- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02323789
Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa (ADSTEM)
A Phase I/II Study Evaluating Allogeneic Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa
Study Overview
Status
Conditions
Intervention / Treatment
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.
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
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London, United Kingdom, SE1 9RT
- Guys and St Thomas' hospital NHS Trust
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Individuals with a diagnosis of RDEB confirmed by DNA analysis and skin immunofluorescence for partial or complete absence of type VII collagen.
- Individuals ≥ 18 years and ≤ 65 years of age, both male and female
- Individuals that have voluntarily signed and dated an informed consent form (ICF) prior to the first study intervention.
Exclusion Criteria:
- Subjects who have had other investigational medicinal products within 90 days prior to screening or during the treatment phase.
- Subjects who have received immunotherapy including oral corticosteroids for more than 1 week (intranasal and topical preparations are permitted).
- Subjects with a known allergy to any of the constituents of the investigational product.
- Subjects with a medical history or evidence of malignancy, including cutaneous squamous cell carcinoma.
- 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 Plan
How is the study designed?
Design Details
- Primary Purpose: Other
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Intervention arm
10 patients with RDEB will be selected to receive the intervention - mesenchymal stromal cells.
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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 Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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Lack of serious and severe adverse events (SAEs) related to the administration of the investigational medicinal product.
Time Frame: 12 months
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12 months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
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Day 14, Day 28, Day 60, Day 100 and Month 6.
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Change in general markers of inflammation
Time Frame: Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
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Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
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Changes in specific markers of inflammation
Time Frame: Day 14, Day 28, Day 60 and Month 6 compared to baseline
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Day 14, Day 28, Day 60 and Month 6 compared to baseline
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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.
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Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12.
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Differences in quality of life data
Time Frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
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Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
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Change in BEBSS and EBDASI scores
Time Frame: at Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
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at Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
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Change in Pain scores
Time Frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
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Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
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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.
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Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline.
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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
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Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
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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
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Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
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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.
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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.
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between screening and Day 0, between Day 28 and Day 60, and between Month 6 and Month 12.
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Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: John McGrath, FRCP/FSci/MD, King's College London
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- ADSTEM001
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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