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. (ToxHD)

Toulouse University Hospital

Status and phase

Completed

Phase 1

Conditions

Hailey-Hailey Disease

Darier Disease

Treatments

Drug: Botulism Toxin Treatment

Study type

Interventional

Funder types

Other

Identifiers

NCT02782702

14 7316 02

Details and patient eligibility

About

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.

Enrollment

30 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.