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

ARCE Therapeutics, Inc.

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Acute Myeloid Leukemia Refractory; MDS (Myelodysplastic Syndrome); Acute Myeloid Leukemia, in Relapse

Treatments: Drug: Cyclophosphamide; Biological: ARD103; Drug: Fludarabine

Study type: Interventional

Funder types: Industry

Identifiers

NCT06680752

ARD103_ARCE-CL-P-001

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 49 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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49 participants in 1 patient group

phase 1 (Dose Escalation) and phase 2 (Dose Expansion)

Experimental group

Description:

In Phase 1, three escalating dose levels will be tested using the 3 + 3 design. The MTD and RP2D will be identified. The Phase 2 will be conducted in 2 stages. In Stage I, evaluable participants from Phase 1 treated at RP2D will be enrolled. And the enrollment will continue into Stage II (additional evaluable participants) at the maximum RP2D participants for preliminary overall assessment of efficacy and safety.

Treatment:

Drug: Fludarabine

Biological: ARD103

Drug: Cyclophosphamide