A Phase 1/2, Open-label, Preliminary Pharmacokinetics (PK) and Safety Study of CLN-049 (An Fms-like Tyrosine Kinase 3 [FLT3] x Cluster of Differentiation 3 [CD3] Bispecific T Cell Engager) in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) CLN-049

What's the purpose of this trial?

CLN-049-001 is a Phase 1, open-label, multicenter, first-in-human trial of CLN-049 in patients with Relapsed/Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

This trial is currently open and accepting patients.


What will happen during the trial?

 

You may be able to join this trial if you:

The following criteria is a partial list of reasons why patients may be eligible to participate in this clinical trial. Further evaluation with a medical professional is required.

Inclusion Criteria:

  1. Aged ≥ 18 years of age.
  2. Patient has a confirmed diagnosis of recurrent or refractory AML or MDS:

    Parts A, B, C:

    i. AML either de novo or secondary ii. MDS with at least 5% blasts that is relapsed after or refractory to hypomethylating agent (HMA) therapy iii. Patient has received, and has progressed, recurred, or is intolerant of approved therapeutic options that are available or declines treatment with these therapies

    Part D:

    i. Confirmed diagnosis of de novo or secondary R/R AML (includes patients with MDS/AML) ii. Patient has received no more than 2 prior lines of therapy iii. Patients must have exhausted, be ineligible for, be intolerant of, or otherwise not be considered appropriate candidates for available therapies considered suitable for the individual patient (including targeted therapies) iv. Patients must have a recent assessment of TP53 mutation status by an approved local laboratory.

    Part E:

    i. Patient has a confirmed diagnosis of de novo or secondary newly diagnosed AML (includes patients with MDS/AML) with no prior lines of AML-directed therapy ii. Patient must have evidence of a positive recent assessment of TP53 mutation status

  3. White blood cell (WBC) count at the time of the first dose is < 20,000/uL
  4. Eastern Cooperative Oncology Group (ECOG) performance status is 0 to 2.
  5. Toxicities related to prior study therapy should have resolved to Grade 1 or less according to criteria of NCI CTCAE v5.0
  6. The patient's laboratory values meet the following criteria:

    1. Creatinine clearance (CrCl) as calculated by the Cockcroft-Gault formula must be ≥ 45 mL/min;
    2. Total bilirubin ≤ 1.5 × ULN.
    3. AST and ALT ≤ 3.0 × ULN (unless attributed to leukemic involvement).

Exclusion Criteria:

  1. Diagnosis of acute promyelocytic leukemia (APL)
  2. Active central nervous system (CNS) leukemia.
  3. Isolated extramedullary relapse
  4. Prior organ allograft
  5. Prior history of allogeneic hematopoietic transplantation:

    1. In Parts A, B, and C: Prior history of allogeneic hematopoietic transplantation within six months of treatment or with clinical or laboratory evidence of GVHD, or requiring ongoing treatment with immune suppression
    2. In Parts D and E: Prior history of allogeneic hematopoietic transplantation within twelve months of treatment or with clinical or laboratory evidence of GVHD, or requiring ongoing treatment with immune suppression
  6. Treatment with any of the following:

    1. Radiation therapy (XRT) within 28 days of the first dose of CLN-049, or craniospinal XRT within 8 weeks of the first dose of CLN-049, or history of total body irradiation (TBI).
    2. Prior immunotherapy with checkpoint inhibitors ≤ 42 days prior to the first dose of CLN-049.
    3. Prior history of chimeric antigen receptor (CAR-T) cell therapy or other modified T cell therapy.
    4. Anti-leukemic therapy except hydroxyurea for cytoreduction, and intrathecal chemotherapy ≤ 14 days or 5 half-lives, whichever is shorter, prior to the first dose of CLN-049.
    5. Short-acting hematopoietic growth factors ≤ 7 days prior to the first dose of CLN-049
    6. Long-acting growth factors ≤ 14 days prior to the first dose of CLN-049.
    7. Systemic glucocorticoid therapy (except equivalent of < 10 mg prednisone daily) or other immune-suppressive drugs ≤ 14 days prior to the first dose of CLN-049
    8. Prior treatment with a FLT3-directed bispecific molecule, or a FLT3-targeted antibody.
  7. Currently participating/previously participated in an interventional study and received an investigational drug within 14 days (or five half-lives, whichever is longer) prior to the first dose of CLN-049.
  8. Patients with concomitant second malignancies requiring active treatment in the past 12 months, or if additional therapy is required or anticipated during study participation.
  9. Patients with any active autoimmune disease or a history of known or suspected autoimmune disease, or history of a syndrome that requires systemic corticosteroids or immunosuppressive medications
  10. A serious uncontrolled medical disorder that would impair the ability of the patient to receive protocol therapy or whose control may be jeopardized by the complications of this therapy.
  11. Any concurrent condition, therapy, or laboratory abnormality that, in the opinion of the investigator, might compromise patient safety or interfere with the evaluation of the safety of the drug.
  12. Active uncontrolled infection within seven days of first dose of CLN-049.
  13. Has a history of, or a positive test for Human Immunodeficiency Virus (HIV) 1/2 or primary immunodeficiency disease such as HIV.
  14. Known history of hepatitis B (with positive testing for either hepatitis B surface antigen [HBsAg] or hepatitis B core Ab), hepatitis C (HCV) infection (with positive testing for HCV antibody and/or HCV ribonucleic acid [RNA] in serum), or acute hepatitis A (with positive testing for hepatitis A IgM).
  15. Active severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection
  16. History of the following events in conjunction with prior treatment with immunotherapy: Grade 3 or greater neurotoxicity, ocular toxicity, pneumonitis, myocarditis, or colitis; liver dysfunction meeting the laboratory criteria for Hy's Law.
  17. Live virus vaccines within 28 days of the first dose of CLN-049, during treatment, and until the end of last dose of CLN-049.
  18. QT interval corrected for heart rate using Fridericia's formula (QTcF) of ≥ 480 milliseconds.
  19. Patient has history of drug-related anaphylactic reactions to any components of CLN-049. History of Grade 4 anaphylactic reaction to any bispecific molecule or monoclonal antibody therapy.
  20. Known history of prior human anti-human antibody response. Patients will not be screened for human anti-human antibody prior to study participation.

Additional Trial Information

Phase 1/2

Enrollment: 275 patients (estimated)

View More

Published Results

CLN-049 achieves 53% response rate with reduced cytokine release toxicity

November 04, 2026

Switching from a one-step to a two-step dosing ramp cut the cytokine release syndrome rate in Cullinan Therapeutics’ CLN-049 trial from 36% down to 26%, and eliminated Grade 3 CRS events entirely in patients who received the adjusted regimen. That safety signal matters because it came alongside the strongest efficacy numbers the program has posted: at the 12 µg/kg target dose, patients who started on a step-up of 1.5 µg/kg or lower hit a 53% overall response rate and a 26% complete remission composite rate, figures Cullinan will present in full at the 68th ASH Annual Meeting in December.

The dataset now covers 87 patients across 13 cohorts, with 80 assessed for efficacy. CLN-049 is a FLT3xCD3 bispecific T-cell engager, a mechanistic category distinct from CAR-T, targeting FLT3-expressing blasts in relapsed or refractory AML and MDS. Transaminase elevations had been a concern at 14% overall for Grade 3 or worse events, but the two-step regimen brought that down to 4% (1 of 27 patients), and the elevations recorded were asymptomatic and reversible. Neurological toxicity remained modest: immune effector cell-associated neurotoxicity occurred in 6% of all patients, with one Grade 3 event, and all events were self-limiting.

Trial Locations

All Trial Locations

View all clinical trial locations sorted by state.

Alabama

University of Alabama at Birmingham O'Neal Comprehensive Cancer Center

Birmingham, AL

Open and Accepting

Arizona

Mayo Clinic (Arizona)

Phoenix, AZ

Not Yet Accepting

California

City of Hope Comprehensive Cancer Center Beckman Research Institute

Duarte, CA

Not Yet Accepting

UCLA Medical Center of Hematology/Oncology

Los Angeles, CA

Open and Accepting

City of Hope

South Pasadena, CA

Not Yet Accepting

City of Hope

West Covina, CA

Not Yet Accepting

Colorado

University of Colorado Cancer Center Anschutz Cancer Pavilion

Aurora, CO

Open and Accepting

Florida

Mayo Clinic (Jacksonville)

Jacksonville, FL

Open and Accepting

Moffitt Cancer Center Magnolia Campus

Tampa, FL

Open and Accepting

Georgia

Winship Cancer Institute Emory University

Atlanta, GA

Open and Accepting

Illinois

City of Hope

Zion, IL

Not Yet Accepting

Minnesota

Mayo Clinic (Rochester)

Rochester, MN

Not Yet Accepting

New York

Roswell Park Cancer Institute

Buffalo, NY

Open and Accepting

Weill Cornell

New York, NY

Open and Accepting

North Carolina

Duke Cancer Center Duke University Medical Center

Durham, NC

Open and Accepting

Texas

MD Anderson Cancer Center The University of Texas

Houston, TX

Open and Accepting
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