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)

• ClinicalTrials.gov Identifier: NCT02782702
• Recruitment Status: Completed
• First Posted: May 25, 2016
• Last Update Posted: December 3, 2018
• Sponsor: University Hospital, Toulouse
• Information provided by (Responsible Party): University Hospital, Toulouse

Tracking Information

• First Submitted Date: May 20, 2016
• First Posted Date: May 25, 2016
• Last Update Posted Date: December 3, 2018
• Study Start Date: September 2015
• Actual Primary Completion Date: November 2017 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 20, 2016)

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 Measures: Same as current

Current Secondary Outcome Measures (submitted: May 20, 2016)

• 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 Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief 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.
• 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 Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Other

Condition

• Hailey-Hailey Disease
• Darier Disease

Intervention

Drug: Botulism Toxin Treatment

Injection of 50 UI of botulism toxin for treated zone

Study Arms

Experimental: Botulism Toxin treatment

Injection of 50 UI Botulism toxin for the treated zone

Intervention: Drug: Botulism Toxin Treatment

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: May 20, 2016): 30
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: November 2017
• Actual Primary Completion Date: November 2017 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Not Provided

Administrative Information

• NCT Number: NCT02782702
• Other Study ID Numbers: 14 7316 02
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Responsible Party: University Hospital, Toulouse
• Study Sponsor: University Hospital, Toulouse
• Collaborators: Not Provided

Investigators

• Principal Investigator: Aude MAZA RIOLAND, MD; University Hospital, Toulouse

• PRS Account: University Hospital, Toulouse
• Verification Date: November 2018