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Acute Myeloid Leukemia (AML)Phase 1NCT04230265

Phase 1 Study of UniCAR02-T-CD123 in Patients With Selected CD123 Positive Hematologic Malignancies

A Phase 1 study testing Cyclophosphamide (Non-IMP), Fludarabine (Non-IMP) and TM123 (IMP) in people with Acute Myeloid Leukemia (AML), run by AvenCell Europe GmbH at 10 sites in 2 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This dose-escalating phase I trial assesses for the first time the safety, the side effects and the harmlessness, as well as the therapeutical benefit of the new study drug UniCAR02-T-CD123 in patients with hematologic and lymphatic malignancies positive for CD123 marker. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Acute Myeloid Leukemia (AML)
What is being tested?
Cyclophosphamide (Non-IMP), Fludarabine (Non-IMP) and TM123 (IMP)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
32 participants
Where
10 sitesin 2 countries
Study dates
28 Jan 2020to 11 Apr 2025
Phase
Phase 1
Are there results?
None posted in the registry

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

  • Male or female patients, age ≥ 18 years
  • Documented definitive diagnosis of Relapsed or refractory AML (according to standard of care testing) and CD123 positivity of ≥20 % of blasts. MRD+ AML without morphological relapse or refractoriness may be included with the sponsor's approval
  • Eastern Cooperative Oncology Group (ECOG) of 0 to 1
  • Life expectancy of at least 2 months
  • Adequate renal and hepatic laboratory assessments
  • Adequate cardiac function
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  • Long-term venous access existing (e.g. port-system) resp. acceptance of implantation of a device
  • Able to give written informed consent
  • Weight ≥ 45 kg
  • Negative pregnancy test; routinely using a highly effective method of birth control
  • Male or female patients, age ≥ 18 years
  • Relapsed or refractory AML (according to standard of care testing), having up to 30% blasts in a bone marrow assessment at either screening or prescreening, or patients having between 30% and 40% blasts for 2 consecutive bone marrow assessments with a minimum of 1 months and no more than 2 months apart, and without hyperproliferative disease requiring cytoreductive treatment, up to 3rd relapse, without further approved curative or life-extending treatment options, and documented CD123 positivity of ≥ 20 % of blasts. Exceptions to BM blast criterion are only possible in minor deviations in timing and/or blast count in clinically stable patients, and only with written sponsor approval. Exemptions to CD123 expression are not allowed. MRD+ AML without morphological relapse or refractoriness may be included with the sponsor's approval.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1
  • Life expectancy of at least 2 months
  • Adequate renal and hepatic laboratory assessments:
  • Aspartate aminotransferase (AST), alanine aminotransferase (ALT) and alkaline phosphatase (ALP) ≤ 2.5× upper limit of normal (ULN)
  • Total bilirubin ≤ 1.5× ULN
  • Serum creatinine clearance at least 70 mL/min)
  • Adequate cardiac function, i.e., left ventricular ejection fraction (LVEF) of ≥ 50 %.
  • Long-term venous acces existing (e.g., port-system) resp. acceptance of implantation of a device
  • Able to give written informed consent
  • Weight ≥ 45kg
  • Negative pregnancy test; routinely using a highly effective method of birth control

Who cannot join

  • Acute promyelocytic leukemia
  • AML with only extramedullary manifestations (e.g., chloroma, primary myeloid sarcoma).
  • Refractory disease under anti-leukemic treatment lasting longer than 6 months
  • Current manifestation of AML in central nervous system
  • Bone marrow failure syndromes (e.g. Fanconi anemia, Kostman syndrome, Shwachman syndrome)
  • Significant cardiac disease: i.e., heart failure (NYHA III or IV); unstable coronary artery disease, myocardial infarction or serious cardiac ventricular arrhythmias requiring anti-arrhythmic therapy within the last 12 months prior to study entry that may in the Investigator's opinion interfere with participation in the trial.
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  • Patients undergoing renal dialysis
  • Pulmonary disease with clinically relevant hypoxia
  • Parkinson's disease, epilepsy, stroke, seizures, significant paresis or aphasia with clinical symptoms in the previous 12 months that may in the Investigator's opinion interfere with participation in the trial.
  • Disseminated intravascular coagulation (DIC) within 3 months prior to the planned start of the study treatment.
  • Hemorrhagic cystitis
  • Active infectious disease considered by investigator to be incompatible with protocol or being contraindications for lymphodepletion therapy
  • Allogeneic stem cell transplantation within last two months or GvHD requiring systemic immunosuppressive therapy
  • Vaccination with live viruses within 2 weeks prior to lymphodepletion therapy
  • Major surgery within 28 days (prior to start of TM123 infusion)
  • Other malignancy requiring active therapy, but adjuvant endocrine therapy is allowed
  • Treatment with any investigational drug substance or experimental therapy within 4 weeks or 5 half-lives (whatever is shorter) of the substance prior to the day of apheresis
  • Prior treatment with gene therapy products unless approved by the sponsor
  • Use of checkpoint inhibitors within 5 half-lives of the respective substance
  • Pregnant or breastfeeding women
  • Currently significant psychologic disorder, including substance abuse
  • Known history of human immunodeficiency virus (HIV) or human T-lymphotropic virus (HTLV) or active/chronic infection with hepatitis C virus (HCV) or hepatitis B virus (HBV)
  • Any significant autoimmune disease requiring systemic immunosuppressive therapy or that may otherwise, in the Investigator's opinion, interfere with participation in the trial, or documented presence of autoantibodies against La/SS-B.
  • Phase 1b Dose Expansion:
  • Acute promyelocytic leukemia (t15;17)
  • AML with only extramedullary manifestations (e.g., chloroma, primary myeloid sarcoma)
  • Refractory disease under anti-leukemic treatment lasting longer than 6 months
  • Current manifestion of AML in central nervous system
  • Bone marrow failure syndromes (e.g. Fanconi anemia, Kostman syndrome, Schwachman syndrome)
  • Significant cardiac disease: i.e., heart failure (NYHA III or IV); unstable coronary artery disease, myocardial infarction or serious cardiac ventricular arrhythmias requiring anti-arrhythmic therapy within the last 12 months prior to study entry that may in the Investigator''s opinion interfere with participation in the trial.
  • Patients undergoing renal dialysis
  • Pulmonary disease with clinically relevant hypoxia
  • Parkinson's disease, epilepsy, stroke, seizures, significant paresis or aphasia with clinical symptoms in the previous 12 months that may in the Investigator's opinion interfere with participation in the trial.
  • Disseminated intravascular coagulation (DIC) within 3 months prior to the planned start of the study treatment.
  • Hemorrhagic cystitis
  • Active infections disease considered by investigator to be incompatible with protocol or being contraindications for lymphodepletion therapy.
  • Allogenic stem cell transplantation within last two months or GvHD requiering systemic immunosuppressive therapy.
  • Vaccination with live viruses within 2 weeks prior to lymphodepletion therapy.
  • Major surgery within 28 days (prior to start of TM123 infusion)
  • Other malignancy requiring active therapy, but adjuvant endocrine therapy is allowed.
  • Treatment with any investigational drug substance or experimental therapy within 4 weeks or 5 half-lives (whatever is shorter) of the substance prior to the day of apheresis
  • Prior treatment with gene therapy products unless approved by the sponsor.
  • Use of checkpoint inhibitors within 5 half-lives of the respective substance.
  • Pregnatn or breastfeeding women.
  • Currently significant psychologic disorder, including substance abuse.
  • Known history of human immunodeficiency virus (HIV) or human T-lymphotropic virus (HTLV) or active/chronic infection with hepatitis C virus (HCV) or hepatitis B virus (HBV).
  • Any significant autoimmune disease requiring systemic immunosuppressive therapy or that may otherwise, in the Investigator''s opinion, interfere with participation in the trial, or documented presence of autoantibodies against La/SS-B.

Trial locations and contacts

10 sites in 2 countries.

10 sites in 2 countries

Germany8 sites

  • Uniklinik RWTH Aachen

    Aachen, North Rhine-Westphalia, 52074

  • Universitätsklinikum Dresden

    Dresden, Saxony, 01307

  • Universitätsklinikum Hamburg-Eppendorf

    Hamburg, 20246

  • Universitätsklinikum Leipzig

    Leipzig, Saxony, 04103

  • Philipps-Universität Marburg

    Marburg, Hesse, 35032

  • Klinikum der Universität München

    Munich, Bavaria, 81377

  • Universitätsklinikum Ulm

    Ulm, Baden-Wurttemberg, 89081

  • Universitätsklinikum Würzburg

    Würzburg, Bavaria, 97080

Other trials in Germany

Netherlands2 sites

  • Universitair Medisch Centrum Groningen

    Groningen, GZ, 9713

  • Erasmus University Medical Center

    Rotterdam, Gelderland, 3015

Other trials in Netherlands

About this study

Official title: Multicenter, Open-label, Adaptive Design Phase I Trial With Genetically Modified T-cells Carrying Universal Chimeric Antigen Receptors (UniCAR02-T) in Combination With CD123 Target Module (TM123) for the Treatment of Patients With Hematologic and Lymphatic Malignancies Positive for CD123

This dose-escalating phase I trial assesses for the first time the safety, the side effects and the harmlessness, as well as the therapeutical benefit of the new study drug UniCAR02-T-CD123 in patients with hematologic and lymphatic malignancies positive for CD123 marker. The UniCAR02-T-CD123 drug is a combination of a cellular component (UniCAR02-T) with a recombinant antibody derivative (TM123) which together forms the active drug.

Being tested
Cyclophosphamide (Non-IMP), Fludarabine (Non-IMP), TM123 (IMP), UniCAR02-T (IMP)
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Cyclophosphamide (Non-IMP)Drug

    Intravenous infusion over 3 days

  • Fludarabine (Non-IMP)Drug

    Intravenous infusion over 3 days

  • TM123 (IMP)Drug

    Intravenous Infusion for 20 days

  • UniCAR02-T (IMP)Drug

    Intravenous infusion of single dose

Study groups in NCT04230265
Study groupRoleWhat they receive
UniCAR02-T-CD123 (4 mg/day TM123)ExperimentalPreconditioning (lymphodepletion) with cyclophosphamide and fludarabine, followed by combination treatment of genetically modified T-cells carrying universal chimeric antigen receptors (UniCAR02-T) with 4 mg/day of the recombinant antibody derivative TM123.
Receives: Drug: Cyclophosphamide (Non-IMP); Drug: Fludarabine (Non-IMP); Drug: TM123 (IMP); Drug: UniCAR02-T (IMP)
UniCAR02-T-CD123 (8 mg/day TM123)ExperimentalPreconditioning (lymphodepletion) with cyclophosphamide and fludarabine, followed by combination treatment of genetically modified T-cells carrying universal chimeric antigen receptors (UniCAR02-T) with 8 mg/day of the recombinant antibody derivative TM123.
Receives: Drug: Cyclophosphamide (Non-IMP); Drug: Fludarabine (Non-IMP); Drug: TM123 (IMP); Drug: UniCAR02-T (IMP)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety and tolerability

    Incidence and intensity of adverse events graded according to CTCAE V5.0 with the exception of CRS and ICANS graded according to Lee et al. 2014 and Lee et al. 2019 respectively

    Measured: Infusion period of TM123 (up to 20 days) + 7 days resp. 14 days in patients with complete blast clearance during the IC or until Safety Follow-up 1 (infusion period of TM123 + 28 days + 3 months)

  2. Recommended phase 2 dose (RP2D)

    Establishing recommended phase 2 dose (RP2D) and schedule

    Measured: Infusion period of TM123 (up to 20 days) + 7 days or + 14 days in patients with complete blast clearance)

  3. Response

    Complete remission (CR, CRh, CRi, CRMRDneg, CRMRDpos) and partial remission (PR) at any time point and duration of responses

    Measured: Infusion period of TM123 (up to 20 days) + 7 days or + 14 days in patients with complete blast clearance)

Secondary outcomes (7)

  1. Establishing recommended phase 2 dose (RP2D)

    The RP2D will be determined based on MTD, all available efficacy data, and all available safety data, including information derived from additional treatment cycles.

    Measured: DLT period (infusion period of TM123 (up to 20 days) + 7 days or + 14 days in patients with complete blast clearance)

  2. Complete (CR, CRh, CRi ) and partial remission (PR)

    CR: Bone marrow blasts < 5%, absence of extramedullary disease, absolute neutrophil count > 1 Gpt/L and platelet count > 100 Gpt/L. Level of MRD should be measured in patients achieving CR in case as suitable marker exists.

    CRi: All criteria for CR except residual thrombocytopenia (platelets < 100 Gpt/L) and/or neutropenia (absolute neutrophil count < 1 Gpt/L).

    PR: All hematological criteria for CR with bone marrow blasts 5-25% and decrease of pre-treatment bone marrow blast percentage by at least 50 %.

    Measured: until fifteen years after last UniCAR02-T administration

  3. Disease stabilization (DS)

    Reduction of blast percentage by 25% compared to baseline without normalization of peripheral blood counts to levels not qualifying for PR or CR.

    Measured: until fifteen years after last UniCAR02-T administration

  4. Best response rate

    The best observed response during observational period. Response states are ordered descending as follows: CR > CRi > PR > DS > refractory disease.

    Measured: until fifteen years after last UniCAR02-T administration

  5. Progression free survival (PFS)

    The time from first treatment with TM123 and UniCAR02-T until disease progression or death. If no progress of death was observed during the observational period, the patient's progression free survival time will be censored on the date the patient was last seen progression-free and alive.

    Measured: until fifteen years after last UniCAR02-T administration

  6. Overall survival (OS)

    The number of days between the first study drug administration and death from any cause. If death was not observed during the observational period, the patient's overall survival time will be censored on the date the patient was last seen alive.

    Measured: until fifteen years after last UniCAR02-T administration

  7. Toxicity and efficacy in repeated cycles of TM123 administration

    Patients who tolerate TM123 and UniCAR02-T without DLT and achieve a clinical benefit are candidates for consolidation cycles

    Measured: duration of consolidation cycle treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Jan 2020
  2. Study start28 Jan 2020
  3. Primary completion11 Apr 2025
  4. Study completion11 Apr 2025

Why it stopped: business decision by sponsor

Resources

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
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Phase 1b Dose Expansion: An expansion cohort of up to 20 patients was initiated.
Masking
None (open label)
Enrolment
32 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04230265
Sponsor's protocol ID
UC02-123-01
Other IDs
2019-001339-30, 2024-515827-12-00, 2019
Status last verified
Jan 2026
First submitted
10 Jan 2020
Last updated
22 Jan 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Loff S, Dietrich J, Meyer JE, Riewaldt J, Spehr J, von Bonin M, Grunder C, Swayampakula M, Franke K, Feldmann A, Bachmann M, Ehninger G, Ehninger A, Cartellieri M. Rapidly Switchable Universal CAR-T Cells for Treatment of CD123-Positive Leukemia. Mol Ther Oncolytics. 2020 Apr 29;17:408-420. doi: 10.1016/j.omto.2020.04.009. eCollection 2020 Jun 26.

    PubMed 32462078

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Cyclophosphamide (Non-IMP), Fludarabine (Non-IMP) and TM123 (IMP) in people with Acute Myeloid Leukemia (AML), run by AvenCell Europe GmbH at 10 sites in 2 countries.

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 23 inclusion and 47 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: stopped early, as of the sponsor's last update on 22 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in Germany, Netherlands. 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 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: business decision by sponsor). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety and tolerability (time frame: Infusion period of TM123 (up to 20 days) + 7 days resp. 14 days in patients with complete blast clearance during the IC or until Safety Follow-up 1 (infusion period of TM123 + 28 days + 3 months)). 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?

On the ClinicalTrials.gov record for NCT04230265, which also lists 1 linked publication.

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 22 Jan 2026.

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