CAR T-cell therapy for children with hard-to-treat blood cancers

CAR T-cell Therapy Directed to CD70 for Pediatric Patients With Hematological Malignancies

Phase 1 Interventional St. Jude Children's Research Hospital · NCT06326463

This study is testing a new CAR T-cell therapy to see if it can safely help children and young adults with tough-to-treat blood cancers like leukemia and lymphoma.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment18 (estimated)
AgesN/A to 21 Years
SexAll
SponsorSt. Jude Children's Research Hospital Academic / other
Drugs / interventionschemotherapy, cyclophosphamide, Fludarabine, CAR T
Locations1 site (Memphis, Tennessee)
Trial IDNCT06326463 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and maximum tolerated dose of CD70-CAR T cells in pediatric patients (up to 21 years old) with relapsed or refractory CD70+ hematological malignancies, including acute myelogenous leukemia (AML), acute lymphoblastic leukemia (ALL), and lymphoma. Participants will undergo a lymphodepleting chemotherapy regimen followed by an infusion of autologous CD70-CAR T cells. The study aims to assess both the safety of the treatment and its effectiveness in combating these difficult-to-treat cancers.

Who should consider this trial

Good fit: Ideal candidates are children and young adults aged 21 years or younger with relapsed or refractory CD70+ hematological malignancies.

Not a fit: Patients with hematological malignancies that do not express CD70 or those who are older than 21 years may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could provide a new treatment option for children with resistant forms of blood cancers.

How similar studies have performed: Other studies using CAR T-cell therapies have shown promising results, particularly in treating various forms of leukemia and lymphoma, indicating potential success for this approach.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria

Age ≤21 years old

Relapsed/refractory CD70+ hematological malignancy

Relapsed disease: Patients developing recurrent disease after a prior complete remission (CR)

Refractory disease: Patients with persistent disease despite 3 cycles of induction chemotherapy.

* Relapsed/refractory CD70+ AML or MDS:

  * Relapsed disease that is CD70 positive
  * Refractory disease that is persistent despite 3 cycles of chemotherapy
* Relapsed/refractory CD70+ B-cell ALL:

  * Relapsed disease that is CD70 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
* Relapsed/refractory CD70+ T-cell ALL:

  * Relapsed /refractory disease that is CD70 positive
* Mixed Phenotype Acute Leukemia (MPAL):

  * Relapsed/refractory that is CD70 positive
* Relapsed/refractory CD70+ lymphoma:

  * Relapsed disease that is CD70 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

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 HCT donor

For females of childbearing age:

i. Not lactating with intent to breastfeed

ii. Not pregnant with negative serum or urine pregnancy test within 7 days prior to enrollment

Exclusion Criteria

* Known primary immunodeficiency
* Known history of HIV positivity
* Severe intercurrent bacterial, viral or fungal infection
* History of hypersensitivity to cornstarch or hydroxyethyl starch
* Patients with acute promyelocytic leukemia (APL)
* Known contraindication to protocol defined lymphodepleting
* chemotherapy regimen of Fludarabine/cyclophosphamide

Where this trial is running

Memphis, Tennessee

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Hematologic MalignancyALL, ChildhoodAML, ChildhoodLymphomaMDS
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.