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Relapsed AMLPhase 1AMLNCT06768476

CART123 + Ruxolitinib in Relapsed/Refractory AML

A Phase 1 study testing CART123 Cells, Ruxolitinib 10 MG and Ruxolitinib 5 MG in people with Relapsed AML and Refractory AML, run by University of Pennsylvania at 1 site 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 study to assess the safety, feasibility, pharmacokinetics, and preliminary efficacy of CART123 cells given in combination with ruxolitinib in patients with relapsed or refractory acute myeloid leukemia (AML). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Relapsed AML, Refractory AML
What is being tested?
CART123 Cells, Ruxolitinib 10 MG and Ruxolitinib 5 MG
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
1 siteUnited States
Study dates
28 Feb 2025to Mar 2045
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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 1. Signed informed consent form 2. Male or female age ≥ 18 years. 3. Patients with active acute myeloid leukemia (AML) with no available curative treatment options using currently available therapies. This is specifically defined as one of the following:
  • AML that has not achieved a complete remission or morphologic leukemia free state by ELN 2022 criteria57 after at least 2 cycles of induction (includes partial remission or refractory/primary refractory disease), OR:
  • AML relapsed following allogeneic stem cell transplantation (including MDS evolved to AML post-allogeneic stem cell transplant).
  • Note: Morphologic relapse is not required; Patient may have persistent/recurrent disease-associated molecular, phenotypic or cytogenetic abnormalities (i.e., MRD) at any time after allogeneic HCT to qualify. Mutations involving DNMT3A, ASXL1 or TET2 should not count as molecular MRD+ disease unless accompanied by other, more specific disease-related molecular or cytogenetic abnormalities.
  • 4. Patients with relapsed disease after prior allogeneic SCT must be at least 3 months from transplantation (where receipt of the stem cell product is defined as day 0) and must not require systemic immunosuppression (for prevention or treatment of GVHD).
  • 5. Patients must have a suitable stem cell donor identified with projected ability to proceed to transplant within 6-8 weeks of CART123 infusion if clinically indicated. Donor may be matched or mismatched and must be found to be suitable according to the institution's standard criteria.
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  • 6. Adequate organ function defined as:
  • Estimated creatinine clearance > 35mL/min using the CKD-EPI equation for Glomerular Filtration Rate (GFR); Patients must not be on dialysis
  • ALT/AST ≤ 5x upper limit of normal range
  • Direct bilirubin ≤ 2.0 mg/dl, unless the subject has Gilbert's syndrome (≤3.0 mg/dl)
  • Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygen > 92% on room air
  • Left Ventricle Ejection Fraction (LVEF) ≥ 40% confirmed by transthoracic echocardiography or MUGA scan.
  • 7. ECOG Performance Status 0-2.

Who cannot join

  • 1. Patients with the JAK2 V617F mutation by PCR or next generation sequencing. 2. Patients with signs or symptoms indicative of active CNS involvement. A CNS evaluation should be performed as clinically appropriate to rule out CNS involvement.
  • 3. Patients with relapsed AML with t(15:17). 4. Active hepatitis B, active hepatitis C, or other active, uncontrolled infection.
  • 5. Active acute or chronic GVHD requiring systemic therapy. 6. Class III/IV cardiovascular disability according to the New York Heart Association Classification.
  • 7. Clinically apparent arrhythmia or arrhythmias that are not stable on medical management within two weeks of physician-investigator confirmation of eligibility.
  • 8. Dependence on systemic steroids or immunosuppressant medications. For additional details regarding use of steroid and immunosuppressant medications.
  • 9. Planned use of fluconazole within the anticipated study treatment window. For additional details on concomitant medication restrictions.
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  • 10. Receipt of prior CART123 therapy. 11. Pregnant or nursing (lactating) patients. Participants of reproductive potential must agree to use acceptable birth control methods.
  • 12. Active autoimmune disease requiring systemic immunosuppressive treatment equivalent to ≥ 10mg of prednisone. Patients with autoimmune neurologic diseases (such as MS) will be excluded.
  • 13. History of allergy or hypersensitivity to study product excipients (human serum albumin, DMSO, and Dextran 40).

Trial locations and contacts

1 site in 1 country.

United States1 site

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

Other trials in United States

About this study

Official title: Phase I Trial of CART123 Cells Given in Combination With Ruxolitinib in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML)

Phase I, open-label study to assess the safety, feasibility, pharmacokinetics, and preliminary efficacy of CART123 cells given in combination with ruxolitinib in patients with relapsed or refractory acute myeloid leukemia (AML). All subjects will receive a single infusion of CART123 cells following ruxolitinib administration and lymphodepletion. Ruxolitinib dosing will begin at initiation of lymphodepleting chemotherapy (Day -6 ±1d) and continue for up to 14 days post CART123 administration.

Being tested
CART123 Cells, Ruxolitinib 10 MG, Ruxolitinib 5 MG
Keywords
CART, refractory, relapsed, acute, myeloid, leukemia, AML
Medical subject headings
Leukemia, Myeloid, Acute, Recurrence, Leukemia

What participants receive

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

  • CART123 CellsBiological

    1.3x10^8 CART123 cells

  • Ruxolitinib 10 MGDrug

    Twice Daily

  • Ruxolitinib 5 MGDrug

    Twice Daily

Study groups in NCT06768476
Study groupRoleWhat they receive
Arm A: DL1Experimental* LD Chemo: Day -6 to Day -4 (±1d) * Ruxolitinib: Daily beginning on Day -6 (±1d) through D14 post CART123 infusion. * CART123 Cells: Single infusion on Day 0
Receives: Biological: CART123 Cells; Drug: Ruxolitinib 10 MG
Arm A: DL-1Experimental* LD Chemo: Day -6 to Day -4 (±1d) * Ruxolitinib: Daily beginning on Day -6 (±1d) through D14 post CART123 infusion. * CART123 Cells: Single infusion on Day 0
Receives: Biological: CART123 Cells; Drug: Ruxolitinib 5 MG
Arm B: DL-1Experimental* LD Chemo: Day -6 (-1d) to Day -2 (-1d) * Venetoclax: Day -6 (-1d) through Day +7 - Day +14 post-CART123 infusion * Ruxolitinib: Daily beginning on Day -1 through Day +7 post CART123 infusion. * CART123 Cells: Single infusion on Day 0
Receives: Biological: CART123 Cells; Drug: Ruxolitinib 5 MG

What is being measured

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

  1. Evaluate the safety of CART123 cells when given in combination with ruxolitinib

    Type, frequency, severity, and attribution of AEs/SAEs, including the incidence and severity of IEC-associated adverse events.

    Measured: 15 Years

  2. Evaluate the safety of CART123 cells when given in combination with ruxolitinib

    Occurrence of treatment-limiting toxicities (TLTs)

    Measured: 3 months

  3. Evaluate the safety of CART123 cells when given in combination with ruxolitinib

    The proportion of subjects requiring ruxolitinib dose modifications by assigned dose.

    Measured: 3 months

Secondary outcomes (8)

  1. Evaluate study feasibility

    The proportion of eligible subjects who receive both investigational products as per protocol.

    Measured: 3 months

  2. Evaluate study feasibility

    The proportion of eligible subjects who receive all planned doses of ruxolitinib.

    Measured: 3 months

  3. Describe preliminary efficacy of CART123 cells given in combination with ruxolitinib

    Objective Response Rate (ORR) at Day 28

    Measured: From enrollment to Day 28, post treatment.

  4. Describe preliminary efficacy of CART123 cells given in combination with ruxolitinib

    Duration of Response (DOR)

    Measured: 15 years

  5. Describe preliminary efficacy of CART123 cells given in combination with ruxolitinib

    Progression-free survival (PFS)

    Measured: 15 Years

  6. Describe preliminary efficacy of CART123 cells given in combination with ruxolitinib

    Overall Survival (OS)

    Measured: 15 Years

  7. Describe preliminary efficacy of CART123 cells given in combination with ruxolitinib

    Reduction of blast count in the peripheral blood and marrow using standard clinical criteria (CBC with differential count, marrow aspirate with differential count) and flow cytometry at Day 28

    Measured: From enrollment to Day 28, post treatment.

  8. Describe preliminary efficacy of CART123 cells given in combination with ruxolitinib

    Percentage of subjects undergoing alloHCT

    Measured: 15 Years

Timeline

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

  1. First posted10 Jan 2025
  2. Study start28 Feb 2025
  3. Primary completionMar 2045 (estimated)
  4. Study completionMar 2045 (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
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06768476
Sponsor's protocol ID
50424
Acronym
AML
Status last verified
Jan 2026
First submitted
6 Jan 2025
Last updated
16 Jan 2026
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 CART123 Cells, Ruxolitinib 10 MG and Ruxolitinib 5 MG in people with Relapsed AML and Refractory AML, run by University of Pennsylvania at 1 site 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 13 inclusion and 9 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: under way, not looking for participants, as of the sponsor's last update on 16 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 1 site 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?

Evaluate the safety of CART123 cells when given in combination with ruxolitinib (time frame: 15 Years). 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 NCT06768476.

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 16 Jan 2026.

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