Ignite Creation Date:
2025-12-25 @ 1:07 AM
Ignite Modification Date:
2026-02-21 @ 1:20 AM
Study NCT ID:
NCT03773393
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-04-13
First Post:
2018-10-23
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF)
Sponsor:
Cellenkos, Inc.
Organization:
Study Overview
Official Title:
Phase I Trial to Evaluate the Safety and Feasibility of CK0801 in Treatment of Bone Marrow Failure Syndrome
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
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
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
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?: