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Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa (ADSTEM)

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ClinicalTrials.gov Identifier: NCT02323789
Recruitment Status : Unknown
Verified March 2017 by King's College London.
Recruitment status was:  Active, not recruiting
First Posted : December 23, 2014
Last Update Posted : March 3, 2017
Sponsor:
Information provided by (Responsible Party):
King's College London

Tracking Information
First Submitted Date  ICMJE December 18, 2014
First Posted Date  ICMJE December 23, 2014
Last Update Posted Date March 3, 2017
Actual Study Start Date  ICMJE June 2015
Estimated Primary Completion Date July 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 18, 2014)
Lack of serious and severe adverse events (SAEs) related to the administration of the investigational medicinal product. [ Time Frame: 12 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 25, 2016)
  • 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 Measures  ICMJE
 (submitted: December 18, 2014)
  • 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.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa
Official Title  ICMJE 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 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  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Condition  ICMJE Recessive Dystrophic Epidermolysis Bullosa
Intervention  ICMJE 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
Study Arms  ICMJE Experimental: Intervention arm
10 patients with RDEB will be selected to receive the intervention - mesenchymal stromal cells.
Intervention: Drug: Mesenchymal stromal cells
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Actual Enrollment  ICMJE
 (submitted: December 18, 2014)
10
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE July 2017
Estimated Primary Completion Date July 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02323789
Other Study ID Numbers  ICMJE ADSTEM001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party King's College London
Study Sponsor  ICMJE King's College London
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: John McGrath, FRCP/FSci/MD King's College London
PRS Account King's College London
Verification Date March 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP