Dystrophic Epidermolysis BullosaNCT02004600
Pilot Study Evaluating the Efficiency and the Tolerance of the PDT in the Treatment of Epidermal Dysplasia for Patients Affected by Hereditary DEB
A study testing Photodynamic therapy (PDT) in people with Dystrophic Epidermolysis Bullosa, run by Centre Hospitalier Universitaire de Nice at 2 sites in France.
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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Dystrophic Epidermolysis Bullosa
- What is being tested?
- Photodynamic therapy (PDT)
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 2 sitesFrance
- Study dates
- Dec 2013to Jun 2015
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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 :
Show all 9Show fewer
- 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
Trial locations and contacts
2 sites in 1 country.
About this study
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
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.
Read the 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).
- Conditions
- Dystrophic Epidermolysis Bullosa
- Being tested
- Photodynamic therapy (PDT)
- Medical subject headings
- Epidermolysis Bullosa Dystrophica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Photodynamic therapy (PDT)Procedure or surgery
Also called 1 session of Photodynamic therapy (PDT)
1 session of Photodynamic therapy (PDT) on epidermis dysplasia
| Study group | Role | What they receive |
|---|---|---|
| patient receiving one PDT session | Experimental | patient receiving one photodynamic therapy (PDT) session Receives: Procedure: Photodynamic therapy (PDT) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Histological examination of a cutaneous biopsy
Histological examination of a cutaneous biopsy at M2 on the epidermal dysplasia area treated with PDT session
Measured: 2 MONTHS AFTER ENROLLMENT
Secondary outcomes (1)
Tolerance of PDT
Tolerance of PDT : evaluation of pain every 10 minutes during PDT session
Measured: every 10 minutes during PDT session
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2013 (estimated)
- Study startDec 2013
- Primary completionJun 2014 (estimated)
- Study completionJun 2015 (estimated)
Why it stopped: health statement of pre-screenined patients were too bad for being enrolled in the study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dystrophic Epidermolysis BullosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Nice
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Christine CHIAVERINI, MD, Principal investigator, Nice University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02004600
- Sponsor's protocol ID
- 13-AOI-11
- Status last verified
- Sep 2015
- First submitted
- 26 Nov 2013
- Last updated
- 30 Sep 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02004600
- Acronym / sponsor ID
- 13-AOI-11
- Conditions
- Dystrophic Epidermolysis Bullosa
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: health statement of pre-screenined patients were too bad for being enrolled in the study
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Centre Hospitalier Universitaire de NiceUniversity, hospital or other
- Study start
- Dec 2013
- Primary completion
- Jun 2014 (estimated)
- Study completion
- Jun 2015 (estimated)
- First posted
- 9 Dec 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| patient receiving one PDT session | Experimental | Procedure: Photodynamic therapy (PDT)patient receiving one photodynamic therapy (PDT) session |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Photodynamic therapy (PDT)Also: 1 session of Photodynamic therapy (PDT)1 session of Photodynamic therapy (PDT) on epidermis dysplasia | Procedure or surgery | patient receiving one PDT session |
Primary outcome
Histological examination of a cutaneous biopsy
Time frame: 2 MONTHS AFTER ENROLLMENT
Histological examination of a cutaneous biopsy at M2 on the epidermal dysplasia area treated with PDT session
1 secondary outcome
Tolerance of PDT
Time frame: every 10 minutes during PDT session
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2015.
Related trials for Dystrophic Epidermolysis Bullosa
RecruitingPhase 2
A Phase 2 Study of TCP-25 Gel in Patients With Epidermolysis Bullosa, STEP-study
Epidermolysis Bullosa (EB), Dystrophic Epidermolysis Bullosa
RecruitingPhase 2
A Study to Evaluate ENERGI-F703EB Cream in Epidermolysis Bullosa
Epidermolysis Bullosa (EB), Junctional Epidermolysis Bullosa
RecruitingObservational study
A Study to Assess the Incidence of Skin Cancers in Patients With Epidermolysis Bullosa Receiving Filsuvez
Epidermolysis Bullosa, Dystrophic, Epidermolysis Bullosa, Junctional
Not yet recruitingPhase 4
Phase 4 Master Protocol for Patients Prescribed Prademagene Zamikeracel for the Treatment of Wounds
Epidermolysis Bullosa (EB), Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Enrolling by invitationObservational study
A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB
RDEB
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christine CHIAVERINI, MD principal investigator
Nice University Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Photodynamic therapy (PDT) in people with Dystrophic Epidermolysis Bullosa, run by Centre Hospitalier Universitaire de Nice at 2 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 30 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Histological examination of a cutaneous biopsy (time frame: 2 MONTHS AFTER ENROLLMENT). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Sep 2015.