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NCT Number: NCT02782702

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 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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Key information

Conditions

Hailey-Hailey Disease Congenital, Hereditary, and Neonatal Diseases and Abnormalities Darier Disease Genetic Diseases, Inborn Keratosis Pemphigus, Benign Familial Skin Diseases Skin Diseases, Genetic Skin Diseases, Vesiculobullous Skin and Connective Tissue Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Botulism Toxin Treatment

Drug

Injection of 50 UI of botulism toxin for treated zone

Primary outcomes

  1. Evaluation of quality of life measured by change in the DLQI score

    Time frame: Day 0 and day 30

    Variation of DLQI score between Baseline and M1

Secondary outcomes

  1. Evaluation of quality of life measured by change in the DLQI score

    Time frame: Day 0 and day 90

    Variation of DLQI score between Baseline and M3

  2. Evaluation of quality of life measured by change in the DLQI score

    Time frame: Day 0 and day 180

    Variation of DLQI score between Baseline and M6

  3. Evaluation of skin improvement in treated areas using change the IGA score

    Time frame: Day 0 and Day 30

    Variation of IGA score between Baseline and M1

  4. Evaluation of skin improvement in treated areas using change the IGA score

    Time frame: Day 0 and Day 90

    Variation of IGA score between Baseline and M3

  5. Evaluation of skin improvement in treated areas using change the IGA score

    Time frame: Day 0 and Day 180

    Variation of IGA score between Baseline and M6

  6. Evaluation of psychosocial impairment at measured by change in the HidroQoL score

    Time frame: Day 0 and Day 30

    Variation of HidroQoL score between Baseline and M1

  7. Evaluation of psychosocial impairment measured by change in the HidroQoL score

    Time frame: Day 0 and Day 90

    Variation of HidroQoL score between Baseline and M3

  8. Evaluation of psychosocial impairment measured by change in the HidroQoL score

    Time frame: Day 0 and Day 180

    Variation of HidroQoL score between Baseline and M6

  9. 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

    Variation of treated lesions severity between Baseline and M1

  10. 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

    Variation of treated lesions severity between Baseline and M3

  11. 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

    Variation of treated lesions severity between Baseline and M6

  12. Evaluation of patient's satisfaction Using the IGA score " Improvement Global Assessment "

    Time frame: Day 180

  13. Evaluation of patient treatment acceptability using visual analogic pain scale

    Time frame: Day 0 after injection

  14. Evaluation of acceptability over the medium to long term as assessed by occurence of side effects

    Time frame: Day 30

  15. Evaluation of acceptability over the medium to long term as assessed by occurence of side effects

    Time frame: Day 90

  16. Evaluation of acceptability over the medium to long term as assessed by occurence of side effects

    Time frame: Day 180

  17. Evaluation of long term efficacy as assessed by percentage of non-responder patients with IGA score egal to 0

    Time frame: Day 30

  18. Evaluation of long term efficacy as assessed by delay for significant relapse (reappearance of skin lesions justifying treatment)

    Time frame: Up to 180 days

  19. 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

Sponsors and collaborators

Lead sponsor

University Hospital, Toulouse

Other

Registry information

Official study 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

Acronym: ToxHD

Important dates

Study start
2015
Primary completion
2017
Study completion
2017
First posted
May 25, 2016
Registry last updated
Dec 13, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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