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

NCT02004600 | Withdrawn

• 1 other identifier: 13-AOI-11
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Dystrophic Epidermolysis Bullosa

Outcome Measures

Primary Outcomes (1)

• Histological examination of a cutaneous biopsy

  Histological examination of a cutaneous biopsy at M2 on the epidermal dysplasia area treated with PDT session

  2 MONTHS AFTER ENROLLMENT

Secondary Outcomes (1)

• Tolerance of PDT

  every 10 minutes during PDT session

Study Arms (1)

patient receiving one PDT session

EXPERIMENTAL

patient receiving one photodynamic therapy (PDT) session

Procedure: Photodynamic therapy (PDT)

Interventions

Photodynamic therapy (PDT) PROCEDURE

1 session of Photodynamic therapy (PDT) on epidermis dysplasia

Also known as: 1 session of Photodynamic therapy (PDT)

patient receiving one PDT session

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The size of the lesion will be between 10 cm² and 1cm².
• Systematic Obtaining of the signed informed consent
• Patient affiliated to Social Security

You may not qualify if:

• 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

Sponsors & Collaborators

• Centre Hospitalier Universitaire de Nice: lead

Study Sites (2)

Nice University Hospital

Nice, 06000, France

Location

Saint Louis Hospital

Paris, France

Location

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Interventions

Photochemotherapy

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Intervention Hierarchy (Ancestors)

Combined Modality Therapy; Therapeutics; Drug Therapy; Phototherapy

Study Officials

• Christine CHIAVERINI, MD

  Nice University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 26, 2013
• First Posted: December 9, 2013
• Study Start: December 1, 2013
• Primary Completion: June 1, 2014
• Study Completion: June 1, 2015
• Last Updated: September 30, 2015

Record last verified: 2015-09

Locations

FR (2)