Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa

• Status: Unknown status
• Trial end date: 2017-07-01
• Target enrollment: 0
• Participant gender: All

Summary

To assess whether intravenously administered third-party bone marrow-derived mesenchymal stromal cells (MSCs) are safe and have an impact on disease severity in RDEB

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: King's College London

Criteria

Inclusion Criteria:

1. Individuals with a diagnosis of RDEB confirmed by DNA analysis and skin immunofluorescence for partial or complete absence of type VII collagen.

2. Individuals ≥ 18 years and ≤ 65 years of age, both male and female

3. Individuals that have voluntarily signed and dated an informed consent form (ICF) prior to the first study intervention.

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).

3. Subjects with a known allergy to any of the constituents of the investigational product.

4. Subjects with a medical history or evidence of malignancy, including cutaneous squamous cell carcinoma.

5. 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.