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

NCT00987142 | Completed Phase 2

• 1 other identifier: CX501/TCEB
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

EPIDERMOLYSIS BULLOSA

Outcome Measures

Primary Outcomes (1)

• Time to epithelization

  21 days

Secondary Outcomes (2)

• Fibroblast persistence

  3, 8 and 12 months

• Incidence of Adverse Events

  12 months

Study Arms (2)

CX501

EXPERIMENTAL

Cultured chimeric skin

Drug: CX501

Non adherent dressing

ACTIVE COMPARATOR

Occlusive non adherent dressing

Device: Occlusive non adherent dressing

Interventions

CX501 DRUG

Cultured chimeric skin

CX501

Occlusive non adherent dressing DEVICE

Application of an occlusive non adherent dressing in the skin donor site

Non adherent dressing

Eligibility Criteria

• Age: 28 Days+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Tigenix S.A.U.: lead

Study Sites (2)

Fundación Mir-Mir

Barcelona, Spain

Location

Hospital La Paz

Madrid, Spain

Location

MeSH Terms

Conditions

Epidermolysis Bullosa

Condition Hierarchy (Ancestors)

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

Study Officials

• Juan Carlos López, MD

  Hospital La Paz

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 29, 2009
• First Posted: September 30, 2009
• Study Start: December 1, 2006
• Primary Completion: May 1, 2010
• Study Completion: May 1, 2010
• Last Updated: August 1, 2019

Record last verified: 2019-07

Locations

ES (2)