CART123 Cells With or Without Ruxolitinib for Relapsed/Refractory AML

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a phase 1 trial at Children’s Hospital of Philadelphia evaluating the safety and potential effectiveness of CAR T cells targeting CD123 (CART123) in pediatric and young adult patients with relapsed or refractory AML. Participants are assigned to two cohorts: CART123 alone (Cohort A) and CART123 with the JAK1/2 inhibitor ruxolitinib (Cohort B). Cohort A uses escalating CART123 doses after lymphodepletion with fludarabine and cyclophosphamide, while Cohort B uses a fixed CART123 dose with enhanced lymphodepletion and planned ruxolitinib dosing. The study involves outpatient CART infusions and careful post‑treatment follow‑up, enrolling roughly 18 patients in Cohort A and 12 in Cohort B. Primary goals center on safety and feasibility, with exploratory signals of efficacy monitored in both groups.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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:

  1. Second or greater relapse defined as bone marrow flow cytometric confirmation of myeloid leukemia of at least 0.1% or development of extramedullary disease by imaging or biopsy after second documented complete remission; OR
  2. Any detectable disease post-allogeneic transplant with bone marrow flow cytometric confirmation of myeloid leukemia of at least 0.1% or development of extramedullary disease by imaging or biopsy; OR
  3. Refractory disease, defined as: Persistent bone marrow involvement with ≥0.1% disease by flow cytometry or persistent extramedullary disease by imaging or biopsy after two courses of induction chemotherapy for patients at initial presentation, ≥ 0.1% bone marrow disease by flow cytometry or persistent extramedullary disease by imaging or biopsy after one course of induction chemotherapy for patients who have relapsed after previously achieving a CR , and ≥ 0.1% bone marrow disease by flow cytometry or persistent extramedullary disease by imaging or biopsy after one course of AML-directed chemotherapy for those with myeloid lineage switch.
* 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:

  1. Serum creatinine based on age/gender.
  2. Adequate liver function: ALT ≤ 500 U/L, Bilirubin ≤3x the upper limit of normal, and ALT and/or bilirubin results that exceed this range are acceptable if, in the opinion of the physician-investigator (or as confirmed by liver biopsy), the abnormalities are directly related to AML infiltration of the liver.
  3. Must have a minimum level of pulmonary reserve defined as ≤Grade 1 dyspnea and \<Grade 3 hypoxia; DLCO ≥ 40% (corrected for anemia if necessary) if PFTs are clinically appropriate as determined by the treating investigator.
  4. Left Ventricular Shortening Fraction (LVSF) ≥ 28% or Ejection Fraction (LVEF) ≥ 45% confirmed by echocardiogram or another scan.
* 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 Criteria:

* 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

Where it's running

• Children's Hospital of Philadelphia
  Philadelphia, Pennsylvania, United States Recruiting

Study contacts

• Principal investigator: Lucy Cain, MBBS — Children's Hospital of Philadelphia
• Study coordinator: Cell Therapy Nurse Navigator
• Email: CARTNurseNavigator@chop.edu
• Phone: 445-942-5891

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.