Clinical trial NCT06680752: Clinical Study of ARD103 CAR-T Therapy for Patients With R/R AML or MDS
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Acute Myeloid Leukemia, in Relapse
Acute Myeloid Leukemia Refractory
MDS (Myelodysplastic Syndrome)
Lead sponsor: ARCE Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2024-11-08
Enrollment: 49
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
timeFrame: 28 days post ARD103 infusion
measure: To determine the RP2D of ARD103
timeFrame: 28 days post ARD103 infusion
measure: To evaluate overall response rate (ORR)
timeFrame: Up to 24 months
Secondary outcomes:
measure: Overall Survival (OS)
timeFrame: First infusion date of ARD103 up to 15 years
measure: Progression-free survival (PFS)
timeFrame: Up to 24 months
measure: Time to best response
timeFrame: First infusion date of ARD103 up to 24 months
Other outcomes:
Locations:
city: Charlotte
country: United States
facility: Novant Health Cancer Institute
state: North Carolina
city: Winston Salem
country: United States
facility: Novant Health Cancer Institute
state: North Carolina
Study officials:
References: