The purpose of this study is to assess the safety, tolerability and efficacy of two infusions of CYP-001 in adults with steroid-resistant GvHD.
This is a multi-centre, open label, dose escalation study to assess the safety, tolerability and efficacy of two infusions of CYP-001, in adults who have steroid-resistant GvHD. Participants will receive standard of care treatment throughout the study, according to local procedures. The first eight participants will be enrolled in Cohort A and receive a CYP-001 dose of 1 million cells per kg, up to a maximum dose of 100 million cells, on Day 0 and Day 7. Subject to a safety review of data from Cohort A, an additional eight participants will be enrolled into Cohort B and receive a CYP-001 dose of 2 million cells/kg, up to a maximum dose of 200 million cells, on Day 0 and Day 7. The primary evaluation period concludes for each participant 100 days after the first dose of CYP-001. Participants will have study visits on Days 0, 3, 7, 14, 21, 28, 60 and 100. Subsequently, participants will enter a long term follow-up period, which concludes 2 years after the first dose of CYP-001.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
16
The active agent in CYP-001 is allogeneic mesenchymoangioblast-derived mesenchymal stem cells (MCA-derived MSCs), which are produced using the proprietary Cymerus™ platform technology. Cymerus™ refers to the process of generating cell-based products from intermediate cells, MCAs, which in turn are derived from induced pluripotent stem cells or iPSCs. The iPSCs used in the Cymerus™ process were derived from blood donated by a fully-consented healthy adult donor, and were reprogrammed using a transgene-free, viral-free and feeder-free technique.
Sydney Local Health District
Sydney, New South Wales, Australia
Royal Adelaide Hospital
Adelaide, South Australia, Australia
NHS Foundation Trust
Bristol, United Kingdom
NHS Trust
Leeds, United Kingdom
Incidence and severity of treatment emergent adverse events [safety and tolerability]
Safety
Time frame: 28 days
Incidence and severity of serious adverse events deemed possibly related to CYP-001 [safety and tolerability]
Safety
Time frame: 100 days
Complete Response by Day 28
Proportion of participants who show a Complete Response (absence of any signs or symptoms of GvHD) by Day 28
Time frame: 28 days
Partial Response by Day 28
Proportion of participants who show a Partial Response (improvement in the severity of GvHD by at least one grade compared to baseline) by Day 28
Time frame: 28 days
Overall Survival at Day 28
Proportion of participants who survive until Day 28
Time frame: 28 days
Complete Response by Day 100
Proportion of participants who show a Complete Response by Day 100
Time frame: 100 days
Partial Response by Day 100
Proportion of participants who show a Partial Response by Day 100
Time frame: 100 days
Overall Survival at Day 100
Proportion of participants who survive until Day 100
Time frame: 100 days
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
NHS Foundation Trust
Liverpool, United Kingdom
NHS Foundation Trust
Manchester, United Kingdom
NHS Foundation Trust
Nottingham, United Kingdom