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

NCT05143996 | Recruiting Phase 1 FDA Drug

• 2 other identifiers: CLN-049-0012020-005195-35
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 11

Brief Summary

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)

Conditions

Relapsed/Refractory Acute Myeloid Leukemia (AML); Myelodysplastic Syndrome (MDS)

Outcome Measures

Primary Outcomes (5)

• Number of treatment emergent events (TEAEs)

  TEAEs will be defined as adverse events that are reported for the first time following study drug administration for worsening of a pre-existing event after the first dose

  28 days

• Cmax of CLN-049

  Maximum drug concentration

  28 Days

• Ctrough of CLN-049

  The observed plasma concentration just prior to the beginning of, or at the end of a dosing interval

  28 Days

• Tmax of CLN-049

  Time to Cmax

  28 Days

• T1/2 of CLN-049

  28 Days

  Up to 28 days

Secondary Outcomes (1)

• Immunogenicity of CLN-049

  28 days

Study Arms (3)

Part A - Single ascending dose (SAD) design of IV administered CLN-049

EXPERIMENTAL

Patients with relapsed/refractory AML or MDS will receive CLN-049 via IV administration

Drug: CLN-049

Part B - Multiple ascending dose (MAD) design of IV administered CLN-049

EXPERIMENTAL

Patients with relapsed/refractory AML or MDS will receive CLN-049 via IV administration

Drug: CLN-049

Part C - Multiple ascending dose (MAD) design of SC administered CLN-049

EXPERIMENTAL

Patients with relapsed/refractory AML or MDS will receive CLN-049 via SC injection

Drug: CLN-049

Interventions

CLN-049 DRUG

\[FLT3\] x \[CD3\] bispecific T cell engager

Part A - Single ascending dose (SAD) design of IV administered CLN-049; Part B - Multiple ascending dose (MAD) design of IV administered CLN-049; Part C - Multiple ascending dose (MAD) design of SC administered CLN-049

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged ≥ 18 years of age.
• 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.
• Patient has a confirmed diagnosis of recurrent or refractory AML or MDS.
• Patient has received, and has progressed, recurred, or is intolerant of approved therapeutic options that are available, or declines treatment with these therapies.
• 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).
• Eastern Cooperative Oncology Group (ECOG) performance status is 0 to 2.
• 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.
• The patient's laboratory values meet the following criteria:
• Creatinine clearance (CrCl) as calculated by the Cockcroft-Gault formula (Appendix 1) must be ≥ 60 mL/min;
• 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;
• AST and ALT ≤ 3.0 × ULN (unless attributed to leukemic involvement).

You may not qualify if:

• Diagnosis of acute promyelocytic leukemia (APL)
• 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.
• Isolated extramedullary relapse
• Prior organ allograft
• 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.
• Treatment with any of the following:
• 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).
• Prior immunotherapy with checkpoint inhibitors ≤ 42 days prior to the first dose of CLN-049.
• Prior history of chimeric antigen receptor (CAR-T) cell therapy or other modified T cell therapy.
• 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.
• Short-acting hematopoietic growth factors ≤ 7 days prior to the first dose of CLN-049
• Long-acting growth factors ≤ 14 days prior to the first dose of CLN-049.
• 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.
• Prior treatment with a FLT3-directed bispecific molecule, or a FLT3-targeted antibody.
• 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.

Sponsors & Collaborators

• Cullinan Therapeutics Inc.: lead

Study Sites (11)

University of Alabama O'Neal Cancer Center

Birmingham, Alabama, 35233, United States

RECRUITING

UCLA

Los Angeles, California, 90095, United States

RECRUITING

University of Miami Health System

Miami, Florida, 33136, United States

RECRUITING

Moffitt Cancer Center

Tampa, Florida, 33612, United States

RECRUITING

Emory University Hospital

Atlanta, Georgia, 30322, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

COMPLETED

Roswell Park Cancer Institute

Buffalo, New York, 14263, United States

RECRUITING

New York University

New York, New York, 10016, United States

RECRUITING

Duke Cancer Center

Durham, North Carolina, 27710, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

WITHDRAWN

MD Anderson

Houston, Texas, 77030, United States

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Central Study Contacts

Timna Serino

CONTACT

+1 617 410 4650; ClinOps@cullinantx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 26, 2021
• First Posted: December 3, 2021
• Study Start: November 18, 2021
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): June 1, 2027
• Last Updated: January 16, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (11)