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

Primary Purpose

Bone Marrow Disease

Status

Active

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

CK0801

About this trial

This is an interventional treatment trial for Bone Marrow Disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Subjects who fulfill the diagnostic criteria of bone marrow failure syndrome including: aplastic anemia, myelodysplastic syndrome, or myelofibrosis.
HLA matched (≥ 3/6) cord blood unit available for CK0801 generation.
Subjects age ≥ 18 years.
Bilirubin ≤ 2 x ULN and SGPT (ALT) ≤ 2 x ULN (unless Gilbert's syndrome is documented).
Calculated creatinine clearance of > 50mL/min using the Cockcroft-Gault equation.
Zubrod performance status ≤ 2.
Female subjects of child bearing potential (FPCP) must have a negative urine or serum pregnancy test. NOTE: FPCP is defined as premenopausal and not surgically sterilized. FPCP must agree to use maximally effective birth control or to abstain from heterosexual activity throughout the study. Effective contraceptive methods include intra-uterine device, oral and/or injectable hormonal; contraception, or 2 adequate barrier methods (e.g., cervical cap with spermicide, diaphragm with spermicide).
Subject has agreed to abide by all protocol required procedures including study-related assessments, visits and long term follow up.
Subject is willing and able to provide written informed consent.

Exclusion Criteria:

Subject has received an investigational agent within 4 weeks prior to CK0801 infusion.
Subject has received radiation or chemotherapy within 21 days prior to CK0801 infusion.
Subject has received prior cord blood-derived T-regulatory therapy.
HIV seropositivity.
Subject has uncontrolled infection, not responding to appropriate antimicrobial agents after seven days of therapy. The Protocol PI is the final arbiter of eligibility.
Subjects with uncontrolled inter-current illness that in the opinion of the investigator would place the patient at greater risk of severe toxicity and/or impair the activity of CK0801
Subjects is pregnant or breastfeeding.
Bone marrow failure caused by stem cell transplantation.
Subjects who are unable to provide consent or who, in the opinion of the Investigator will be unlikely to fully comply with protocol requirements.

Sites / Locations

Sarcoma Oncology Research Center, Cancer Center of Southern California
The University of Texas MD Anderson Cancer Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

CK0801

Arm Description

All subjects will receive adoptive therapy with an infusion of unrelated cord blood-derived regulatory T cells: CK0801. Subjects will receive one intravenous dose of CK0801 (Treg cells) on study Day 0.

Outcomes

Primary Outcome Measures

Number of Participants with Severe Infusion Toxicity as Assessed by CTCAE v4.0

Number of Participants with Severe (Grade 3 or 4) Toxicity

Number of Participants with Regimen Related Death

Number of Participants with Severe Cytokine Release Syndrome (CRS)

Number of Participants with Severe (Grade 3 or 4) CRS

Secondary Outcome Measures

Full Information

NCT ID

NCT03773393

First Posted

October 23, 2018

Last Updated

August 28, 2023

Sponsor

Cellenkos, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT03773393

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

January 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

May 30, 2019 (Actual)

Primary Completion Date

January 25, 2024 (Anticipated)

Study Completion Date

January 30, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Cellenkos, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

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 Severe (grade 3 or 4) infusion toxicity within 24 hours (NCI-CTCAE V4.0) Regimen related death within 30 days Severe (grade 3 or 4) Cytokine Release Syndrome within 30 days Secondary Objective: Preliminary assessment of disease-specific response Duration of disease-specific response

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Bone Marrow Disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

18 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

CK0801

Arm Type

Experimental

Arm Description

Intervention Type

Biological

Intervention Name(s)

CK0801

Intervention Description

CK0801 (a Cord blood-derived T-regulatory cell product)

Primary Outcome Measure Information:

Title

Number of Participants with Severe Infusion Toxicity as Assessed by CTCAE v4.0

Description

Number of Participants with Severe (Grade 3 or 4) Toxicity

Time Frame

24 hours post-intervention

Title

Number of Participants with Regimen Related Death

Description

Number of Participants with Regimen Related Death

Time Frame

30 days post-intervention

Title

Number of Participants with Severe Cytokine Release Syndrome (CRS)

Description

Number of Participants with Severe (Grade 3 or 4) CRS

Time Frame

30 days post-intervention

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Subjects who fulfill the diagnostic criteria of bone marrow failure syndrome including: aplastic anemia, myelodysplastic syndrome, or myelofibrosis. HLA matched (≥ 3/6) cord blood unit available for CK0801 generation. Subjects age ≥ 18 years. Bilirubin ≤ 2 x ULN and SGPT (ALT) ≤ 2 x ULN (unless Gilbert's syndrome is documented). Calculated creatinine clearance of > 50mL/min using the Cockcroft-Gault equation. Zubrod performance status ≤ 2. Female subjects of child bearing potential (FPCP) must have a negative urine or serum pregnancy test. NOTE: FPCP is defined as premenopausal and not surgically sterilized. FPCP must agree to use maximally effective birth control or to abstain from heterosexual activity throughout the study. Effective contraceptive methods include intra-uterine device, oral and/or injectable hormonal; contraception, or 2 adequate barrier methods (e.g., cervical cap with spermicide, diaphragm with spermicide). Subject has agreed to abide by all protocol required procedures including study-related assessments, visits and long term follow up. Subject is willing and able to provide written informed consent. Exclusion Criteria: Subject has received an investigational agent within 4 weeks prior to CK0801 infusion. Subject has received radiation or chemotherapy within 21 days prior to CK0801 infusion. Subject has received prior cord blood-derived T-regulatory therapy. HIV seropositivity. Subject has uncontrolled infection, not responding to appropriate antimicrobial agents after seven days of therapy. The Protocol PI is the final arbiter of eligibility. Subjects with uncontrolled inter-current illness that in the opinion of the investigator would place the patient at greater risk of severe toxicity and/or impair the activity of CK0801 Subjects is pregnant or breastfeeding. Bone marrow failure caused by stem cell transplantation. Subjects who are unable to provide consent or who, in the opinion of the Investigator will be unlikely to fully comply with protocol requirements.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Tapan M Kadia, MD

Organizational Affiliation

The University of Texas MD Anderson Cancer Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

Sarcoma Oncology Research Center, Cancer Center of Southern California

City

Santa Monica

State/Province

California

ZIP/Postal Code

90403

Country

United States

Facility Name

The University of Texas MD Anderson Cancer Center

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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