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

Bone Marrow Disease Clinical Trial

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

Status Active, not recruiting

Clinical Trial 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.

Clinical Trial 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 ;

Related Conditions & MeSH terms

• Bone Marrow Disease
• Bone Marrow Diseases
• Bone Marrow Failure Disorders
• Pancytopenia

• NCT number: NCT03773393
• Study type: Interventional
• Source: Cellenkos, Inc.
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: May 30, 2019
• Completion date: May 30, 2024