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

January 22, 2026 updated by: St. Jude Children's Research Hospital

The study participant has one of the following blood cancers: acute myelogenous leukemia (AML)/myelodysplastic syndrome (MDS), acute lymphoblastic leukemia (B-ALL, T-ALL) or Lymphoma. Your cancer has been difficult to treat (refractory) or has come back after treatment (relapse).

Primary Objective

To determine the safety and maximum tolerated dose of intravenous infusions of escalating doses of CD70-CAR T cells in patients (≤21 years) with recurrent/refractory CD70+ hematological malignancies after lymphodepleting chemotherapy.

Secondary Objectives

To evaluate the antileukemic activity of CD70-CAR T cells. We will determine the anti- leukemic activity of the CD70-CAR T cells in the bone marrow and in the treatment of extramedullary disease.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

The primary interventions are a lymphodepleting chemotherapy regimen (fludarabine and cyclophosphamide), followed by a single autologous infusion of CD70-CAR T cells.

Phase I study evaluating three (3) dose levels of CD70-CAR T cells.

Study Type

Interventional

Enrollment (Estimated)

18

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • Recruiting
        • St. Jude Children's Research Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: CD70- CAR T cell Therapy
Patients will receive autologous (their own) cells.
Drug: Fludarabine
40mg/m2, Day -4, -3 and -2
Other Names:
  • Fludara
Drug: Cyclophosphamide

Day -3 and Day-2

REST DAY, -1

Other Names:
  • Cytoxan
Drug: CD70-CAR T cell infusion (Autologous)
Day 0 or +1
Drug: Mesna
Mesna is generally dosed at approximately 25% of the cyclophosphamide dose. It is generally given intravenously prior to and again at 3, 6 and 9 hours following each dose of cyclophosphamide
Other Names:
  • Mesnex

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Maximum tolerated dose of CD70-CAR T cells
Time Frame: 28 days after CD70-CAR T-cell infusion
Phase I design to determine the maximum tolerated dose (MTD) of autologous, CD70-CAR T cells. Three (3) dose levels will be evaluated (1x10e6, 3x10e6, and 1x10e7 cells/kg).
28 days after CD70-CAR T-cell infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

St. Jude Children's Research Hospital

Investigators

  • Principal Investigator: Swati Naik, MBBS, St. Jude

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 16, 2024

Primary Completion (Estimated)

July 1, 2030

Study Completion (Estimated)

July 1, 2031

Study Registration Dates

First Submitted

March 15, 2024

First Submitted That Met QC Criteria

March 15, 2024

First Posted (Actual)

March 22, 2024

Study Record Updates

Last Update Posted (Actual)

January 23, 2026

Last Update Submitted That Met QC Criteria

January 22, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DIRECT70
  • NCI-2024-03240 (Other Identifier: NCI Clinical Trial Registration Program)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.

IPD Sharing Time Frame

Data will be made available at the time of article publication.

IPD Sharing Access Criteria

Data will be provided to researchers following a formal request with the following information: full name of requestor, affiliation, data set requested, and timing of when data is needed. As an informational point, the lead statistician and study principal investigator will be informed that primary results datasets have been requested.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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