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Relapsed/Refractory Acute Myeloid LeukemiaPhase 1AMELI-01NCT03190278

Study Evaluating Safety and Efficacy of UCART123v1.2 in Patients With Relapsed/Refractory Acute Myeloid Leukemia

A Phase 1 study testing UCART123v1.2 in people with Relapsed/Refractory Acute Myeloid Leukemia, run by Cellectis S.A. at 8 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

Phase I, open-label, dose-escalation and dose-expansion study evaluating the safety and efficacy of Universal Chimeric Antigen Receptor T-cell (UCART) targeting the Cluster of Differentiation 123 (CD123) in patients with relapsed/refractory acute myeloid leukemia (AML). Read more about this study

Who is it for?
Ages 18 to 65 years
What is being tested?
UCART123v1.2
Type of treatment
Vaccines
Could I receive a placebo?
No, everyone receives an active treatment
How many people
29 participants
Where
8 sitesUnited States
Study dates
19 Jun 2017to Dec 2025
Phase
Phase 1
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 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Main Inclusion Criteria:

  • Patients with relapsed or primary refractory AML (as defined in World Health Organization [WHO] criteria) with ≥5% bone marrow blasts
  • Patients with CD123+ blast cells (verified by flow cytometry)
  • Eastern Cooperative Oncology Group Performance Status (ECOG-PS) of ≤1
  • Adequate organ function, including bone marrow, renal, hepatic, pulmonary, and cardiac function based on the last assessment performed within screening period
  • (Dose-escalation) Identified donor and transplant strategy prior to lymphodepletion (LD)
  • Other criteria may apply

Main Exclusion Criteria:

  • Patients with acute promyelocytic leukemia (APL) or central nervous system (CNS) Leukemia
  • Previous investigation gene or cell therapy (including CAR)
  • > 1 prior allogeneic stem cell transplantations (SCTs)
  • Prior treatment with rituximab or other anti-cluster of differentiation 20 (anti-CD20) therapy within 3 months
  • Any known active or uncontrolled infection
  • Other criteria may apply

Trial locations and contacts

8 sites in 1 country.

8 sites in 1 country

United States8 sites

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

  • Roswell Park Cancer Institute

    Buffalo, New York, 14263

  • Northwestern University

    Chicago, Illinois, 60201

  • MD Anderson Cancer Center

    Houston, Texas, 77030

  • Weill Medical College of Cornell University

    New York, New York, 10021

  • University of Pennsylvania - Abramson Cancer Center

    Philadelphia, Pennsylvania, 19104

  • University of California, San Francisco (UCSF) - Helen Diller Family Comprehensive Cancer Center

    San Francisco, California, 94143

  • H. Lee Moffitt Cancer Center & Research Institute

    Tampa, Florida, 33612

Other trials in United States

About this study

Official title: Phase I, Open Label Dose Escalation and Dose-Expansion Study to Evaluate the Safety, Expansion, Persistence, and Clinical Activity of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor), Administered in Patients With Relapsed/Refractory Acute Myeloid Leukemia

Phase I, open-label, dose-escalation and dose-expansion study evaluating the safety and efficacy of Universal Chimeric Antigen Receptor T-cell (UCART) targeting the Cluster of Differentiation 123 (CD123) in patients with relapsed/refractory acute myeloid leukemia (AML). The purpose of this study is to evaluate the safety and clinical activity of Universal Chimeric Antigen Receptor T-cells targeting CD123 (UCART123v1.2) and determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D).

Being tested
UCART123v1.2
Keywords
Acute Myeloid Leukemia, Relapsed/Refractory Acute Myeloid Leukemia, Chimeric Antigen Receptor T-Cell (CAR-T) therapy, Allogeneic, Transcription Activator-Like Effector Nuclease (TALEN)
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.

  • UCART123v1.2Biological

    Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor Biological/vaccine: CLLS52 A monoclonal antibody that recognizes the CD52 antigen Other Names: Alemtuzumab

Study groups in NCT03190278
Study groupRoleWhat they receive
Dose EscalationExperimentalUCART123v1.2 tested at several dose levels with different lymphodepletion regimens to establish Maximum Tolerated Dose (MTD) and identify Recommended Phase 2 Dose (RP2D) Dose Expansion: UCART123v1.2 administered at the RP2D determined from the dose escalation phase
Receives: Biological: UCART123v1.2

What is being measured

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

  1. Incidence of adverse events (AE)/serious adverse events (SAE)/Dose Limiting Toxicities (DLT) [Safety and Tolerability]

    Safety of UCART123v1.2 - Incidence, nature, and severity of AE and SAEs throughout the study

    Measured: 24 Months

  2. Dose escalation and expansion part: Occurrence of DLTs

    Measured: Up to Day 28 post last UCART123v1.2 infusion

Secondary outcomes (16)

  1. Investigators assessed overall response rate according to the European Leukemia Net (ELN) Response Criteria

    Measured: At Day 28, Day 56, Day 84, Month 3, Month 6, Month 9, Month 12, Month 15, Month 18, Month 21 and Month 24

  2. Duration of Response

    Measured: From the date of the initial response to the date of disease progression or death from any cause, whichever occurs first, assessed up to Month 24

  3. Progression Free Survival

    Measured: From the first day of study treatment to the date of disease progression or death from any cause, whichever occurs first, assessed up to Month 24

  4. Overall Survival

    Measured: From the first day of study treatment to the date of death from any cause, assessed up to Month 24

  5. Pharmacokinetic (PK) Analysis: Standard PK Analysis will be completed to obtain Maximum plasma concentration (Cmax)

    Measured: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  6. Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain time to reach Cmax (Tmax)

    Measured: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  7. Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain total area under curve from zero to infinity (AUC-infinity)

    Measured: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  8. Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain Terminal Rate

    Measured: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  9. Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain Terminal Half-life

    Measured: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  10. Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain Clearance

    Measured: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  11. Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain Volume of Distribution

    Measured: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  12. Pharmacodynamic Analysis: Pharmacodynamics Monitoring of the incidence of anti-cluster of differentiation 52 (anti-CD52; alemtuzumab) antibodies (ADA) in serum Pre-alemtuzumab administration and through Day 84

    Measured: From screening through Day 84

  13. Pharmacodynamic Analysis: Pharmacodynamics Quantitation of T cells in peripheral blood

    Measured: From screening through Day 84

  14. Pharmacodynamic Analysis: Pharmacodynamics Quantitation of B cells in peripheral blood

    Measured: From screening through Day 84

  15. Pharmacodynamic Analysis: Pharmacodynamics Quantitation of natural killer (NK) cells in peripheral blood

    Measured: From screening through Day 84

  16. Pharmacodynamic Analysis: Pharmacodynamics Quantitation of total lymphocytes in peripheral blood

    Measured: From screening through Day 84

Timeline

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

  1. First posted16 Jun 2017
  2. Study start19 Jun 2017
  3. Primary completion24 Dec 2024
  4. Study completionDec 2025 (estimated)

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
29 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03190278
Sponsor's protocol ID
UCART123_01
Acronym
AMELI-01
Status last verified
Aug 2025
First submitted
14 Jun 2017
Last updated
7 Aug 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing UCART123v1.2 in people with Relapsed/Refractory Acute Myeloid Leukemia, run by Cellectis S.A. at 8 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?

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: under way, not looking for participants, as of the sponsor's last update on 7 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 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 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?

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?

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?

Incidence of adverse events (AE)/serious adverse events (SAE)/Dose Limiting Toxicities (DLT) [Safety and Tolerability] (time frame: 24 Months). The study lists 2 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 NCT03190278.

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 7 Aug 2025.

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