Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa

NCT02323789 | Unknown Phase 1 DMC

• 1 other identifier: ADSTEM001
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Outcome Measures

Primary Outcomes (1)

• Lack of serious and severe adverse events (SAEs) related to the administration of the investigational medicinal product.

  12 months

Secondary Outcomes (11)

• Presence of new type VII collagen at the dermal-epidermal junction post treatment.

  Day 14, Day 28, Day 60, Day 100 and Month 6.

• Change in general markers of inflammation

  Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline

• Changes in specific markers of inflammation

  Day 14, Day 28, Day 60 and Month 6 compared to baseline

• 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.

• Differences in quality of life data

  Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline

Study Arms (1)

Intervention arm

EXPERIMENTAL

10 patients with RDEB will be selected to receive the intervention - mesenchymal stromal cells.

Drug: Mesenchymal stromal cells

Interventions

Mesenchymal stromal cells DRUG

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.

Also known as: TC-MSC

Intervention arm

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• King's College London: lead

Study Sites (1)

Guys and St Thomas' hospital NHS Trust

London, SE1 9RT, United Kingdom

Location

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Study Officials

• John McGrath, FRCP/FSci/MD

  King's College London

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 18, 2014
• First Posted: December 23, 2014
• Study Start: June 1, 2015
• Primary Completion: July 1, 2017
• Study Completion: July 1, 2017
• Last Updated: March 3, 2017

Record last verified: 2017-03

Locations

GB (1)