Clinical Study of ARD103 CAR-T Therapy for Patients With R/R AML or MDS

The investigational product (IP) for this study is ARD103, a C-type lectin-like molecule-1 (CLL-1) autologous chimeric antigen receptor T-cells (CAR-T). CLL-1 is highly expressed on both myeloid blasts and leukemia stem cells (LSCs) but is absent on normal hematopoietic stem cells (HSCs), suggesting CLL-1 as an excellent therapeutic target for AML and hence other potential myeloid malignancies.

Inclusion criteria

• Documented diagnosis of AML with either refractory or relapsed disease or diagnosis of MDS and ≥ 5% BM blasts
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
• Adequate hematologic status:

• Absolute lymphocyte count (ALC) > 100/mm3
• Adequate renal, hepatic, cardiac and pulmonary function:

• ALT and AST < 3.0 × the ULN
• Creatinine clearance ≥ 45.0 mL/min as estimated by Cockcroft-Gault and independent dialysis
• Total bilirubin ≤ 2.0 mg/dL
• Pregnancy testing: females of childbearing potential must have a negative serum or urine pregnancy test
• Contraception: males and females of childbearing potential must agree to use an effective method of contraception
• Participant is capable of giving signed informed consent

Exclusion criteria

• Participants with acute promyelocytic leukemia
• Presence of active and clinically relevant central nervous system (CNS) disorder
• Autoimmune disease requiring immunosuppressive treatment
• Participants with known hepatic bridging cirrhosis
• Currently active infection with hepatitis B or C
• Previous treatment with investigational gene or cell therapy (including CAR therapy)
• Any active acute GvHD or systemic treatment of more than 10 mg prednisone daily (or equivalent)
• Previous chemotherapy including biologic/targeted therapy or immunological agents directed to the pathology within 14 days prior to screening and all along the study duration