RecruitingPhase 1

CLN-049 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

A Phase 1, 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)

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This Phase 1 study tests CLN-049 in people for the first time. It is for adults with acute myeloid leukemia (AML), a blood cancer, or myelodysplastic syndrome (MDS), a disease that affects how blood cells are made. Their disease must have come back or not responded to treatment.

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)

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • You are 18 or older.
  • You agree in writing to join and can follow study rules, including repeated blood and bone marrow samples.
  • Tests confirm that your AML or MDS has come back or has not responded to treatment.
  • Available approved treatments did not work, stopped working, caused side effects you could not tolerate, or were declined.
  • Your white blood cell count is below 20,000 per microliter before the first dose. Hydroxyurea may be used to lower it.
  • You can care for yourself and are up and about for at least half of your waking hours.
  • Side effects from past study treatment are mild or gone, with exceptions for hair loss and low blood cell counts. Stable, long-term side effects may be allowed with approval.
  • Your kidney and liver blood tests meet study limits. Some exceptions apply for certain liver test results.

Who can't join

  • You have acute promyelocytic leukemia (APL), active leukemia in the brain or spinal cord, or disease that returned only outside the bone marrow.
  • You have had a donor organ transplant, a donor blood stem cell transplant within 6 months, or certain ongoing problems or immune-suppressing treatment after a transplant.
  • You have had certain cancer treatments, radiation, study drugs, blood cell growth drugs, immune-suppressing drugs, or live vaccines too recently. Some past treatments, including CAR-T cell therapy, are not allowed at any time.
  • You have another cancer needing treatment, certain diseases where the immune system attacks the body, a serious uncontrolled illness, certain heart rhythm changes, or another condition that could make treatment unsafe.
  • You have a recent uncontrolled infection, HIV, certain immune system disorders, or certain hepatitis infections. Some controlled hepatitis B or C infections are allowed. Active or suspected COVID-19, a pending test, or a past positive test without a later negative test also rules out joining.
  • You had certain severe side effects from immune-based treatment, certain severe allergic reactions, or a known past immune response against human antibody medicines.
  • You are pregnant or breastfeeding, or plan pregnancy, fathering a child, or sperm donation within 120 days after the last dose. Required birth control must be used during treatment and for 120 days afterward.
  • You have active alcohol or drug abuse, cannot make decisions for yourself, or are being held in custody against your will.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: 1. Aged ≥ 18 years of age. 2. Willing and able to give written informed consent and adhere to protocol requirements; written informed consent and any locally required authorization must be obtained from the patient prior to performing any protocol-related procedures, including screening evaluations, and serial samples of bone marrow and peripheral blood. 3. Patient has a confirmed diagnosis of recurrent or refractory AML or MDS. 4. Patient has received, and has progressed, recurred, or is intolerant of approved therapeutic options that are available, or declines treatment with these therapies. 5. White blood cell (WBC) count at the time of the first dose is \< 20,000/uL (hydroxyurea is permitted according to standard institutional practice). Following first dose, WBC should be checked prior to subsequent CLN-049 administration and if WBC \> 20,000/μL, CLN-049 treatment should be postponed (see Section 6.1 for further guidance). 6. Eastern Cooperative Oncology Group (ECOG) performance status is 0 to 2. 7. Toxicities related to prior study therapy should have resolved to Grade 1 or less according to criteria of NCI CTCAE v5.0, except for alopecia, lymphopenia, neutropenia, leukopenia, anemia, thrombocytopenia. Patients with chronic but stable toxicities may be allowed to enroll after agreement between the Investigator and Sponsor. 8. The patient's laboratory values meet the following criteria: 1. Creatinine clearance (CrCl) as calculated by the Cockcroft-Gault formula (Appendix 1) must be ≥ 60 mL/min; 2. Total bilirubin ≤ 1.5 × ULN. This does not apply for patients with confirmed Gilbert's Syndrome, hemolysis, or chronic blood transfusions, for whom total bilirubin must be less than 3.0 mg/dL with a conjugated bilirubin less than 0.5 mg/dL; 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. For patients with a history of CNS leukemia, a lumbar puncture should be performed during screening to exclude the presence of active CNS involvement. 3. Isolated extramedullary relapse 4. Prior organ allograft 5. Allogeneic hematopoietic transplantation within six months of treatment, or with clinical or laboratory evidence of GVHD, or requiring ongoing treatment with immune suppression within 2 months of the first dose of CLN-049. 6. Treatment with any of the following: 1. Radiation therapy (XRT) within 28 days of the first dose of CLN049, 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 (see separate guidelines for patients who are post allogeneic hematopoietic transplantation). The transient use of corticosteroids for transfusion premedication or the treatment of infusion or transfusion reactions will not be considered for this criterion. Topical corticosteroids and steroid eye drops are allowed, and will not exclude the patient from eligibility. 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, except for patients with vitiligo, psoriasis (in consultation with the Sponsor), resolved childhood asthma/atopy or autoimmune thyroid disorders on stable thyroid hormone supplementation. 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. Prophylactic use of systemic antiviral, antibacterial, or antifungal agents for treatment of chronic, controlled infection or as prophylaxis is permitted. 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). Note: patients with chronic HCV with undetectable viral load defined by sustained virologic response 24 weeks (SVR24) after completion of anti-hepatitis C treatment will be eligible. Patients with hepatitis B surface antigen \[HBsAg\] or hepatitis B core Ab with negative viral load will be eligible. 15. Active severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection including history of positive SARS-CoV-2 testing without subsequent documentation of negative test results, patients with results that are pending but not yet known, or patients with suspected active infection based on clinical features 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. Woman of child-bearing potential (WOCBP) who is pregnant or breast-feeding, plans to become pregnant within 120 days of last study drug administration, or declines to use an acceptable method to prevent pregnancy during study treatment and for 120 days after the last dose of study drug administration. 19. Male patient who plans to father a child or donate sperm within 120 days of last study drug administration, or who has a partner who is a WOCBP, and declines to use acceptable method to prevent pregnancy during study treatment and for 120 days after the last dose of study drug administration. 20. QT interval corrected for heart rate using Fridericia's formula (QTcF) of ≥ 480 milliseconds. 21. 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. 22. Known history of prior human anti-human antibody response. Patients will not be screened for human anti-human antibody prior to study participation. 23. Known active alcohol or drug abuse. 24. Patients who are incapacitated or involuntarily incarcerated.

Study sites (11)

AL

CA

FL

GA

MA

NC

NY

OH

TX