Actively Recruiting
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
Led by ARCE Therapeutics, Inc. · Updated on 2025-12-10
49
Participants Needed
3
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating ARD103, an investigational CAR-T cell therapy targeting CLL-1, in patients with relapsed or refractory acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). This phase 1/2, open-label, multicenter study aims to assess the safety and effectiveness of ARD103. CLL-1 is a molecule found on leukemia cells but not on normal blood stem cells, making it a promising target for treatment in these blood cancers. The study includes a phase 1 dose escalation part where three increasing dose levels of ARD103 will be tested to find the maximum tolerated dose and recommended phase 2 dose. Phase 2 has two stages: the first involves patients from phase 1 treated at the recommended dose, and the second enrolls additional patients to further evaluate safety and overall response. ARD103 is given as a single intravenous infusion after patients receive lymphodepletion chemotherapy with cyclophosphamide and fludarabine. Participants will be monitored for treatment-related side effects for 28 days after infusion and followed for up to 24 months to measure overall response rate, progression-free survival, and time to best response. Survival will be observed up to 15 years. Assessments include blood tests, physical exams, and other evaluations to track safety and effectiveness. The total duration of participation varies with long-term follow-up planned to understand outcomes and safety over time.
CONDITIONS
Brief Title
Clinical Study of ARD103 CAR-T Therapy for Patients With R/R AML or MDS
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Documented diagnosis of AML with either refractory or relapsed disease or diagnosis of MDS with at least 5% bone marrow blasts
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
- Absolute lymphocyte count greater than 100/mm3
- Adequate liver function with ALT and AST less than 3 times the upper limit of normal
- Adequate kidney function with creatinine clearance at least 45 mL/min as estimated by Cockcroft-Gault and no dialysis
- Total bilirubin less than or equal to 2.0 mg/dL
- Negative pregnancy test for females of childbearing potential
- Agreement to use effective contraception for males and females of childbearing potential
- Ability to give signed informed consent
You will not qualify if you...
- Diagnosis of acute promyelocytic leukemia
- Active and clinically relevant central nervous system disorder
- Autoimmune disease requiring immunosuppressive treatment
- Known hepatic bridging cirrhosis
- Active infection with hepatitis B or C
- Previous treatment with investigational gene or cell therapy including CAR therapy
- Active acute graft-versus-host disease or systemic treatment with more than 10 mg prednisone daily (or equivalent)
- Chemotherapy including biologic, targeted, or immunological agents within 14 days prior to screening and during the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 28 days post ARD103 infusion
Participants receive lymphodepletion with cyclophosphamide and fludarabine followed by a single intravenous infusion of ARD103 CAR-T cell therapy targeting CLL-1.
Multiple visits during the treatment period including infusion and monitoring
Duration - Up to 24 months for response and progression-free survival; up to 15 years for overall survival
Participants are monitored for safety, efficacy, and long-term outcomes after treatment.
Regular follow-up visits over the monitoring period
Trial Site Locations
Total: 3 locations
1
Novant Health Cancer Institute
Charlotte, North Carolina, United States, 28204
Actively Recruiting
2
Novant Health Cancer Institute
Winston-Salem, North Carolina, United States, 27201
Actively Recruiting
3
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
A
ARCE Therapeutics, Inc
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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