Trial To Assess Efficacy Of A Chimeric Skin In Patients With Epidermolysys Bullosa

Study Overview
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Brief Summary
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A phase II, comparative, open label, prospective, multicentre clinical trial where each patient will undergo two procedures; implant of a patch of cultured chimeric skin (experimental therapy) in a half of the skin lesion and an occlusive non-adherent dressing (control) in the other half for 12 months of follow-up in two Spanish centres.

Detailed Description
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Reconstruction of interdigital commisures in patients with severe skin syndactyly using laminar grafts that will be uniformly and systematically taken with an electrical or compressed air dermatome on an aseptic area and with the same extent and depth in microns in all patients. Immediately after the surgical procedure, a patch of cultured chimeric skin (experimental therapy) will be implanted in a half of the skin defect of the patient, and an occlusive non - adherent dressing (control) will be implanted in the other half. Patients will initially be followed up every two days until epithelisation occurs (approximately 21 days after surgery) and at 3,8,and 12 months of follow-up.

Official Title
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A Comparative Open-Label Multicentre Clinical Trial To Assess The Efficacy And Safety Of A New Therapy With Cultured Chimeric Skin For The Treatment Of Skin Lesions In Patients With Epidermolysis Bullosa

Conditions
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EPIDERMOLYSIS BULLOSA

Intervention / Treatment
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* Drug: CX501
* Device: Occlusive non adherent dressing


Participation Criteria
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Eligibility Criteria
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Inclusion criteria: Patients of both sexes over 28 days of age. Patients with recessive dystrophic epidermolysis bullosa diagnosed by clinical and historical criteria with severe skin syndactyly in whom reconstructive surgery is indicated. Patients whose legal guardians have given written informed consent for participation in the study before any study procedure is performed. Patients aged ≥ 12 years who have given written informed consent for participation in the study before any study procedure is performed. Exclusion criteria: Women of childbearing age not using effective contraceptive methods (oral contraception) Pregnant or nursing women Documented or suspected hypersensitivity to any of the therapeutic agents included in the study, including anaesthetic drugs. Patients with a history of malignant tumor in the past 5 years Patients with a diagnosis of active tuberculosis at the time of recruitment Patients with prior positive markers for any of the following pathogens: hepatitis b and c, hiv-1 and hiv-2 Patients with a history of clinically relevant hepatic, gastrointestinal, haematological, pulmonary, or neurological disease no directly related to epidermolysis bullosa. Any other medical condition which, in the investigator´s judgment, might interfere with optimal participation in the study or involve a significant risk for the patient. Alcoholism, drug addiction, psychiatric disorders, or other factors present in legal guardians which,in the investigator´s judgment may complicate patient participation in the study.

Ages Eligible for Study
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Minimum Age: 28 Days

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: CX501<br>Cultured chimeric skin | Drug: CX501<br>* Cultured chimeric skin<br>|
| Active Comparator: Non adherent dressing<br>Occlusive non adherent dressing | Device: Occlusive non adherent dressing<br>* Application of an occlusive non adherent dressing in the skin donor site<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to epithelization |  | 21 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Fibroblast persistence |  | 3, 8 and 12 months | 
| Incidence of Adverse Events |  | 12 months |