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CART123 Cells With or Without Ruxolitinib for Relapsed/Refractory AML

This study is looking at the safety of a new immunotherapy called CART123 cells, either alone or combined with ruxolitinib (JAKAVI®), for children and young adults (ages 0-29) with acute myeloid leukemia (AML) that has come back or hasn't responded to other treatments. CART123 cells are a type of CAR T-cell therapy, which uses your own modified immune cells to fight cancer. Ruxolitinib is a drug that targets specific proteins in the body. The study aims to find the safest dose of CART123 cells. This trial is for patients with AML in second or greater relapse, post-transplant relapse, or with chemotherapy-refractory disease. The current recruitment status is unclear.

Study design
This is an interventional study with a planned enrollment of 30 participants. It will evaluate CART123 cells alone (Cohort A) or in combination with ruxolitinib (Cohort B).
What's involved
Participants will receive CART123 cells intravenously after chemotherapy, with some receiving ruxolitinib orally. Infusions will occur in an outpatient setting with close follow-up.
Compensation
Not stated in the trial record.
Follow-up
The safety of CART123 and the combination with Ruxolitinib will be evaluated for 5 years.

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NCT07464951

CART123 Cells With or Without Ruxolitinib in Relapsed/Refractory Acute Myeloid Leukemia

Recruiting
PHASE1Ages 0–29InterventionalTreatment
Stephan Grupp MD PhD
~30 participants
Updated 2026-07-07 on ClinicalTrials.gov
What's tested:Anti-CD123 LV redirected T cells (CART123)Ruxolitinib (JAKAVI®)

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Evaluate the Safety of CART123
Measured over 5 years
+2 more outcomes measured

Conditions

Acute Myeloid Leukemia (AML)

Explore related trials

Acute Myeloid Leukemia

Where it's being run

1 sites across 1 states
Pennsylvania1

Study leadership

  • Lucy Cain, MBBS · PRINCIPAL_INVESTIGATOR · Children's Hospital of Philadelphia
  • Stephan Grupp, MD, PhD · STUDY_DIRECTOR · Children's Hospital of Philadelphia

Who to contact

Cell Therapy Nurse Navigator
[email protected]
445-942-5891
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Eligibility criteria

Inclusion

1\. Age at time of consent: Cohort A: 0-29 years. Cohort B: 1-29 years (Note: the first subject at each dose level of Cohort B must be ≥12 years old)
2\. Subjects with AML in second or greater relapse, post-transplant relapse, or with chemotherapy-refractory disease. Specifically:
3\. Subjects must have an identified stem cell donor with the ability to proceed rapidly to transplant following CART123 treatment if indicated.
4\. Adequate organ function defined as:
5\. Adequate performance status defined as Lansky or Karnofsky performance score ≥ 50.
6\. Subjects of reproductive potential must agree to use acceptable birth control methods.

Exclusion

1\. Active hepatitis B or active hepatitis C
2\. HIV infection
3\. Active acute or chronic GVHD requiring systemic therapy
4\. Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.
5\. CNS disease that is progressive on therapy, or with CNS parenchymal lesions that might increase the risk of CNS toxicity.
6\. Pregnant or nursing (lactating) subjects.
7\. Uncontrolled active infection

What this trial measures

  • Evaluate the Safety of CART1235 years

    Frequency of Adverse events will be measured by evaluating the frequency and severity of treatment related adverse events following administration of CART123

  • Safety of CART123 in combination with Ruxolitinib5 Years

    Frequency of Adverse events will be measured by evaluating the frequency and severity of treatment related adverse events following administration of CART123 and Ruxolitinib

  • Determine Maximum Tolerated Dose of CART1235 years

    The Maximum Tolerated Dose will be determined by measuring the incidence of dose limiting toxicities following administration of the CART123 product.