NCT03773393

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF)

Phase I Trial to Evaluate the Safety and Feasibility of CK0801 in Treatment of Bone Marrow Failure Syndrome

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: Cellenkos, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical research study is to determine whether it is safe and practical to give CK0801 (a Cord blood-derived T-regulatory cell product) to patients with bone marrow failure syndrome. Researchers want to determine the highest possible dose that is safe to be given. Researchers also want to learn if CK0801 may improve the symptoms of bone marrow failure syndrome. Patients enrolled in this study will all have been diagnosed with treatment refractory bone marrow failure syndrome (which includes aplastic anemia, myelodysplastic syndrome, or myelofibrosis). Participants eligible to participate in this study are unable or unwilling to be treated with standard therapy or have failed standard therapy.

Detailed description

Primary Objective: To determine dose-limiting toxicity of CK0801 as defined as any of the events each start at the time of CK00801 infusion 1. Severe (grade 3 or 4) infusion toxicity within 24 hours (NCI-CTCAE V4.0) 2. Regimen related death within 30 days 3. Severe (grade 3 or 4) Cytokine Release Syndrome within 30 days Secondary Objective: 1. Preliminary assessment of disease-specific response 2. Duration of disease-specific response

Conditions

• Bone Marrow Disease

Interventions

Type	Name	Description
BIOLOGICAL	CK0801	CK0801 (a Cord blood-derived T-regulatory cell product)

Timeline

• Start date: 2019-05-30
• Primary completion: 2027-05-25
• Completion: 2027-05-30
• First posted: 2018-12-12
• Last updated: 2025-04-13

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study