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

A Phase 1/2, Open-label, Multicenter Trial to Assess the Safety and Efficacy of ARD103 in Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

This is a phase I/2, interventional, open-label, multicenter study to assess the safety and efficacy of ARD103 in patients with relapsed or refractory acute myeloid leukemia or myelodysplastic syndrome.

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