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.

Hailey-Hailey Disease Clinical Trial

— ToxHD  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02782702
• Other study ID #: 14 7316 02
• Status: Completed
• Phase: Phase 1
• Start date: September 2015
• Est. completion date: November 2017

Study information

• Verified date: November 2021
• Source: University Hospital, Toulouse
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: November 2017
• Est. primary completion date: November 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Botulism
• Darier Disease
• Hailey-Hailey Disease

Intervention

Drug:
• Botulism Toxin Treatment
Injection of 50 UI of botulism toxin for treated zone

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Toulouse

References & Publications (1)

Dreyfus I, Maza A, Rodriguez L, Merlos M, Texier H, Rousseau V, Sommet A, Mazereeuw-Hautier J. Botulinum toxin injections as an effective treatment for patients with intertriginous Hailey-Hailey or Darier disease: an open-label 6-month pilot interventiona — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evaluation of quality of life measured by change in the DLQI score	Variation of DLQI score between Baseline and M1	Day 0 and day 30
Secondary	Evaluation of quality of life measured by change in the DLQI score	Variation of DLQI score between Baseline and M3	Day 0 and day 90
Secondary	Evaluation of quality of life measured by change in the DLQI score	Variation of DLQI score between Baseline and M6	Day 0 and day 180
Secondary	Evaluation of skin improvement in treated areas using change the IGA score	Variation of IGA score between Baseline and M1	Day 0 and Day 30
Secondary	Evaluation of skin improvement in treated areas using change the IGA score	Variation of IGA score between Baseline and M3	Day 0 and Day 90
Secondary	Evaluation of skin improvement in treated areas using change the IGA score	Variation of IGA score between Baseline and M6	Day 0 and Day 180
Secondary	Evaluation of psychosocial impairment at measured by change in the HidroQoL score	Variation of HidroQoL score between Baseline and M1	Day 0 and Day 30
Secondary	Evaluation of psychosocial impairment measured by change in the HidroQoL score	Variation of HidroQoL score between Baseline and M3	Day 0 and Day 90
Secondary	Evaluation of psychosocial impairment measured by change in the HidroQoL score	Variation of HidroQoL score between Baseline and M6	Day 0 and Day 180
Secondary	Evaluation by the investigator of the treated lesions global severity change as assessed by comparison using measurement of the affected area	Variation of treated lesions severity between Baseline and M1	Day 0 and Day 30
Secondary	Evaluation by the investigator of the treated lesions global severity change as assessed by comparison using measurement of the affected area	Variation of treated lesions severity between Baseline and M3	Day 0 and Day 90
Secondary	Evaluation by the investigator of the treated lesions global severity change as assessed by comparison using measurement of the affected area	Variation of treated lesions severity between Baseline and M6	Day 0 and Day 180
Secondary	Evaluation of patient's satisfaction Using the IGA score " Improvement Global Assessment "		Day 180
Secondary	Evaluation of patient treatment acceptability using visual analogic pain scale		Day 0 after injection
Secondary	Evaluation of acceptability over the medium to long term as assessed by occurence of side effects		Day 30
Secondary	Evaluation of acceptability over the medium to long term as assessed by occurence of side effects		Day 90
Secondary	Evaluation of acceptability over the medium to long term as assessed by occurence of side effects		Day 180
Secondary	Evaluation of long term efficacy as assessed by percentage of non-responder patients with IGA score egal to 0		Day 30
Secondary	Evaluation of long term efficacy as assessed by delay for significant relapse (reappearance of skin lesions justifying treatment)		Up to 180 days
Secondary	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		Up to 180 days