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

NCT06326463 | Recruiting Phase 1 FDA Drug

• 2 other identifiers: DIRECT70NCI-2024-03240
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• Maximum tolerated dose of CD70-CAR T cells

  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

Study Arms (1)

CD70- CAR T cell Therapy

EXPERIMENTAL

Patients will receive autologous (their own) cells.

Drug: Fludarabine; Drug: Cyclophosphamide; Drug: CD70-CAR T cell infusion (Autologous); Drug: Mesna

Interventions

Fludarabine DRUG

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

Also known as: Fludara

CD70- CAR T cell Therapy

Cyclophosphamide DRUG

Day -3 and Day-2 REST DAY, -1

Also known as: Cytoxan

CD70- CAR T cell Therapy

CD70-CAR T cell infusion (Autologous) DRUG

Day 0 or +1

CD70- CAR T cell Therapy

Mesna 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

Also known as: Mesnex

CD70- CAR T cell Therapy

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

RECRUITING

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

MeSH Terms

Conditions

Hematologic Neoplasms; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Myeloid, Acute; Lymphoma

Interventions

fludarabine; fludarabine phosphate; Cyclophosphamide; Mesna

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Leukemia, Myeloid

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Sulfhydryl Compounds; Sulfur Compounds; Sulfonic Acids; Sulfur Acids

Study Officials

• Swati Naik, MBBS

  St. Jude

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Swati Naik, MBBS

CONTACT

866-278-5833; referralinfo@stjude.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 15, 2024
• First Posted: March 22, 2024
• Study Start: October 16, 2024
• Primary Completion (Estimated): July 1, 2030
• Study Completion (Estimated): July 1, 2031
• Last Updated: January 23, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data will be made available at the time of article publication.
• 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.

Locations

US (1)