Leading in MPNs Beyond Ruxolitinib in Combo With T-Regs (TREG108)

December 19, 2023 updated by: Cellenkos, Inc.

Phase Ib, Open-label Study of Add on Therapy With CK0804 in Participants With Myelofibrosis, With Suboptimal Response to Ruxolitinib

To assess the safety and tolerability of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

  1. Safety Run-in

    The study will employ a 3+3+3 design to assess the safety and tolerability of the treatment based on treatment-limiting toxicities (TLTs) occurring up to 1 Cycle (28 days) after the first infusion.

  2. Expansion

After a total of 9 participants completed 28 days and are evaluated for tolerability in the safety run-in phase, additional participants may be included in the expansion cohort in order to have approximately 24 evaluable myelofibrosis

Study Type

Interventional

Enrollment (Estimated)

24

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • California
      • Sacramento, California, United States, 95817
        • Recruiting
        • UC Davis Health
        • Contact:
        • Principal Investigator:
          • Mehrdad Abedi, MD
    • New York
      • Bronx, New York, United States, 10461
        • Recruiting
        • Montefiore Einstein Cancer Center
        • Contact:
          • Clinical Research Coordinator
        • Principal Investigator:
          • Swati Goel, MD
      • New York, New York, United States, 10032
        • Not yet recruiting
        • Columbia University
        • Contact:
        • Principal Investigator:
          • Mark Heaney, MD
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • The University of Texas MD Anderson Cancer Center
        • Contact:
        • Principal Investigator:
          • Lucia Masarova, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Ability to comprehend and willingness to sign a written informed consent form (ICF) for the study.
  2. Age above 18 years inclusive at the time of signing the ICF.
  3. Participants who fulfill the diagnostic criteria of myelofibrosis including primary myelofibrosis and myelofibrosis arising from polycythemia vera and essential thrombocythemia
  4. Life expectancy is greater than 6 months.
  5. Subject has been receiving ruxolitinib therapy, is unlikely to benefit from further ruxolitinib monotherapy in the opinion of the investigator; AND meeting the following criteria: receiving ruxolitinib >3 months prior to enrollment; AND stable dose for 8 weeks before starting therapy with CK0804

  6. Subject with evidence of evaluable residual burden of disease following ruxolitinib monotherapy treatment, consisting of:

    • presence of grade ≥2 anemia or thrombocytopenia or neutropenia, OR
    • presence of disease-related symptoms, as determined by a Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN SAF TSS) score of ≥10 points, OR
    • documented splenomegaly of at least 5 cm below the costal margin as measured by physical examination or splenomegaly as documented by ultrasound or MRI.

  7. Willingness to avoid pregnancy or fathering children based on the criteria below

    • Men must agree to take appropriate precautions to avoid fathering children (with at least 99% certainty) from screening through 90 days after the last study treatment dose and must refrain from donating sperm during this period. Permitted methods that are at least 99% effective in preventing should be communicated to the participants and their understanding confirmed.
    • Women of childbearing potential must have a negative serum pregnancy test at screening before the first dose (within 3 days of the first study treatment dose) and must agree to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from screening through the safety follow-up visit and must not donate oocytes during this period. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the participants and their understanding confirmed,
    • Women of nonchildbearing potential (ie, surgically sterile with a hysterectomy and/or bilateral oophorectomy OR ≥ 12 months of amenorrhea and at least 50 years of age) are eligible.
  8. ECOG performance status of 0 to 2

Exclusion Criteria:

  1. Any major surgery within 28 days before the first dose of study treatment.
  2. Undergone any prior allogenic or autologous stem cell transplantation or a candidate for such transplantation.
  3. Received chemotherapy, immunomodulatory drug therapy, immunosuppressive therapy, biological therapy, endocrine therapy, targeted therapy, antibody or hypomethylating agent to treat the participant's disease, with the exception of ruxolitinib, within 5 half-lives or 28 days (whichever is shorter) before the first dose of study treatment.
  4. Participant has received splenic irradiation within the past 6 months.
  5. Significant concurrent, uncontrolled medical condition or infections, which in the opinion of the principal investigator may interfere in the study participation.
  6. Inability or unlikeliness of the participant to comply with the dose schedule and study evaluations, in the opinion of the investigator.
  7. Women who are pregnant or breastfeeding.
  8. Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study treatment and attending required study visits; pose a significant risk to the participant; or interfere with interpretation of study data.

  9. Participants with laboratory values at screening as defined

    • Platelets < 50 × 10^9/L without the assistance of growth factors, thrombopoietic factors, or platelet transfusions
    • ANC < 0.5 × 10^9/L
    • ALT ≥ 2.5 × ULN
    • AST ≥ 2.5 × ULN
    • Direct Bilirubin > 2.0 × ULN
    • ALP ≥ 3 × ULN
    • Creatinine clearance < 50 mL/min according to Cockcroft-Gault formula.
  10. Unwillingness to be transfused with blood components including RBC and platelet transfusions.
  11. Inability of the participant (or parent, guardian, or legally authorized representative) to comprehend the ICF or unwillingness to sign the ICF.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Arm 1
CK0804 will be administered intravenously (IV) 100 million Treg Cells every 28 days up to 6 infusions.
Drug: CK0804
CK0804 is a cryopreserved, allogeneic T-regulatory cell product that is manipulated to traffic to the bone marrow.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To determine Treatment limiting toxicity (TLT) as defined below
Time Frame: 28 days
  • severe (grade 3 or 4) infusion-related toxicity within 24 hours (NCI-CTCAE V5.0) of exposure that does not resolve with standard of care treatment within 72 hours.
  • regimen related death within 28 days
28 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assessment of overall response rate (ORR) (measured as CR or PR) and its duration, using modified International Working Group-Myeloproliferative Neoplasm Research and Treatment (IWG-MRT) and European Leukemia Net (ELN) consensus report.
Time Frame: 6 months
To determine the efficacy of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib
6 months
Rate of anemia response as per modified IWG-MRT ELN response criteria.
Time Frame: 6 months
To determine the efficacy of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib
6 months
Rate of spleen response by imaging at and after 24 weeks as per IWG-MRT ELN response criteria
Time Frame: 6 months
To determine the efficacy of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib
6 months
Percentage of Participants who will Achieve Total Symptom Score Reduction Greater Than or Equal to (≥) 50% (TSS50) as Measured by Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
Time Frame: 6 months
To determine the efficacy of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cellenkos, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 27, 2022

Primary Completion (Estimated)

April 30, 2024

Study Completion (Estimated)

April 30, 2024

Study Registration Dates

First Submitted

June 10, 2022

First Submitted That Met QC Criteria

June 15, 2022

First Posted (Actual)

June 21, 2022

Study Record Updates

Last Update Posted (Estimated)

December 20, 2023

Last Update Submitted That Met QC Criteria

December 19, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CK0804.101.1

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

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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