RecruitingPhase 1

PLAT-08: A Study Of SC-DARIC33 CAR T Cells In Pediatric And Young Adults With Relapsed Or Refractory CD33+ AML

Pediatric And Young Adult Leukemia Adoptive Therapy (PLAT)-08: A Phase 1 Study Of SC-DARIC33 In Pediatric And Young Adults With Relapsed Or Refractory CD33+ AML

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 30 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

Not stated in the study description — ask the study team about visits and procedures.

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 early study tests the safety of SC-DARIC33 CAR T cells and whether this treatment can be given. It uses a patient's own immune cells, changed in a lab to fight leukemia. It is for people age 30 or younger with CD33-positive acute myeloid leukemia (AML) that has come back or has not responded to treatment.

A phase 1, open-label, non-randomized study enrolling pediatric and young adult patients with relapsed or refractory CD33+ leukemia with and without prior history of allogeneic hematopoietic cell transplantation, to examine the safety and feasibility of administering an autologous T cell product that has been genetically modified to express a Dimerizing Agent Regulated Immunoreceptor Complex (DARIC).

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 30 or younger. The first 3 patients must be at least 18.
  • Have AML with a marker called CD33. It must have returned after a donor stem cell transplant, returned within 6 months of diagnosis, or returned at least twice. Other qualifying cases are a later first return with more than 0.1% leukemia cells after another treatment attempt, or more than 1% leukemia cells after 2 starting chemotherapy cycles.
  • Be able to have blood cells collected by a machine, or have enough stored cells to make the treatment. People needing collection must meet the study's immune cell count requirement.
  • Be expected to live at least 8 weeks, have a suitable stem cell donor source, and meet the study's daily activity score.
  • If suitable stored cells are not available, stop cancer treatments for the required times and recover from major short-term side effects of past treatment.
  • Meet the study's kidney, liver, heart, and breathing test requirements. Have negative HIV and hepatitis B tests, and meet the hepatitis C testing rules.
  • If able to become pregnant or father a child, agree to use highly effective birth control from consent until 12 months after the cell treatment.
  • The patient or a legal representative must sign the study consent form.

Who can't join

  • Have another active cancer besides AML.
  • Have a history of certain brain or nervous system problems with symptoms, or ongoing problems needing treatment. Some well-controlled seizure disorders are allowed. AML in the brain or spinal cord also excludes patients if it causes symptoms that cannot be controlled before treatment.
  • After a donor stem cell transplant, have active graft-versus-host disease, where donor cells attack the body, or have used medicines to prevent or treat it within 4 weeks.
  • Have a severe active infection, or an immune system disorder you were born with.
  • Have had virus-based treatment before, or be pregnant or breastfeeding.
  • Be unwilling to agree to 15 years of follow-up if the study cell treatment is given.
  • Have a condition the study doctor thinks makes treatment unsuitable, or be unable to tolerate treatment that lowers immune cell levels.
  • Have a medical reason you cannot receive rapamycin.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: 1. Subject age ≤ 30 years. The first three enrolled subjects must be ≥ 18 years of age. 2. AML that expresses CD33 by flow cytometry and meets one of the below definitions: 1. For subjects who have previously received an allogeneic HCT, any evidence of AML re-emergence post HCT detectable by flow cytometry 2. First relapse of AML ≤ 6 months of initial diagnosis 3. First relapse of AML \> 6 months after initial diagnosis, with MRD of \>0.1% by flow cytometry (MPF) after at least one re-induction (single cycle) attempt 4. Second or greater relapse AML 5. Refractory AML, defined as \>1% leukemic cells determined by flow cytometry after 2 cycles of induction chemotherapy 3. Able to tolerate apheresis, or subject with sufficient existing apheresis product or T cells for manufacturing investigational product. 4. Life expectancy ≥ 8 weeks 5. Has an appropriate stem cell donor source identified 6. Lansky performance status score of ≥ 50 for subjects \<16 years of age or Karnofsky score ≥ 50 for subjects ≥ 16 years. Subjects who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for purposes of assessing performance status 7. If a subject does not have a previously obtained apheresis product that is acceptable and available for manufacturing of DARIC T cells, the subject must discontinue all anticancer agents and radiotherapy and, in the opinion of the investigator, have fully recovered from significant acute toxic effects of all prior chemotherapy, immunotherapy, and radiotherapy: a. Chemotherapy and biologic agents: All chemotherapy and biologic therapy not specifically mentioned below must be discontinued ≥ 7 days prior to enrollment, with the exception of intrathecal chemotherapy for which there is not a required washout period b. Must be ≥ 30 days from last gemtuzumab ozogamicin dose. c. Steroid use: All corticosteroid therapy (unless physiologic replacement dosing) must be discontinued ≥ 7 days prior to enrollment d. Tyrosine Kinase Inhibitor (TKI) use: All TKIs must be discontinued ≥ 3 days prior to enrollment e. Hydroxyurea: must be discontinued ≥ 1 day prior to enrollment. f. Gene Modified cellular therapy: i. must be at least 30 days from most recent gene modified cell therapy infusion and document no evidence of modified cells in the peripheral blood OR ii. must be at least 60 days from most recent gene modified cell therapy 8. Adequate organ function as indicated by: 1. Renal: Serum creatinine ≤ 1.5 X the upper limit of normal (ULN) 2. Hepatic: Total bilirubin ≤ 3 times ULN for age OR conjugated bilirubin ≤ 2 mg/dL AND ALT (SGPT) ≤ 5 times ULN 3. Cardiac: Shortening fraction ≥ 28% OR ejection fraction ≥ 50% as measured by echocardiogram 4. Respiratory: Oxygen saturation ≥ 92% on room air without supplemental oxygen or mechanical ventilation 9. Laboratory values meet the following criteria: a. Subjects requiring apheresis: Absolute Lymphocyte Count (ALC) ≥ 100 cells/uL b. Virology Testing negative within 3 months prior to enrollment, to include: i. HIV antigen \& antibody ii. Hepatitis B surface antigen iii. Hepatitis C antibody OR if positive, Hepatitis C PCR is negative 10. If subject is of childbearing or child-fathering potential, must agree to use highly effective contraception from the time of initial consent through 12 months following the infusion of investigational product on this trial. 11. Subject and/or legally authorized representative has signed the Informed Consent Form for this study Exclusion Criteria: 1. Active malignancy other than acute myeloid leukemia 2. History of symptomatic non-AML CNS disease or ongoing symptomatic CNS disease requiring medical intervention, including paresis, aphasia, cerebrovascular ischemia/hemorrhage, severe brain injury, dementia, cerebellar disease, organic brain syndrome, psychosis, coordination or movement disorder (subjects with non-febrile seizure disorder controlled on anti-epileptic medication and without seizure activity within 1 month are eligible). 3. CNS AML involvement that is symptomatic and in the opinion of the investigator, cannot be controlled during the interval between enrollment and DARIC T cell infusion 4. If history of allogeneic stem cell transplant: active GVHD, or receiving immunosuppressive therapy for treatment or prevention of GVHD within 4 weeks prior to enrollment 5. Presence of active severe infection, defined as: i. positive blood culture within 48 hours of enrollment, OR ii. fever above 38.2° C, AND clinical signs of infection within 48 hours of enrollment 6. Primary immunodeficiency syndrome 7. Subject has received prior virotherapy 8. Pregnant or breastfeeding 9. Subject and/or legally authorized representative unwilling to provide consent/assent for participation in the 15-year follow-up period, required if DARIC T cell therapy is administered 10. Presence of any condition that, in the opinion of the investigator, would prohibit the subject from undergoing treatment under this protocol 11. Considered by the investigator to be unable to tolerate a lymphodepleting regimen 12. Subject has a contraindication to receiving rapamycin

Study sites (1)

WA