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In a major world first, Australian stem cell company Cynata Therapeutics (ASX:CYP) announced it received advice from the UK Medicines and Healthcare products Regulatory Agency (MHRA) that its Phase I clinical trial application has been approved.
The clinical trial has been named “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” (NCT02923375).
It will be the world’s first clinical trial involving a therapeutic product derived from allogeneic (unrelated to the patient) induced pluripotent stem cells (iPSCs).
Highlights of Cynata Therapeutics’ Upcoming Phase 1 Clinical Trial (Protocol Number: CYP-GvHD-P1-01)
Participants for Cynata’s upcoming Phase I clinical trial must be adults who have undergone an allogeneic haematopoietic stem cell transplant (HSCT) to treat a haematological disorder and subsequently been diagnosed with steroid-resistant Grade II-IV GvHD.
The first eight participants will be enrolled in Cohort A and receive two infusions of CYP-001 at a dose of 1 million cells per kilogram of body weight (cells/kg), up to a maximum dose of 100 million cells.
There will be one week between the two CYP-001 infusions in each patient.
The next eight participants will be enrolled into Cohort B and receive two infusions of CYP 001 at a dose of 2 million cells/kg, up to a maximum dose of 200 million cells.
The primary objective of the trial is to assess safety and tolerability, while the secondary objective is to evaluate the efficacy of two infusions of CYP-001 in adults with steroid-resistant GvHD.
Efficacy will be assessed on the basis of response to treatment (as determined by change in GvHD Grade) and overall survival at 28 and 100 days after the administration of the first dose.
Participants will also be followed up for up to two years under a separate non-interventional study protocol.
Importantly, Cynata’s Cymerus™ mesenchymal stem cell (MSC) technology is a second generation process that does not rely on multiple donors or massive expansion of the end product, and therefore, is a commercially viable solution to the manufacture of consistent, robust MSC therapeutic products.