Clinical Trial: 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.

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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

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.


Detailed Summary:
Sponsor: University Hospital, Toulouse

Current Primary Outcome: 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


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • Evaluation of patient's satisfaction Using the IGA score " Improvement Global Assessment " [ Time Frame: Day 180 ]
  • Evaluation of patient treatment acceptability using visual analogic pain scale [ Time Frame: Day 0 after injection ]
  • Evaluation of acceptability over the medium to long term as assessed by occurence of side effects [ Time Frame: Day 30 ]
  • Evaluation of acceptability over the medium to long term as assessed by occurence of side effects [ Time Frame: Day 90 ]
  • Evaluation of acceptability over the medium to long term as assessed by occurence of side effects [ Time Frame: Day 180 ]
  • Evaluation of long term efficacy as assessed by percentage of non-responder patients with IGA score egal to 0 [ Time Frame: Day 30 ]
  • Evaluation of long term efficacy as assessed by delay for significant relapse (reappearance of skin lesions justifying treatment) [ Time Frame: 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 [ Time Frame: Up to 180 days ]


Original Secondary Outcome: Same as current

Information By: University Hospital, Toulouse

Dates:
Date Received: May 20, 2016
Date Started: September 2015
Date Completion: August 2016
Last Updated: May 20, 2016
Last Verified: May 2016