Acute Myeloid Leukemia, in RelapsePhase 1/2NCT06680752
Clinical Study of ARD103 CAR-T Therapy for Patients With R/R AML or MDS
A Phase 1/2 study testing ARD103, Cyclophosphamide and Fludarabine in people with Acute Myeloid Leukemia, in Relapse and Acute Myeloid Leukemia Refractory, run by ARCE Therapeutics, Inc. at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia Refractory, MDS (Myelodysplastic Syndrome)
- What is being tested?
- ARD103, Cyclophosphamide and Fludarabine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 49 participants
- Where
- 3 sitesUnited States
- Study dates
- 20 May 2025to Dec 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Novant Health Cancer Institute
Charlotte, North Carolina, 28204
Kunal Shah, 980-302-6297, kashah@novanthealth.org
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
Dr. Abhishek Maiti, 713.745.3228, AMaiti@mdanderson.org
- Recruiting
Novant Health Cancer Institute
Winston-Salem, North Carolina, 27201
Gait Jordan, 336-718-8553, PJordan@novanthealth.org
About this study
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
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.
Read the 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.
- Conditions
- Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia Refractory, MDS (Myelodysplastic Syndrome)
- Being tested
- ARD103, Cyclophosphamide, Fludarabine
- Medical subject headings
- Leukemia, Myeloid, Acute, Anemia, Refractory, with Excess of Blasts
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARD103Biological
ARD103 autologous CAR-T cell therapy targeting CLL-1, single iv. infusion
CyclophosphamideDrug
iv administration for lymphodepletion
FludarabineDrug
iv administration for lymphodepletion
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: ARD103; Drug: Cyclophosphamide; Drug: Fludarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
The records of AEs and severity following the first infusion of ARD103.
Measured: 28 days post ARD103 infusion
To determine the RP2D of ARD103
RP2D of ARD103 following a 3+3 dose escalation schema (Phase 1)
Measured: 28 days post ARD103 infusion
To evaluate overall response rate (ORR)
The ORR will be evaluated by European Leukemia Net (ELN) criteria
Measured: Up to 24 months
Secondary outcomes (3)
Overall Survival (OS)
OS is defined as the time from ARD103 infusion to the date of death from any cause.
Measured: First infusion date of ARD103 up to 15 years
Progression-free survival (PFS)
PFS is defined as the time from the infusion of ARD103 infusion to the first recorded response of disease progression, relapse, or death due to any cause, whichever occurs first.
Measured: Up to 24 months
Time to best response
Time to best response is defined as the time from the infusion of ARD103 to the BOR.
Measured: First infusion date of ARD103 up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2024
- Study start20 May 2025
- Primary completionOct 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia, in RelapseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 49 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ARCE Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06680752
- Sponsor's protocol ID
- ARD103_ARCE-CL-P-001
- Status last verified
- Dec 2025
- First submitted
- 29 Oct 2024
- Last updated
- 10 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06680752
- Acronym / sponsor ID
- ARD103_ARCE-CL-P-001
- Conditions
- Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia Refractory, MDS (Myelodysplastic Syndrome)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 49 participants (estimated)
- Lead sponsor
- ARCE Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 20 May 2025 (actual)
- Primary completion
- Oct 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 8 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| phase 1 (Dose Escalation) and phase 2 (Dose Expansion) | Experimental | Biological: ARD103, Drug: Cyclophosphamide, Drug: FludarabineIn 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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:
- Adequate renal, hepatic, cardiac and pulmonary function:
- Pregnancy testing: females of childbearing potential must have a negative serum or urine pregnancy test
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ARD103ARD103 autologous CAR-T cell therapy targeting CLL-1, single iv. infusion | Biological | phase 1 (Dose Escalation) and phase 2 (Dose Expansion) |
| Cyclophosphamideiv administration for lymphodepletion | Drug | phase 1 (Dose Escalation) and phase 2 (Dose Expansion) |
| Fludarabineiv administration for lymphodepletion | Drug | phase 1 (Dose Escalation) and phase 2 (Dose Expansion) |
Primary outcome
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame: 28 days post ARD103 infusion
The records of AEs and severity following the first infusion of ARD103.
To determine the RP2D of ARD103
Time frame: 28 days post ARD103 infusion
RP2D of ARD103 following a 3+3 dose escalation schema (Phase 1)
To evaluate overall response rate (ORR)
Time frame: Up to 24 months
The ORR will be evaluated by European Leukemia Net (ELN) criteria
3 secondary outcomes
Overall Survival (OS)
Time frame: First infusion date of ARD103 up to 15 years
Progression-free survival (PFS)
Time frame: Up to 24 months
Time to best response
Time frame: First infusion date of ARD103 up to 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Dec 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ARD103, Cyclophosphamide and Fludarabine in people with Acute Myeloid Leukemia, in Relapse and Acute Myeloid Leukemia Refractory, run by ARCE Therapeutics, Inc. at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact ARCE Therapeutics, Inc (+886-2-26581868, contact@arcetherapeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: 28 days post ARD103 infusion). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Dec 2025.