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Clinical Trial Details — Status: Withdrawn

Administrative data

NCT number NCT02004600
Other study ID # 13-AOI-11
Secondary ID
Status Withdrawn
Phase N/A
First received November 26, 2013
Last updated September 29, 2015
Start date December 2013
Est. completion date June 2015

Study information

Verified date September 2015
Source Centre Hospitalier Universitaire de Nice
Contact n/a
Is FDA regulated No
Health authority France: Agence Nationale de Sécurité du Médicament et des produits de santéFrance: Committee for the Protection of Personnes
Study type Interventional

Clinical Trial 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.


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).


Recruitment information / eligibility

Status Withdrawn
Enrollment 0
Est. completion date June 2015
Est. primary completion date June 2014
Accepts healthy volunteers No
Gender Both
Age group 18 Years to 65 Years
Eligibility 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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

Procedure:
Photodynamic therapy (PDT)
1 session of Photodynamic therapy (PDT) on epidermis dysplasia

Locations

Country Name City State
France Nice University Hospital Nice
France Saint Louis Hospital Paris

Sponsors (1)

Lead Sponsor Collaborator
Centre Hospitalier Universitaire de Nice

Country where clinical trial is conducted

France, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 No
Secondary Tolerance of PDT Tolerance of PDT : evaluation of pain every 10 minutes during PDT session every 10 minutes during PDT session No
See also
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Recruiting NCT04917887 - Long-Term Follow-up Protocol
Completed NCT04917874 - A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa Phase 3
Terminated NCT03578029 - Evaluation of the Safety and Efficacy Study of RGN-137 Topical Gel for Junctional and Dystrophic Epidermolysis Bullosa Phase 2
Completed NCT03472287 - To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB) Phase 1
Recruiting NCT05157958 - Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa Phase 2
Withdrawn NCT01528306 - A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa Phase 2
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Completed NCT03183934 - A Follow-up Study to Evaluate the Efficacy and Safety of ALLO-ASC-DFU in ALLO-ASC-EB-101 Clinical Trial
Completed NCT02579369 - Study to Evaluate the Safety of ALLO-ASC-DFU in the Subjects With Dystrophic Epidermolysis Bullosa Phase 1/Phase 2
Completed NCT04491604 - Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB Phase 3
Not yet recruiting NCT05529134 - Study of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene Phase 1/Phase 2
Not yet recruiting NCT04173650 - MSC EVs in Dystrophic Epidermolysis Bullosa Phase 1/Phase 2