A Long-term Follow-up Study of Patients With ARD103 CAR-T Cell Therapies

A Long-term Follow-up Study of Patients Enrolled in ARD103_ARCE-CL-P-001 Phase 1/2 Study and Treated With ARD103 CAR-T Cell Therapies

This study will evaluate the long-term safety of ARD103 cellular therapies

Study Overview

• Status: Not yet recruiting
• Conditions: Acute Myeloid Leukemia, in Relapse; MDS (Myelodysplastic Syndrome); Acute Myeloid Leukemia, in Relapsed or Refractory
• Intervention / Treatment: Other: Non interventional study

Detailed Description

Participants who receive administration of ARD103 (i.e., a partial dose or a full dose) will roll into the LTFU study upon completion of the 24-month interventional study period (Study: ARD103_ARCE-CL-P-001), or after early discontinuation from the interventional study protocol. The LTFU study is a study intended to characterize long-term safety for 15 years post-ARD103 administration. Participants who complete the full 24-month interventional study period will undergo an additional 13 years of monitoring under the LTFU study. No IP will be administered during the LTFU study. The number and percentage of participants with ARD103 CAR-T cell therapy related AEs, SAEs, and AESIs will be recorded throughout the LTFU Period.

• Study Type: Observational
• Enrollment (Estimated): 49

Contacts and Locations

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

Study Locations

• United States
  • North Carolina
    • Charlotte, North Carolina, United States, 28204
      • Novant Health Cancer Institute
      • Contact: Kunal Shah; Phone Number: 980-302-6297; Email: kashah@novanthealth.org
    • Winston-Salem, North Carolina, United States, 27201
      • Novant Health Cancer Institute
      • Contact: Gait Jordan; Phone Number: 336-718-8553; Email: PJordan@novanthealth.org
  • Texas
    • Houston, Texas, United States, 77030
      • 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: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study population will comprise participants with R/R AML or MDS treated with ARD103. All subjects with AML or MDS who are enrolled in a parent study and were received ARD103 CAR-T infusion will be asked to participate in this long-term follow-up (LTFU) study.

Description

Inclusion Criteria:

• The participant has R/R AML or MDS and has previously received ARD103 CAR-T cell therapies in the parent study.
• The participant is able to understand and comply with protocol-required study procedures and has provided a written informed consent document.

Exclusion Criteria:

• There are no specific exclusion criteria for this study

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Non Interventional; All subjects with AML or MDS who are enrolled in a parent study and were exposed to ARD103 CAR-T cellular therapy will be asked to participate in this long-term follow-up (LTFU) study.	Other: Non interventional study; Non Intervention, an observation LTFU study

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The incidence and duration of AEs, SAEs, and AESIs related to ARD103 treatment.	The number and percentages of participants experiencing TEAEs will be tabulated. Serious TEAEs will be summarized and AESIs will be summarized.	15 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The long-term efficacy of ARD103 in participants with R/R AML or MDS	Overall survival (OS): the time from the infusion of ARD103 CAR-T in the parent study until death for any cause. Duration of Response (DOR): the time from the first recorded response after ARD103 infusion to the first recorded efficacy result of disease progression, relapse, or death due to any cause, whichever occurs first. PFS: the time from the infusion of ARD103 in the parent study to the first recorded response of disease progression, relapse, or death due to any cause, whichever occurs first.	15 years

Collaborators and Investigators

• Sponsor: ARCE Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Estimated): March 15, 2026
• Primary Completion (Estimated): May 30, 2040
• Study Completion (Estimated): October 31, 2040

Study Registration Dates

• First Submitted: March 26, 2025
• First Submitted That Met QC Criteria: March 26, 2025
• First Posted (Actual): April 2, 2025

Study Record Updates

• Last Update Posted (Actual): December 8, 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
• Other Study ID Numbers: ARD103_ARCE-CL-P-002

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No