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

NCT06680752 | Recruiting Phase 1 FDA Drug

• 1 other identifier: ARD103_ARCE-CL-P-001
• Study Type: interventional
• Target: 49
• Locations: 1 country
• Sites: 3

Brief Summary

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.

Conditions

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

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (3)

• Overall Survival (OS)

  First infusion date of ARD103 up to 15 years

• Progression-free survival (PFS)

  Up to 24 months

• Time to best response

  First infusion date of ARD103 up to 24 months

Study Arms (1)

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

EXPERIMENTAL

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; Drug: Cyclophosphamide; Drug: Fludarabine

Interventions

ARD103 BIOLOGICAL

ARD103 autologous CAR-T cell therapy targeting CLL-1, single iv. infusion

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

Cyclophosphamide DRUG

iv administration for lymphodepletion

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

Fludarabine DRUG

iv administration for lymphodepletion

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• ARCE Therapeutics, Inc.: lead

Study Sites (3)

Novant Health Cancer Institute

Charlotte, North Carolina, 28204, United States

RECRUITING

Novant Health Cancer Institute

Winston-Salem, North Carolina, 27201, United States

RECRUITING

MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Anemia, Refractory, with Excess of Blasts

Interventions

Cyclophosphamide; fludarabine

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Anemia, Refractory; Anemia; Myelodysplastic Syndromes; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Central Study Contacts

ARCE Therapeutics, Inc

CONTACT

+886-2-26581868; contact@arcetherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 29, 2024
• First Posted: November 8, 2024
• Study Start: May 20, 2025
• Primary Completion (Estimated): October 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: December 10, 2025

Record last verified: 2025-12

Locations

US (3)