NCT05643638

A Study of CYP-001 in Combination With Corticosteroids in Adults With High-risk aGvHD

A Multicenter, Randomized, Double-blind, Placebo-Controlled Phase II Study to Investigate the Efficacy and Safety of CYP-001 in Combination With Corticosteroids vs Corticosteroids Alone for the Treatment of High-Risk Acute Graft Versus Host Disease

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Cynata Therapeutics Limited · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study is a prospective randomized placebo-controlled phase 2 study to compare CYP-001 plus corticosteroids (CS) to placebo plus CS in allogeneic hematologic stem cell transplant recipients with HR-aGvHD. Severity of GvHD will be assessed at screening and throughout the study using Mount Sinai Acute GvHD International Consortium (MAGIC) guidelines. Eligible subjects will be randomized to receive either CYP-001 IV infusion on Days 0 and 4 or placebo on the same days. All subjects will receive ongoing CS therapy as appropriate per institutional guidelines. Subjects will have study visits up to Day 100 during the Primary Evaluation Period. During the Follow-Up Period, subjects will have study visits up to 24 months.

Conditions

• Graft Versus Host Disease, Acute

Interventions

Type	Name	Description
BIOLOGICAL	CYP-001: Cymerus induced pluripotent stem cell (iPSC)-derived mesenchymal stem cells (MSCs)	Cymerus MSCs are derived from iPSCs using the proprietary Cymerus platform technology.
BIOLOGICAL	Placebo	The placebo product is identical to CYP-001, except that it contains no active agent
DRUG	Corticosteroids	All enrolled subjects in this trial must receive corticosteroids at a minimum dose of oral prednisone 2 mg/kg/day (or methylprednisolone 1.6 mg/kg/day IV) as therapy for aGvHD for at least for 72 hours post enrollment.

Timeline

• Start date: 2024-03-04
• Primary completion: 2026-03-01
• Completion: 2027-12-01
• First posted: 2022-12-09
• Last updated: 2025-12-05

Locations

39 sites across 7 countries: United States, Australia, France, Italy, Lithuania, Spain, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study