Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa

Recessive Dystrophic Epidermolysis Bullosa Clinical Trial

— ADSTEM  

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02323789
• Other study ID #: ADSTEM001
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• First received: December 18, 2014
• Last updated: March 1, 2017
• Start date: June 2015
• Est. completion date: July 2017

Study information

• Verified date: March 2017
• Source: King's College London
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 10
• Est. completion date: July 2017
• Est. primary completion date: July 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica
• Recessive Dystrophic Epidermolysis Bullosa

Intervention

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.

Locations

Country	Name	City	State
United Kingdom	Guys and St Thomas' hospital NHS Trust	London

Sponsors (1)

Lead Sponsor	Collaborator
King's College London

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Lack of serious and severe adverse events (SAEs) related to the administration of the investigational medicinal product.		12 months
Secondary	Presence of new type VII collagen at the dermal-epidermal junction post treatment.		Day 14, Day 28, Day 60, Day 100 and Month 6.
Secondary	Change in general markers of inflammation		Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Secondary	Changes in specific markers of inflammation		Day 14, Day 28, Day 60 and Month 6 compared to baseline
Secondary	Change in the clinical changes in the skin assessed with clinical photographs		Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12.
Secondary	Differences in quality of life data		Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Secondary	Change in BEBSS and EBDASI scores		at Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Secondary	Change in Pain scores		Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Secondary	Change in pruritus score using the Leuven Itch Scale (LIS)		Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline.
Secondary	Quantification of total blister numbers over the entire body surface area		Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Secondary	10. Increase in the skin strength measured by time to blister formation after negative pressure skin suction test		Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Secondary	Qualitative analyses based on a series of interview questions	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.	between screening and Day 0, between Day 28 and Day 60, and between Month 6 and Month 12.