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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Botulism Toxin treatment | Experimental | Injection of 50 UI Botulism toxin for the treated zone |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Botulism Toxin Treatment | Drug | Injection of 50 UI of botulism toxin for treated zone |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Evaluation of quality of life measured by change in the DLQI score |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Aude MAZA RIOLAND, MD | University Hospital, Toulouse | Principal Investigator |
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33602313 | Result | 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 interventional study. Orphanet J Rare Dis. 2021 Feb 18;16(1):93. doi: 10.1186/s13023-021-01710-x. |
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Variation of DLQI score between Baseline and M6
| Day 0 and day 180 |
| 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 |
| 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 |
| 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 |
| 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 |
| Evaluation of psychosocial impairment measured by change in the HidroQoL score | Variation of HidroQoL score between Baseline and M3 | Day 0 and Day 90 |
| Evaluation of psychosocial impairment measured by change in the HidroQoL score | Variation of HidroQoL score between Baseline and M6 | 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 | Variation of treated lesions severity between Baseline and M1 | 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 | Variation of treated lesions severity between Baseline and M3 | 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 | Variation of treated lesions severity between Baseline and M6 | Day 0 and Day 180 |
| Evaluation of patient's satisfaction Using the IGA score " Improvement Global Assessment " | Day 180 |
| Evaluation of patient treatment acceptability using visual analogic pain scale | Day 0 after injection |
| Evaluation of acceptability over the medium to long term as assessed by occurence of side effects | Day 30 |
| Evaluation of acceptability over the medium to long term as assessed by occurence of side effects | Day 90 |
| Evaluation of acceptability over the medium to long term as assessed by occurence of side effects | Day 180 |
| Evaluation of long term efficacy as assessed by percentage of non-responder patients with IGA score egal to 0 | Day 30 |
| Evaluation of long term efficacy as assessed by delay for significant relapse (reappearance of skin lesions justifying treatment) | 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 | Up to 180 days |
| ID | Term |
|---|---|
| D016506 | Pemphigus, Benign Familial |
| D007644 | Darier Disease |
| ID | Term |
|---|---|
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012872 | Skin Diseases, Vesiculobullous |
| D007642 | Keratosis |
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