also known as: physiological serum

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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 (β-HCG) for women of childbearing age, performed within the 2 days prior to the study. Breastfeeding.
• Mental or physical or judicial incapacity to fill the questionnaires
• Guardianship patients
• 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

Sponsors & Collaborators

• University Hospital, Toulouse: lead

Study Sites (4)

University Hospital Bordeaux

Bordeaux, 33000, France

RECRUITING

University Hospital Nice

Nice, 06000, France

RECRUITING

Saint-Louis Hospital - APHP

Paris, 75000, France

RECRUITING

Hôpital Larrey - CHU Toulouse

Toulouse, 31059, France

RECRUITING

Related Publications (5)

• 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.

  PMID: 24690439 BACKGROUND

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

  PMID: 19945613 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.

  PMID: 18828848 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.

  PMID: 19153338 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.

  PMID: 24650374 BACKGROUND

MeSH Terms

Conditions

Epidermolysis Bullosa Simplex; Blister

Interventions

abobotulinumtoxinA; Drugs, Investigational

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pharmaceutical Preparations

Study Officials

• Juliette Mazereeuw-Hautier, MD

  University Hospital, Toulouse

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Juliette Mazereeuw-Hautier, MD

CONTACT

5 67 77 81 41; mazereeuw-hautier.j@chu-toulouse.fr

Isabelle DREYFUS, PharmD

CONTACT

5 67 77 81 10; dreyfus.i@chu-toulouse.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Each subject is his own witness, receiving the 2 treatments : left foot vs. right foot

Study Record Dates

• First Submitted: February 6, 2018
• First Posted: March 5, 2018
• Study Start: June 14, 2018
• Primary Completion: September 1, 2023
• Study Completion: December 1, 2023
• Last Updated: August 23, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

FR (4)