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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Bone Marrow Disease
• Intervention / Treatment: Biological: CK0801

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

• Study Type: Interventional
• Enrollment (Estimated): 18
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Santa Monica, California, United States, 90403
      • Sarcoma Oncology Research Center, Cancer Center of Southern California
  • Texas
    • Houston, Texas, United States, 77030
      • The University of Texas Md Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Subjects who fulfill the diagnostic criteria of bone marrow failure syndrome including: aplastic anemia, myelodysplastic syndrome, or myelofibrosis.
2. HLA matched (≥ 3/6) cord blood unit available for CK0801 generation.
3. Subjects age ≥ 18 years.
4. Bilirubin ≤ 2 x ULN and SGPT (ALT) ≤ 2 x ULN (unless Gilbert's syndrome is documented).
5. Calculated creatinine clearance of > 50mL/min using the Cockcroft-Gault equation.
6. Zubrod performance status ≤ 2.
7. 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).
8. Subject has agreed to abide by all protocol required procedures including study-related assessments, visits and long term follow up.
9. Subject is willing and able to provide written informed consent.

Exclusion Criteria:

1. Subject has received an investigational agent within 4 weeks prior to CK0801 infusion.
2. Subject has received radiation or chemotherapy within 21 days prior to CK0801 infusion.
3. Subject has received prior cord blood-derived T-regulatory therapy.
4. HIV seropositivity.
5. Subject has uncontrolled infection, not responding to appropriate antimicrobial agents after seven days of therapy. The Protocol PI is the final arbiter of eligibility.
6. 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
7. Subjects is pregnant or breastfeeding.
8. Bone marrow failure caused by stem cell transplantation.
9. Subjects who are unable to provide consent or who, in the opinion of the Investigator will be unlikely to fully comply with protocol requirements.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CK0801; 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.	Biological: CK0801; CK0801 (a Cord blood-derived T-regulatory cell product)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants with Severe Infusion Toxicity as Assessed by CTCAE v4.0	Number of Participants with Severe (Grade 3 or 4) Toxicity	24 hours post-intervention
Number of Participants with Regimen Related Death	Number of Participants with Regimen Related Death	30 days post-intervention
Number of Participants with Severe Cytokine Release Syndrome (CRS)	Number of Participants with Severe (Grade 3 or 4) CRS	30 days post-intervention

Collaborators and Investigators

• Sponsor: Cellenkos, Inc.
• Investigators: Principal Investigator: Tapan M Kadia, MD, The University of Texas Md Anderson Cancer Center

Study record dates

Study Major Dates

• Study Start (Actual): May 30, 2019
• Primary Completion (Estimated): May 25, 2024
• Study Completion (Estimated): May 30, 2024

Study Registration Dates

• First Submitted: October 23, 2018
• First Submitted That Met QC Criteria: December 10, 2018
• First Posted (Actual): December 12, 2018

Study Record Updates

• Last Update Posted (Estimated): February 7, 2024
• Last Update Submitted That Met QC Criteria: February 5, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Bone Marrow Failure Disorders; Pancytopenia
• Other Study ID Numbers: CK0801-101-1

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No