A prospective phase I/II study to evaluate allogeneic mesenchymal stromal cells for the treatment of skin disease in children with recessive dystrophic epidermolysis bullosa.

Phase 1

• Conditions: Recessive Dystrophic Epidermolysis Bullosa; MedDRA version: 17.0Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

• Registration Number: EUCTR2012-001394-87-GB
• Lead Sponsor: King's College London

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-08-22
• Last Update Posted: 4 December 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

1)Subjects who have a diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) characterised by partial or complete C7 deficiency.
2)Subjects who are = 12 months and = 17 years of age at the time of enrolment.
3)Subjects whose responsible parent/guardian has voluntarily signed and dated an Informed Consent Form (ICF) prior to the first study intervention. Whenever the minor child is able to give consent, the minor’s assent will be obtained in addition to the signed consent of the minor’s legal guardian.

Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Subjects will be excluded from the study if ANY of the following conditions exist:
1)Subjects who have had other investigational medicinal products within 90 days prior to screening or during the treatment phase.
2)Subjects who have received immunotherapy including oral corticosteroids for more than 1 week (intranasal and topical preparations are permitted) or chemotherapy within 60 days of enrolment into this study.
3)Subjects with a known allergy to any of the constituents of the investigational product.
4)Subjects with signs of active infection.
5)Subjects with a medical history or evidence of malignancy, including cutaneous squamous cell carcinoma.
6)Subjects with both a) positive C7 ELISA and b) a positive indirect immunofluorescence (IIF) with binding to the base of salt split skin.
7)Subjects who are pregnant or of child-bearing potential who are not abstinent or practicing an acceptable means of contraception, as determined by the Investigator, for the duration of the treatment phase.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified