Clinical Trial: Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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

Brief 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 Summary:

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.


Sponsor: King's College London

Current Primary Outcome: Lack of serious and severe adverse events (SAEs) related to the administration of the investigational medicinal product. [ Time Frame: 12 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Presence of new type VII collagen at the dermal-epidermal junction post treatment. [ Time Frame: Day 14, Day 28, Day 60, Day 100 and Month 6. ]
  • Change in general markers of inflammation [ Time Frame: Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline ]
  • Changes in specific markers of inflammation [ Time Frame: Day 14, Day 28, Day 60 and Month 6 compared to baseline ]
  • Change in the clinical changes in the skin assessed with clinical photographs [ Time Frame: Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12. ]
  • Differences in quality of life data [ Time Frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline ]
  • Change in BEBSS and EBDASI scores [ Time Frame: at Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline ]
  • Change in Pain scores [ Time Frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline ]
  • Change in pruritus score using the Leuven Itch Scale (LIS) [ Time Frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline. ]
  • Quantification of total blister numbers over the entire body surface area [ Time Frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline ]
  • 10. Increase in the skin strength measured by time to blister formation after negative pressure skin suction test [ Time Frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline ]
  • Qualitative analyses based on a series of interview questions [ Time Frame: between screening and Day 0, between Day 28 and Day 60, and between Month 6 and Month 12. ]
    to reveal objective data on quality of sleep, skin healing time, amount of dressings used, improvement in oral diet, improvement in energy levels, mood, quality of family life/relationships.


Original Secondary Outcome:

  • Presence of new type VII collagen at the dermal-epidermal junction post treatment. [ Time Frame: Day 14, Day 28, Day 60, Day 100 and Month 6. ]
  • Improvement in general markers of inflammation [ Time Frame: Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline ]
  • Changes in specific markers of inflammation [ Time Frame: Day 14, Day 28, Day 60 and Month 6 compared to baseline ]
  • Improvement in the clinical changes in the skin assessed with clinical photographs [ Time Frame: Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12. ]
  • Differences in quality of life data [ Time Frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline ]
  • Improvement in BEBSS and EBDASI scores [ Time Frame: at Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline ]
  • Improvement in Pain scores [ Time Frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline ]
  • Improvement in pruritus score using the Leuven Itch Scale (LIS) [ Time Frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline. ]
  • Quantification of total blister numbers over the entire body surface area [ Time Frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline ]
  • 10. Increase in the skin strength measured by time to blister formation after negative pressure skin suction test [ Time Frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline ]
  • Qualitative analyses based on a series of interview questions [ Time Frame: between screening and Day 0, between Day 28 and Day 60, and between Month 6 and Month 12. ]
    to reveal objective data on quality of sleep, skin healing time, amount of dressings used, improvement in oral diet, improvement in energy levels, mood, quality of family life/relationships.


Information By: King's College London

Dates:
Date Received: December 18, 2014
Date Started: June 2015
Date Completion: July 2017
Last Updated: March 1, 2017
Last Verified: March 2017