Recruiting
Phase 1
Phase 2

ARD103

Sponsor:

ARCE Therapeutics, Inc.

Code:

NCT06680752

Conditions

Acute Myeloid Leukemia, in Relapse

Acute Myeloid Leukemia Refractory

MDS (Myelodysplastic Syndrome)

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

ARD103

Cyclophosphamide

Fludarabine

Study Details

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)

Study ID

NCT06680752

Start date

May 20, 2025

Status verified date

Dec, 2025

Completion date

Dec, 2028

Anticipated

Primary completion date

Oct, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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 Design

Enrollment

49 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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.

Interventions

ARD103

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

Cyclophosphamide

iv administration for lymphodepletion

Fludarabine

iv administration for lymphodepletion

Primary outcome measure

  • Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 28 days post ARD103 infusion ]
  • To determine the RP2D of ARD103 [ Time Frame: 28 days post ARD103 infusion ]
  • To evaluate overall response rate (ORR) [ Time Frame: Up to 24 months ]

Central Contacts and Locations

Central contacts

ARCE Therapeutics, Inc

+886-2-26581868contact@arcetherapeutics.com

Locations

Novant Health Cancer Institute

Recruiting

Charlotte, North Carolina, United States, 28204

Contacts

Kunal Shah

980-302-6297kashah@novanthealth.org

Novant Health Cancer Institute

Recruiting

Winston-Salem, North Carolina, United States, 27201

Contacts

Gait Jordan

336-718-8553PJordan@novanthealth.org

MD Anderson Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Dr. Abhishek Maiti

713.745.3228AMaiti@mdanderson.org

More Information

Sponsor

ARCE Therapeutics, Inc.

Last update posted

Dec 10, 2025

Last verified

Dec, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by ARCE Therapeutics, Inc. on 2025-12-10.