CD123-Directed T-Cell Therapy for Acute Myelogenous Leukemia (CATCHAML)
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- Up to 21 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Procedures mentioned: Infusions
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "Treatment Schedule: Patients will receive lymphodepleting chemotherapy followed by infusion of CD123-CAR T cells Fludarabine on day -4, -3 and -2 Cyclophosphamide on day -3 and -2 REST DAY on day -1 CD123-CAR T cell infusion on day 0 or +1"
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests the highest safe dose of CD123-CAR T cells, immune cells changed to target cancer cells, after chemotherapy. It is for people age 21 or younger with certain blood cancers that have come back or have not responded to treatment and have a marker called CD123. Researchers will also study side effects and whether the treatment fights the cancer.
The CD123-CAR T-cell therapy is a new treatment that is being investigated for treatment of AML/myelodysplastic syndrome (MDS), T- or B- acute lymphoblastic leukemia (ALL) or blastic plasmacytoid dendritic cell neoplasia (BPDCN). The purpose of this study is to find the maximum (highest) dose of CD123-CAR T cells that is safe to give to these patients. This would include studying the side effects of the chemotherapy, as well as the CD123-CAR T-cell product on the recipient's body, disease and overall survival. Primary Objective: * To determine the safety of one intravenous infusion of escalating doses of autologous, CD123-CAR T cells in patients (≤21 years) with recurrent/refractory CD123+ disease (AML/MDS, B-ALL, T-ALL or BPDCN) after lymphodepleting chemotherapy. * To determine the safety of an intravenous infusion of escalating doses of donor derived, CD123-CAR T cells in patients (≤21 years) with recurrent/refractory CD123+ disease (AML/MDS, B-ALL, T-ALL, BPDCN or MPAL) after lymphodepleting chemotherapy. Secondary Objectives \- To evaluate the antileukemia activity of CD123-CAR T cells. Exploratory Objectives * To assess the immunophenotype, clonal structure and endogenous repertoire of CD123-CAR T cells and unmodified T cells * To characterize the cytokine profile in the peripheral blood and CSF after treatment with CD123-CAR T cells * To characterize tumor cells post CD123-CAR T-cell therapy * To compare in vivo properties of donor-derived versus autologous CD123- CAR T cells
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be age 21 or younger with a blood cancer that meets the study’s rules, such as AML, MDS, B-cell or T-cell ALL, or BPDCN.
- The cancer must have the CD123 marker. Before treatment, tests must still find cancer, even in very small amounts. Extra rules apply to B-cell ALL.
- Be expected to live more than 12 weeks before cell collection and more than 8 weeks before treatment. Meet the study’s score for daily activity.
- Have a suitable stem cell transplant donor. After a past donor transplant, be recovered, have no active donor cells attacking your body, and have no donor immune cell infusion in the past 28 days.
- Be able to have your T cells collected, or have had them collected already. Before treatment, the prepared cells must pass required quality checks.
- Before treatment, meet heart, kidney, lung, and liver test requirements and have recovered from severe side effects of past treatment, other than blood-related effects.
- Not be pregnant or producing breast milk with plans to breastfeed. If able to become pregnant, have a negative blood pregnancy test within 7 days before joining.
- If sexually active, agree to use birth control until 3 months after the T-cell infusion. Male partners must use condoms.
Who can't join
- Have a known immune system disorder you were born with, or a history of HIV infection.
- Have a severe infection that is not controlled.
- Have acute promyelocytic leukemia (APL), a type of leukemia, or active cancer in the brain or spinal cord.
- Have had an allergic reaction to products containing mouse proteins, or a severe allergic reaction to cornstarch or hydroxyethyl starch.
- Cannot safely receive the study’s chemotherapy drugs, fludarabine and cyclophosphamide.
- Have received steroid treatment above the allowed dose within 7 days before the T-cell infusion.
- Have received treatment within 14 days that could stop the study cells from working, or rituximab within 30 days before the infusion.
- Have received chemotherapy into the fluid around the brain and spinal cord within 7 days before the infusion.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria for Procurement and T-cell Production: * Age ≤21 years old * Relapsed/refractory CD123+ disease defined as follows: AML/MDS * Relapsed disease: Patients developing recurrent disease after a first complete remission (CR) * Refractory disease: Patients not achieving a CR after 2 cycles of induction chemotherapy B-cell ALL * Relapsed disease that is CD123 positive and CD19 negative/dim or patients otherwise ineligible for CD19 directed therapies including * Patients in 2nd or greater relapse * Patients with relapse after allogeneic HSCT * Refractory disease that is CD123 positive and CD19 negative/dim or patients otherwise ineligible for CD19 directed therapies T-cell All • Relapsed refractory disease that is CD123 positive BPDCN • Relapsed/refractory disease that has failed front-line therapy * Estimated life expectancy of \>12 weeks * Karnofsky or Lansky (age-dependent) performance score ≥50 * Patients with a history of prior allogeneic HCT must be clinically recovered from prior HCT therapy, have no evidence of active GVHD and have not received a donor lymphocyte infusion (DLI) within the 28 days prior to apheresis * Patient must have an identified, suitable HCT donor * For females of child-bearing age: * Not lactating with intent to breastfeed * Not pregnant with negative serum pregnancy test within 7 days prior to enrollment * Meets eligibility criteria to undergo autologous apheresis, or have previously undergone autologous apheresis Exclusion Criteria: * Known primary immunodeficiency * History of HIV infection * Severe intercurrent uncontrolled bacterial, viral or fungal infection (e.g. active hepatitis B or C infection or adenovirus infection) * History of hypersensitivity reactions to murine protein-containing products * Patients with acute promyelocytic leukemia (APL, t (15;17)) * Known contraindication to the protocol defined lymphodepleting chemotherapy regimen of fludarabine/cyclophosphamide. Inclusion Criteria for Treatment: * Age≤21 years old * Detectable disease that is CD123+ (at least MRD+ disease) * Estimated life expectancy of \>8 weeks * Karnofsky or Lansky (age-dependent) performance score≥50 * Patients with a history of prior allogeneic HCT must be clinically recovered from prior HCT therapy, have no evidence of active GVHD and have not received a donor lymphocyte infusion (DLI) within the 28 days prior to planned infusion * Patient must have an identified, suitable HCT donor * Adequate cardiac function defined as left ventricular ejection fraction \>40%, OR shortening fraction ≥25% * EKG without evidence of clinically significant arrhythmia * Adequate renal function defined as creatinine clearance or radioisotope GFR ≥50 ml/min/1.73m2 (GFR ≥40 ml/min/1.73m2 if \< 2 years of age) * Adequate pulmonary function defined as forced vital capacity (FVC)≥50% of predicted value; or pulse oximetry≥92% on room air if patient is unable to perform pulmonary function testing * Total Bilirubin≤3 times the upper limit of normal for age, except in subjects with Gilbert's syndrome * Alanine aminotransferase (ALT) OR aspartate aminotransferase (AST) ≤5 times the upper limit of normal for age * Has recovered from all NCI CTAE grade III-IV, non-hematologic acute toxicities from prior therapy * For females of child-bearing age * Not lactating with intent to breastfeed * Not pregnant with negative serum pregnancy test within 7 days prior to enrollment * If sexually active, agreement to use birth control until 3 months after T- cell infusion. Male partners should use a condom. * Available autologous transduced T-cell product that has met GMP release criteria Exclusion Criteria: * Known primary immunodeficiency * History of HIV infection * Severe intercurrent uncontrolled bacterial, viral or fungal infection * History of hypersensitivity reactions to murine protein-containing products * History of severe hypersensitivity reactions to cornstarch or hydroxyethyl starch. * Receiving systemic steroids therapy exceeding the equivalent of 0.5 mg/kg/day of methylprednisolone, in the 7 days prior to CD123-CAR T- cell infusion * Receiving systemic therapy in the 14 days prior to CD123-CAR T-cell infusion, which will interfere with the activity of the CD123-CAR T cells in vivo (in the opinion of the study PI(s)) * Receiving rituximab therapy in the 30 days prior to CD123-CAR T cell infusion. (This exclusion criterion is intended to prevent premature exposure of CD123-CAR T cells to rituximab, which would activate the safety switch and promote CAR T-cell apoptosis). * Receiving intrathecal chemotherapy in the 7 days prior to CD123-CAR T cell infusion. * Known contraindication to the protocol defined lymphodepleting chemotherapy regimen of fludarabine/cyclophosphamide. * Active CNS disease
Study sites (2)
TN
- St Jude Children's Research Hospital · Memphis, TN 38105Swati Naik, MD · Study contact · 888-226-4343 · referralinfo@stjude.orgSwati Naik, MD · Principal investigatorMore trials in Memphis
- St. Jude Children's Research Hospital · Memphis, TN 38105Paulina Velasquez, MD · Study contact · 888-226-4343 · referralinfo@stjude.orgPaulina Velasquez, MD · Principal investigatorMore trials in Memphis