Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa

NCT ID: NCT02323789

Last Updated: 2017-03-03

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-06-30
• Study Completion Date: 2017-07-31

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.

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: OTHER
• Blinding Strategy: NONE

Study Groups

Intervention arm

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

Group Type: EXPERIMENTAL

Mesenchymal stromal cells

Intervention Type: 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.

Interventions

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.

Intervention Type: DRUG

Other Intervention Names

TC-MSC

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

King's College London

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

John McGrath, FRCP/FSci/MD

Role: PRINCIPAL_INVESTIGATOR

King's College London

Locations

Guys and St Thomas' hospital NHS Trust

London, , United Kingdom

Countries

United Kingdom

Other Identifiers

ADSTEM001

Identifier Type: -

Identifier Source: org_study_id