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

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ClinicalTrials.gov Identifier: NCT03773393
Recruitment Status : Not yet recruiting
First Posted : December 12, 2018
Last Update Posted : December 13, 2018
Sponsor:
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:
CK0801 (Cord blood-derived T-regulatory cells) consists of ex vivo expanded T-regulatory cells with a flow cytometry phenotype of ≥ 60% CD4+CD25+ T-regulatory (Treg) cells and < 10% CD4-CD8+ T-cytotoxic/suppressor cells. The cellular starting material of CK0801 is a single unit of umbilical cord blood (CBU) from a normal, healthy unrelated donor. Because Treg cells are present only at low frequency in circulating blood or umbilical cord blood, production of clinically relevant Treg cell doses requires ex vivo enrichment and expansion of Treg cells with a CD4+CD25+ phenotype. In their natural state, T-regulatory (Treg) cells play an important role in maintaining immune homeostasis and limiting autoimmune responses by modulating both innate and adaptive immunity. Based on literature reports of animal studies showing induction of immune tolerance by Treg cells in autoimmune diseases, graft-versus-host disease, and solid organ transplant rejection, the administration of Treg cells in human clinical trials has become an attractive strategy to induce immune tolerance in patients in a variety of clinical settings.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Intervention Model: Single Group Assignment
Intervention Model Description:

Open-label, Phase I study designed to evaluate the safety of CK0801 in the treatment of bone marrow failure syndromes.

This study will use a Simon 3 + 3 design.

Three patients will be treated at dose level 1: 1x106/kg. If no dose limiting toxicity (DLT) is observed, then the dose will be escalated to dose level 2: 3x106/kg for the next cohort of 3 patients. If no DLT is observed, then the dose will be escalated to dose level 3: 1x107/kg.

If 1 DLT is observed at a dose level, then 3 additional patients will be treated at that level. If no additional DLTs, then that dose level will be defined as MTD.

If ≥ 2 DLTs at dose level 2 or 3, then prior dose level is defined as MTD. If ≥ 2 DLTs at dose level 1, then DSMB will review and evaluate for study continuation. MTD decided when 6 patients are treated at a dose level with < 2 DLTs.

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
Estimated Study Start Date : January 2019
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: CK0801, 50ml

All subjects will receive adoptive therapy with an infusion of unrelated cord blood-derived regulatory T cells: CK0801. Subjects will receive one 50mL intravenous dose of CK0801 (Treg cells) on study Day 0. A total of three cohorts will be evaluated.

Cohort dosing will be as follows:

Dose level 1 = 1x10e6/kg Treg cells per kg recipient ideal body weight (IBW); Dose level 2 = 3x10e6/kg Treg cells per kg recipient ideal body weight (IBW); Dose level 3 = 1x10e7/kg Treg cells per kg recipient ideal body weight (IBW).

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 ]
  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

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 to 70 years.
  4. Bilirubin ≤ 2 x ULN and SGPT (ALT) ≤ 2 x ULN (unless Gilbert's syndrome).
  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 ClinicalTrials.gov identifier (NCT number): NCT03773393


Contacts
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Contact: Erin Horne 800-217-8247 erin.horne@cellenkosinc.com
Contact: Tara Sadeghi 800-217-8247 tara.sadeghi@cellenkosinc.com

Locations
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United States, Texas
The University of Texas MD Anderson Cancer Center Not yet recruiting
Houston, Texas, United States, 77030
Contact: Tapan M Kadia, MD    713-563-3534    tkadia@mdanderson.org   
Principal Investigator: Tapan M Kadia, MD         
Sponsors and Collaborators
Cellenkos, Inc.
Investigators
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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.
ClinicalTrials.gov Identifier: NCT03773393     History of Changes
Other Study ID Numbers: CK0801-101-1
First Posted: December 12, 2018    Key Record Dates
Last Update Posted: December 13, 2018
Last Verified: December 2018
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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Pancytopenia
Anemia, Aplastic
Hemoglobinuria, Paroxysmal
Bone Marrow Diseases
Hematologic Diseases
Anemia
Anemia, Hemolytic
Myelodysplastic Syndromes