Study to evaluate the use of allogeneic mesenchymal stromal cells for the treatment of skin disease in children with recessive dystrophic epidermolysis bullosa

Phase 1

Completed

• Conditions: Recessive dystrophic epidermolysis bullosa; Skin and Connective Tissue Diseases; Epidermolysis bullosa

• Registration Number: ISRCTN46615946
• Lead Sponsor: King's College London (UK)

Brief Summary

2015 results in: https://www.ncbi.nlm.nih.gov/pubmed/25905587

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2012-08-08
• Study Completion: 2014-12-11
• Last Update Posted: 25 March 2019

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

1. Subjects who have a diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) characterised by partial or complete collagen VII deficiency
2. Subjects who are = 12 months and = 17 years of age at the time of enrolment
3. Subjects whose responsible relative/guardian has voluntary 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

Exclusion Criteria

Current exclusion criteria as of 10/09/2013:
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 positive C7 ELISA and 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

Previous exclusion criteria:
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 positive serum antibodies to C7 confirmed by ELISA
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
• Study Design: Not specified