Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa (ADSTEM)
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|ClinicalTrials.gov Identifier: NCT02323789|
Recruitment Status : Active, not recruiting
First Posted : December 23, 2014
Last Update Posted : March 3, 2017
|Condition or disease||Intervention/treatment||Phase|
|Recessive Dystrophic Epidermolysis Bullosa||Drug: Mesenchymal stromal cells||Phase 1 Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I/II Study Evaluating Allogeneic Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa|
|Actual Study Start Date :||June 2015|
|Estimated Primary Completion Date :||July 2017|
|Estimated Study Completion Date :||July 2017|
Experimental: Intervention arm
10 patients with RDEB will be selected to receive the intervention - mesenchymal stromal cells.
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.
Other Name: TC-MSC
- Lack of serious and severe adverse events (SAEs) related to the administration of the investigational medicinal product. [ Time Frame: 12 months ]
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02323789
|Guys and St Thomas' hospital NHS Trust|
|London, United Kingdom, SE1 9RT|
|Principal Investigator:||John McGrath, FRCP/FSci/MD||King's College London|