Pilot Study Evaluating the Efficiency and the Tolerance of the PDT in the Treatment of Epidermal Dysplasia for Patients Affected by Hereditary DEB

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified March 2014 by Centre Hospitalier Universitaire de Nice
Sponsor:
Information provided by (Responsible Party):
Centre Hospitalier Universitaire de Nice
ClinicalTrials.gov Identifier:
NCT02004600
First received: November 26, 2013
Last updated: March 21, 2014
Last verified: March 2014
  Purpose

The hereditary dystrophic epidermolysis bullosa are genodermatosis responsible of a poor adhesion of the epidermis to the dermis pulling a large mucocutaneous fragility and recurrent spontaneous or posttraumatic bullous detachment. They are caused by mutations in the COL7A1 gene encoding for the collagen VII.

No curative treatment is avaible. The main cause of patients death is the development of squamous cell carcinoma, sometimes multiple and paticularly aggressive in repeated healing part. The photodynamic therapy (PDT) is one of technical reference of multiple actinic keratoses lesions for adults, which are also pre-epithelioma lesions. The PDT is well tolerated even by the elderly and requires only a single session.

The main objective of this study is to determine the efficiency of the photodynamic therapy in the treatment of epidermic dysplasies for patients affected by dystrophic epidermolysis bullosa (DEB). The secondary objectives are to evaluate the tolerance of this treatment in terms of pain and healing, and to evaluate the contribution of confocal microscopy in the diagnosis of epidermal dysplasia for patients affected by hereditary dystrophic epidermolysis bullosa. The main evaluation criterion is the cutaneous biopsy before and after (M2) a PDT session of an epidermal dysplasia area. The secondary criteria are the evaluation of the pain during the PDT session and the healing of the cutaneous lesion at M0, M2 and M4 (lesion area and healing time) and correlation histology / MC. Each patient with a suspicious lesion will be biopsied. In case of agreement for this protocol, there will be 1 PDT session followed by a consultation of control at 2 and 4 months after the end of treatment.


Condition Intervention
Dystrophic Epidermolysis Bullosa
Procedure: Photodynamic therapy (PDT)

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Bicentric, Open and Pilot Study Evaluating the Efficiency and the Tolerance of the Photodynamic Therapy in the Treatment of Epidermal Dysplasia for Patients Affected by Hereditary Dystrophic Epidermolysis Bullosa

Resource links provided by NLM:


Further study details as provided by Centre Hospitalier Universitaire de Nice:

Primary Outcome Measures:
  • Histological examination of a cutaneous biopsy [ Time Frame: 2 MONTHS AFTER ENROLLMENT ] [ Designated as safety issue: No ]
    Histological examination of a cutaneous biopsy at M2 on the epidermal dysplasia area treated with PDT session


Secondary Outcome Measures:
  • Tolerance of PDT [ Time Frame: every 10 minutes during PDT session ] [ Designated as safety issue: No ]
    Tolerance of PDT : evaluation of pain every 10 minutes during PDT session


Estimated Enrollment: 5
Study Start Date: December 2013
Estimated Study Completion Date: June 2015
Estimated Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: patient receiving one PDT session
patient receiving one photodynamic therapy (PDT) session
Procedure: Photodynamic therapy (PDT)
1 session of Photodynamic therapy (PDT) on epidermis dysplasia
Other Name: 1 session of Photodynamic therapy (PDT)

Detailed Description:

The hereditary dystrophic epidermolysis bullosa are genodermatosis responsible of a poor adhesion of the epidermis to the dermis pulling a large mucocutaneous fragility and recurrent spontaneous or posttraumatic bullous detachment. They are caused by mutations in the COL7A1 gene encoding for the collagen VII.

No curative treatment is avaible. The main cause of patients death is the development of squamous cell carcinoma, sometimes multiple and paticularly aggressive in repeated healing part. The early treatment of pre-epithelioma cutaneous lesions to moderate at severe dysplasia type would undoubtedly allow to improve the prognosis of patients. Because of the cutaneous fragility of patients, topical treatments such as imiquimod or 5-FU are not possible. The photodynamic therapy (PDT) is one of technical reference of multiple actinic keratoses lesions for adults, which are also pre-epithelioma lesions. The PDT is well tolerated even by the elderly and requires only a single session.

The main objective of this study is to determine the efficiency of the photodynamic therapy in the treatment of epidermic dysplasies for patients affected by dystrophic epidermolysis bullosa (DEB). The secondary objectives are to evaluate the tolerance of this treatment in terms of pain and healing, and to evaluate the contribution of confocal microscopy in the diagnosis of epidermal dysplasia for patients affected by hereditary dystrophic epidermolysis bullosa. The main evaluation criterion is the cutaneous biopsy before and after (M2) a PDT session of an epidermal dysplasia area. The secondary criteria are the evaluation of the pain during the PDT session and the healing of the cutaneous lesion at M0, M2 and M4 (lesion area and healing time) and correlation histology / MC. This is a bicentric, open and pilot study on 5 patients over 18 years affected by hereditary dystrophic epidermolysis bullosa with epidermal displasia. Carcinomas in situ were excluded of this study. Each patient with a suspicious lesion will be biopsied. In case of agreement for this protocol, there will be 1 PDT session followed by a consultation of control at 2 and 4 months after the end of treatment.

Total study duration: 18 months (12 months for inclusions, 4 months for study, 2 months for data analysis).

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patient carrying a widespread EBDR with moderate to severe epidermal dysplasia on a cutaneous biopsy of a suspicious lesion. Any clinically suspicious lesion should be biopsied in these patients, the inclusion will be made only after obtaining the results of the histological examination.
  • The size of the lesion will be between 10 cm² and 1cm².
  • Systematic Obtaining of the signed informed consent
  • Patient affiliated to Social Security

Exclusion Criteria:

  • Pregnant or lactating women.
  • Patients unable to cooperate for all the duration of the study.
  • Squamous cell carcinoma in situ or invasive on biopsy.
  • Patient treated by chemotherapy for another reason.
  • Contraindication at the PDT, patient unable to lie over an hour.
  • Contraindication at fentanyl ( 50mcg Instanyl ) intra nasal :

oHypersensitivity to the active substance or to any of the excipients oUse in patients who have never received opioid treatment. oSevere respiratory depression or severe airway obstruction. oPrevious radiotherapy of the face. oRecurrent episodes of epistaxis oConcomitant administration of monoamine oxidase inhibitors, of potent CYP 3A4 inhibitors, of nasal decongestants, or other drugs (other than oxymetazoline ) administered by nasal way.

oSevere hepatic or renal insufficiency.

•Patients who have participated in a clinical trial in the previous 3 months

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02004600

Contacts
Contact: CHRISTINE CHIAVERINI, MD chiaverini.c@chu-nice.fr

Locations
France
Nice University Hospital Not yet recruiting
Nice, France, 06000
Principal Investigator: CHRISTINE CHIAVERINI, MD         
Saint Louis Hospital Not yet recruiting
Paris, France
Principal Investigator: Emmanuelle BOURRAT, MD         
Sponsors and Collaborators
Centre Hospitalier Universitaire de Nice
Investigators
Principal Investigator: Christine CHIAVERINI, MD Nice University Hospital
  More Information

No publications provided

Responsible Party: Centre Hospitalier Universitaire de Nice
ClinicalTrials.gov Identifier: NCT02004600     History of Changes
Other Study ID Numbers: 13-AOI-11
Study First Received: November 26, 2013
Last Updated: March 21, 2014
Health Authority: France: Agence Nationale de Sécurité du Médicament et des produits de santé
France: Committee for the Protection of Personnes

Additional relevant MeSH terms:
Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Collagen Diseases
Congenital Abnormalities
Connective Tissue Diseases
Genetic Diseases, Inborn
Skin Abnormalities
Skin Diseases
Skin Diseases, Genetic
Skin Diseases, Vesiculobullous

ClinicalTrials.gov processed this record on October 23, 2014