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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)

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ClinicalTrials.gov Identifier: NCT02782702
Recruitment Status : Active, not recruiting
First Posted : May 25, 2016
Last Update Posted : May 25, 2016
Sponsor:
Information provided by (Responsible Party):
University Hospital, Toulouse

Brief 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.


Condition or disease Intervention/treatment Phase
Hailey-Hailey Disease Darier Disease Drug: Botulism Toxin Treatment Phase 1

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
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
Study Start Date : September 2015
Estimated Primary Completion Date : August 2016
Estimated Study Completion Date : August 2016


Arm Intervention/treatment
Experimental: Botulism Toxin treatment
Injection of 50 UI Botulism toxin for the treated zone
Drug: Botulism Toxin Treatment
Injection of 50 UI of botulism toxin for treated zone



Primary Outcome Measures :
  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 Outcome Measures :
  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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02782702


Sponsors and Collaborators
University Hospital, Toulouse
Investigators
Principal Investigator: Aude MAZA RIOLAND, MD University Hospital, Toulouse

Responsible Party: University Hospital, Toulouse
ClinicalTrials.gov Identifier: NCT02782702     History of Changes
Other Study ID Numbers: 14 7316 02
First Posted: May 25, 2016    Key Record Dates
Last Update Posted: May 25, 2016
Last Verified: May 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by University Hospital, Toulouse:
Botulism Toxin

Additional relevant MeSH terms:
Darier Disease
Botulism
Pemphigus, Benign Familial
Clostridium Infections
Gram-Positive Bacterial Infections
Bacterial Infections
Neuromuscular Junction Diseases
Neuromuscular Diseases
Nervous System Diseases
Neurotoxicity Syndromes
Foodborne Diseases
Poisoning
Chemically-Induced Disorders
Skin Diseases, Genetic
Genetic Diseases, Inborn
Keratosis
Skin Diseases
Skin Diseases, Vesiculobullous
Botulinum Toxins
Acetylcholine Release Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Cholinergic Agents
Neurotransmitter Agents
Physiological Effects of Drugs