Overview

This trial is active, not recruiting.

Conditions hailey-hailey disease, darier disease
Treatment botulism toxin treatment
Phase phase 1
Sponsor University Hospital, Toulouse
Start date September 2015
End date August 2016
Trial size 30 participants
Trial identifier NCT02782702, 14 7316 02

Summary

Hailey Hailey and Darier disease are rare genetic dermatoses. Mutations of 2 genes (ATP2C1 or ATP2A2 respectively) are responsible for the diseases. These genes have a key role in calcium pump; their defect create abnormal link between keratinocytes' desmosomes and induce skin lesions. Clinically, patients present with inflammatory lesions located in the folds. Quality of life is impaired because of pain, pruritus and tendency to infections. Lesions are permanent but acute exacerbations occur in hot seasons because of increased sweating. Usual therapies are often not effective (local treatment, laser, phototherapy). Because sweating is a well established inducing or aggravating factor, botulism toxin could be an effective treatment for these diseases.

Botulism toxin is already used in clinical practice and acts via a decreased sweet secretion. Improvement of skin lesions in Hailey-Hailey or Darier diseases has been previously reported in a few cases but there is no study properly evaluating the benefit of such treatment.

The aim of the project is to study the improvement of quality of life for patients suffering from Hailey-Hailey or Darier diseases after a injections of botulism toxin in large skin folds. The principal objective is to estimate the distribution of the variation of quality of life at M1 vs. baseline.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Intervention model single group assignment
Masking open label
Arm
(Experimental)
Injection of 50 UI Botulism toxin for the treated zone
botulism toxin treatment
Injection of 50 UI of botulism toxin for treated zone

Primary Outcomes

Measure
Evaluation of quality of life measured by change in the DLQI score
time frame: Day 0 and day 30

Secondary Outcomes

Measure
Evaluation of quality of life measured by change in the DLQI score
time frame: Day 0 and day 90
Evaluation of quality of life measured by change in the DLQI score
time frame: Day 0 and day 180
Evaluation of skin improvement in treated areas using change the IGA score
time frame: Day 0 and Day 30
Evaluation of skin improvement in treated areas using change the IGA score
time frame: Day 0 and Day 90
Evaluation of skin improvement in treated areas using change the IGA score
time frame: Day 0 and Day 180
Evaluation of psychosocial impairment at measured by change in the HidroQoL score
time frame: Day 0 and Day 30
Evaluation of psychosocial impairment measured by change in the HidroQoL score
time frame: Day 0 and Day 90
Evaluation of psychosocial impairment measured by change in the HidroQoL score
time frame: Day 0 and Day 180
Evaluation by the investigator of the treated lesions global severity change as assessed by comparison using measurement of the affected area
time frame: Day 0 and Day 30
Evaluation by the investigator of the treated lesions global severity change as assessed by comparison using measurement of the affected area
time frame: Day 0 and Day 90
Evaluation by the investigator of the treated lesions global severity change as assessed by comparison using measurement of the affected area
time frame: Day 0 and Day 180
Evaluation of patient's satisfaction Using the IGA score " Improvement Global Assessment "
time frame: Day 180
Evaluation of patient treatment acceptability using visual analogic pain scale
time frame: Day 0 after injection
Evaluation of acceptability over the medium to long term as assessed by occurence of side effects
time frame: Day 30
Evaluation of acceptability over the medium to long term as assessed by occurence of side effects
time frame: Day 90
Evaluation of acceptability over the medium to long term as assessed by occurence of side effects
time frame: Day 180
Evaluation of long term efficacy as assessed by percentage of non-responder patients with IGA score egal to 0
time frame: Day 30
Evaluation of long term efficacy as assessed by delay for significant relapse (reappearance of skin lesions justifying treatment)
time frame: Up to 180 days
Evaluation of long term efficacy as assessed by comparison between the number of infection episodes occurred during the 6 months before the study or during the 6 months of the study
time frame: Up to 180 days

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - Confirmed diagnosis (clinical and histological features) of Hailey Hailey or Darier diseases. - Moderate to very severe lesions located in large folds - Patient aged 18 ans or more - Patient with health coverage - Patient who have signed the consent form - Patient proficient into filling out the questionnaires. Exclusion Criteria: - Hypersensibility to toxin or excipients - Myastheny - Deglutition's problems - Past medical history of dysphagia or aspiration pneumonia - Pregnancy (positive B-HCG test performed a maxima 72h before) or breastfeeding - Mental , physical incapacity to fill in the questionnaires - Guardianship patients - Skin infections at the inclusion visit - Application in the last 7 days at the site of injection of local treatments (apart emollients or antiseptics) or injections of botulism toxin or dynamic phototherapy or laser in the last 6 months. - Systemic treatment with aminosides in the last 15 days - Inclusion in another study in the last 2 months.

Additional Information

Official title Evaluation of the Improvement of Quality of Life of Patients Suffering From Hailey Hailey or Darier Disease After Injections of Botulism Toxin Into Large Folds. Toxin Hailey Darier
Principal investigator Aude MAZA RIOLAND, MD
Trial information was received from ClinicalTrials.gov and was last updated in May 2016.
Information provided to ClinicalTrials.gov by University Hospital, Toulouse.