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

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

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.

Study Overview

• Status: Completed
• Conditions: EPIDERMOLYSIS BULLOSA
• Intervention / Treatment: Drug: CX501; Device: Occlusive non adherent dressing

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 2

Contacts and Locations

Study Locations

• Spain
  • Barcelona, Spain
    • Fundación Mir-Mir
  • Madrid, Spain
    • Hospital La Paz

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Time to epithelization	21 days

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Tigenix S.A.U.
• Investigators: Principal Investigator: Juan Carlos López, MD, Hospital La Paz

Study record dates

Study Major Dates

• Study Start: December 1, 2006
• Primary Completion (Actual): May 1, 2010
• Study Completion (Actual): May 1, 2010

Study Registration Dates

• First Submitted: September 29, 2009
• First Submitted That Met QC Criteria: September 29, 2009
• First Posted (Estimate): September 30, 2009

Study Record Updates

• Last Update Posted (Actual): August 1, 2019
• Last Update Submitted That Met QC Criteria: July 30, 2019
• Last Verified: July 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Skin Diseases, Genetic; Skin Diseases, Vesiculobullous; Skin Abnormalities; Epidermolysis Bullosa
• Other Study ID Numbers: CX501/TCEB