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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 16 December 2017
Main ID:  NCT02323789
Date of registration: 18/12/2014
Prospective Registration: Yes
Primary sponsor: King's College London
Public title: Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa ADSTEM
Scientific title: A Phase I/II Study Evaluating Allogeneic Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa
Date of first enrolment: June 2015
Target sample size: 10
Recruitment status: Active, not recruiting
URL:  https://clinicaltrials.gov/show/NCT02323789
Study type:  Interventional
Study design:  Intervention model: Single Group Assignment. Primary purpose: Other. Masking: None (Open Label).  
Phase:  Phase 1/Phase 2
Countries of recruitment
United Kingdom
Contacts
Name:     John McGrath, FRCP/FSci/MD
Address: 
Telephone:
Email:
Affiliation:  King's College London
Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All
Health Condition(s) or Problem(s) studied
Recessive Dystrophic Epidermolysis Bullosa
Intervention(s)
Drug: Mesenchymal stromal cells
Primary Outcome(s)
Lack of serious and severe adverse events (SAEs) related to the administration of the investigational medicinal product. [Time Frame: 12 months]
Secondary Outcome(s)
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]
Change in BEBSS and EBDASI scores [Time Frame: at Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline]
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.]
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]
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 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.]
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]
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.]
Secondary ID(s)
ADSTEM001
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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