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.

Study Overview

• Status: Recruiting
• Conditions: Acute Myeloid Leukemia, in Relapse; Acute Myeloid Leukemia Refractory; MDS (Myelodysplastic Syndrome)
• Intervention / Treatment: Biological: ARD103; Drug: Cyclophosphamide; Drug: Fludarabine

Detailed 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.

• Study Type: Interventional
• Enrollment (Estimated): 49
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: ARCE Therapeutics, Inc; Phone Number: +886-2-26581868; Email: contact@arcetherapeutics.com

Study Locations

• United States
  • North Carolina
    • Charlotte, North Carolina, United States, 28204
      • Recruiting
      • Novant Health Cancer Institute
      • Contact: Kunal Shah; Phone Number: 980-302-6297; Email: kashah@novanthealth.org
    • Winston-Salem, North Carolina, United States, 27201
      • Recruiting
      • Novant Health Cancer Institute
      • Contact: Gait Jordan; Phone Number: 336-718-8553; Email: PJordan@novanthealth.org
  • Texas
    • Houston, Texas, United States, 77030
      • Recruiting
      • MD Anderson Cancer Center
      • Contact: Dr. Abhishek Maiti; Phone Number: 713.745.3228; Email: AMaiti@mdanderson.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: phase 1 (Dose Escalation) and phase 2 (Dose Expansion); 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.

Biological: ARD103; ARD103 autologous CAR-T cell therapy targeting CLL-1, single iv. infusion; Drug: Cyclophosphamide; iv administration for lymphodepletion; Drug: Fludarabine; iv administration for lymphodepletion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]	The records of AEs and severity following the first infusion of ARD103.	28 days post ARD103 infusion
To determine the RP2D of ARD103	RP2D of ARD103 following a 3+3 dose escalation schema (Phase 1)	28 days post ARD103 infusion
To evaluate overall response rate (ORR)	The ORR will be evaluated by European Leukemia Net (ELN) criteria	Up to 24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival (OS)	OS is defined as the time from ARD103 infusion to the date of death from any cause.	First infusion date of ARD103 up to 15 years
Progression-free survival (PFS)	PFS is defined as the time from the infusion of ARD103 infusion to the first recorded response of disease progression, relapse, or death due to any cause, whichever occurs first.	Up to 24 months
Time to best response	Time to best response is defined as the time from the infusion of ARD103 to the BOR.	First infusion date of ARD103 up to 24 months

Collaborators and Investigators

• Sponsor: ARCE Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): May 20, 2025
• Primary Completion (Estimated): October 1, 2028
• Study Completion (Estimated): December 1, 2028

Study Registration Dates

• First Submitted: October 29, 2024
• First Submitted That Met QC Criteria: November 7, 2024
• First Posted (Actual): November 8, 2024

Study Record Updates

• Last Update Posted (Actual): December 10, 2025
• Last Update Submitted That Met QC Criteria: December 4, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia, Myeloid; Bone Marrow Diseases; Anemia; Leukemia; Myelodysplastic Syndromes; Anemia, Refractory; Hemic and Lymphatic Diseases; Leukemia, Myeloid, Acute; Anemia, Refractory, with Excess of Blasts; Organic Chemicals; Hydrocarbons; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds; Cyclophosphamide; fludarabine
• Other Study ID Numbers: ARD103_ARCE-CL-P-001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No