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

NCT ID: NCT06326463

Last Updated: 2026-01-23

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 18 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-10-16
• Study Completion Date: 2031-07-01

Brief Summary

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.

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.

Conditions

Hematologic Malignancy; ALL, Childhood; AML, Childhood; Lymphoma; MDS

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

CD70- CAR T cell Therapy

Patients will receive autologous (their own) cells.

Group Type: EXPERIMENTAL

Fludarabine

Intervention Type: DRUG

40mg/m2, Day -4, -3 and -2

Cyclophosphamide

Intervention Type: DRUG

Day -3 and Day-2

REST DAY, -1

CD70-CAR T cell infusion (Autologous)

Intervention Type: DRUG

Day 0 or +1

Mesna

Intervention Type: DRUG

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

Interventions

Fludarabine

40mg/m2, Day -4, -3 and -2

Intervention Type: DRUG

Cyclophosphamide

Day -3 and Day-2

REST DAY, -1

Intervention Type: DRUG

CD70-CAR T cell infusion (Autologous)

Day 0 or +1

Intervention Type: 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

Intervention Type: DRUG

Other Intervention Names

Fludara; Cytoxan; Mesnex

Eligibility 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

• Maximum Eligible Age: 21 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

St. Jude Children's Research Hospital

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Swati Naik, MBBS

Role: PRINCIPAL_INVESTIGATOR

St. Jude

Locations

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Swati Naik, MBBS

Role: CONTACT

referralinfo@stjude.org

866-278-5833

Facility Contacts

Swati Naik, MD

Role: primary

referralinfo@stjude.org

866-278-5833

Related Links

http://www.stjude.org

St. Jude Children's Research Hospital

http://www.stjude.org/protocols

Clinical Trials Open at St. Jude

Other Identifiers

NCI-2024-03240

Identifier Type: OTHER

Identifier Source: secondary_id

DIRECT70

Identifier Type: -

Identifier Source: org_study_id