Phase 1 Study CYP-001 for the Treatment of Acute Graft vs Host Disease
Research type
Research Study
Full title
An Open-Label Phase 1 Study to Investigate the Safety and Efficacy of CYP-001 for the Treatment of Adults With Steroid-Resistant Acute Graft Versus Host Disease
IRAS ID
207651
Contact name
Adrian Bloor
Contact email
Sponsor organisation
Cynata Therapeutics Limited
Eudract number
2016-000070-38
Duration of Study in the UK
1 years, 0 months, 1 days
Research summary
Allogeneic (donor) stem cell transplantation is a potentially curative treatment for patients with cancer and bone marrow failure although is associated with significant serious side effects. Acute graft versus host disease (aGvHD) is the result of the donor immune system, mediated by lymphocytes (inflammatory white blood cells), attacking the recipient tissues and is one of the most serious complications of allogeneic transplantation. This typically occurs within the first 3 months following transplant and affects up to two thirds of transplant recipients. Significant aGvHD is treated with corticosteroids although up to half of patients require subsequent lines therapy. Currently available treatments for steroid refractory aGvHD are often ineffective and there is no standard of care and the mortality for patients with resistant aGvHD is high.
Mesenchymal Stem Cells (MSC) are self-renewing cells that can be derived from a range of tissues including bone marrow and fat. They are able to inhibit a range of inflammatory cells including lymphocytes, natural killer cells and dendritic cells, on which basis they have been investigated as a possible treatment for aGvHD. Results of previous studies using MSC in aGvHD have been mixed although this is likely to be explained in part by the different techniques used for isolation of MSC which could impact on their efficacy.
This is a multi-centre, open label, dose escalation study to assess the safety, tolerability and efficacy of two infusions of MSC (CYP 001) produced using a novel technology to facilitate cell expansion without loss of efficacy. The study will be undertaken in adults steroid-resistant aGvHD. Participants will receive standard of care treatment throughout the study, according to local procedures. After screening, eligible participants will receive an intravenous (IV) infusion of CYP-001 on Day 0, and a second IV infusion of CYP 001 on Day 7. The first eight participants will be enrolled in Cohort A. After the eighth participant has completed their Day 28 assessments, safety data will be reviewed by an independent Data and Safety Monitoring Board (DSMB). The DSMB will recommend whether the study can proceed. If the study proceeds, an additional eight participants will be enrolled into a Cohort B at double the dose of Cohort A.
REC name
North East - York Research Ethics Committee
REC reference
16/NE/0316
Date of REC Opinion
10 Nov 2016
REC opinion
Further Information Favourable Opinion