NCT02323789

Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa

A Phase I/II Study Evaluating Allogeneic Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: King's College London · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

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

Detailed description

This is a phase I/II clinical trial with a key objective of evaluating safety of third party bone MSCs intravenous infusions in 10 adults with the inherited severe skin fragility disorder, recessive dystrophic epidermolysis bullosa (RDEB). The main objectives of our study are to: (1) to assess the spectrum of clinical responses in adults with RDEB receiving intravenous MSCs; (2) to identify the best cohort of individuals to target for future trials and therapies; (3) to improve our understanding of in vivo and in vitro responsiveness to MSCs; (4) to identify candidate molecules germane to activating MSCs and making them clinically more potent, independently of the permissive conditions of the patient and (5) to assess its impact on reducing disease morbidity/severity in this population. This is a prospective, non-randomised, open label study. All study participants will receive two intravenous MSC infusions at baseline Day 0 and Day 14 and will be followed up for a 12 month period following the first infusion. Each subject will undergo an initial screening including physical examination, assessment of vital signs and disease severity assessment.

Conditions

• Recessive Dystrophic Epidermolysis Bullosa

Interventions

Type	Name	Description
DRUG	Mesenchymal stromal cells	TC-MSC: a cell product containing mesenchymal stromal cells. Mesenchymal stromal cells are adherent non-haematopoietic multipotent cells that are expanded from bone marrow from healthy donors, using platelet lysate as source of growth factor.

Timeline

• Start date: 2015-06-01
• Primary completion: 2017-07-01
• Completion: 2017-07-01
• First posted: 2014-12-23
• Last updated: 2017-03-03

Locations

1 site across 1 country: United Kingdom