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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03773393
Recruitment Status : Active, not recruiting
First Posted : December 12, 2018
Last Update Posted : July 20, 2022
Information provided by (Responsible Party):
Cellenkos, Inc.

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.

Condition or disease Intervention/treatment Phase
Bone Marrow Disease Biological: CK0801 Phase 1

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Trial to Evaluate the Safety and Feasibility of CK0801 in Treatment of Bone Marrow Failure Syndrome
Actual Study Start Date : May 30, 2019
Estimated Primary Completion Date : August 25, 2022
Estimated Study Completion Date : August 30, 2022

Resource links provided by the National Library of Medicine

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)

Primary Outcome Measures :
  1. Number of Participants with Severe Infusion Toxicity as Assessed by CTCAE v4.0 [ Time Frame: 24 hours post-intervention ]
    Number of Participants with Severe (Grade 3 or 4) Toxicity

  2. Number of Participants with Regimen Related Death [ Time Frame: 30 days post-intervention ]
    Number of Participants with Regimen Related Death

  3. Number of Participants with Severe Cytokine Release Syndrome (CRS) [ Time Frame: 30 days post-intervention ]
    Number of Participants with Severe (Grade 3 or 4) CRS

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03773393

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United States, California
Sarcoma Oncology Research Center, Cancer Center of Southern California
Santa Monica, California, United States, 90403
United States, Texas
The University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
Cellenkos, Inc.
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Principal Investigator: Tapan M Kadia, MD The University of Texas MD Anderson Cancer Center
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Responsible Party: Cellenkos, Inc. Identifier: NCT03773393    
Other Study ID Numbers: CK0801-101-1
First Posted: December 12, 2018    Key Record Dates
Last Update Posted: July 20, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Bone Marrow Failure Disorders
Bone Marrow Diseases
Hematologic Diseases