Prospective Evaluation Of Delayed Effects Of Pediatric Car T Cell Therapy

June 16, 2026 updated by: St. Jude Children's Research Hospital

Prospective Evaluation Of Delayed Effects Of Pediatric Car T Cell Therapy (PROSPER)

This study is being done to learn more about the short-term and long-term side effects of CAR-T cell therapy. Specifically, researchers want to know how often patients get infections, have delays in recovering blood cell counts and/or have damage to the nervous system.

Study Overview

Status

Recruiting

Conditions

Detailed Description

Primary Objectives

  • Bone Marrow Function: To report on the incidence, timing, severity of, and risk factors for bone marrow dysfunction in participants in remission or without bone marrow involvement of disease at 3- and 6-months following CAR T cell therapy. (B-ALL cohort)
  • Infection/Immune Reconstitution: To evaluate the incidence, timing, severity of and risk factors for clinically significant infections following CAR T cell therapy at 3- and 6-months following CAR T cell therapy. (B-ALL cohort)
  • Neurotoxicity: To evaluate the incidence, timing, severity of, and risk factors for persistent ICANS at 3- and 6-months post CAR T cell therapy. (B-ALL cohort)

Secondary Objectives

  • To evaluate bone marrow function, infection/immune reconstitution, and neurotoxicity at 12 months and 24 months post CAR T cell therapy in participants with B-ALL.
  • To characterize bone marrow function, infection/immune reconstitution, and neurotoxicity between 3 and 24 months after CAR T cell therapy in other hematologic malignancies and solid tumor cohorts.

Participants will have an assessment of preexisting morbidity and potential risk factors, collection of specimens for banking, scheduled late effects monitoring, laboratory analysis, and screening studies. Data and biospecimens will be collected at 3 months, 6 months, 1 year and 2 years after CAR T cell infusion.

Study Type

Observational

Enrollment (Estimated)

100

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
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • Childrens Hospital of Colorado
        • Contact:
        • Principal Investigator:
          • Hesham Eissa, MD, MSc
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia
        • Contact:
        • Principal Investigator:
          • Regina Myers, MD
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • Recruiting
        • St. Jude Children's Research Hospital
        • Principal Investigator:
          • Rebecca Epperly, MD
        • Contact:
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Recruiting
        • Primary Children's Hospital
        • Contact:
        • Principal Investigator:
          • Joseph G Dolan, MD
    • Washington
      • Seattle, Washington, United States, 98105
        • Recruiting
        • Seattle Childrens Hospital
        • Contact:
        • Principal Investigator:
          • Mallory Taylor, MD
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Children's Hospital of Wisconsin.
        • Contact:
        • Principal Investigator:
          • Amy Moskop, MD

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

Sampling Method

Non-Probability Sample

Study Population

Participants who have received CAR T cell therapy

Description

Inclusion Criteria:

  • Participants must have received an initial systemically-administered CAR T cell infusion within the last 1-3 months (+/- 14 days).

    • Initial infusion is defined as the first administration of a CAR T cell product the participant has not previously received OR receipt of a CAR T cell product previously received after an interval allogeneic HSCT.
  • Age ≤ 30 years at CAR T cell infusion.

Exclusion Criteria:

  • Active malignancy other than the disease under study.
  • Planned consolidative HSCT within 3 months post CAR T cell infusion.
  • Received or planned additional disease directed therapy post CAR T cell infusion.
  • Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

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

Cohorts and Interventions

Group / Cohort
B cell acute lymphoblastic leukemia (B cell acute lymphoblastic leukemia (B-ALL) ) cohort
B-ALL participants who have received initial CAR T cell therapy within the last 1-3 months.
Other hematologic malignancy
Other hematologic malignancy participants who have received initial CAR T cell therapy within the last 1-3 months.
Solid tumor (ST)
ST participants who have received initial CAR T cell therapy within the last 1-3 months.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Presence of bone marrow dysfunction (BMD)
Time Frame: Within 6 months post CAR T-cell therapy
Among patients in remission or without bone marrow involvement of disease in the B-ALL cohort, we will summarize the rates of prevalent BMD at 3- and 6-months post-infusion and estimate the cumulative incidence of new BMD and BMD recovery for patients with prevalent BMD at 3- and 6-months.
Within 6 months post CAR T-cell therapy
Occurrence of clinically significant infections
Time Frame: Within 6 months post CAR T-cell therapy
The infection density of clinically significant infections in 3-6 months will be summarized in the B-ALL cohort.
Within 6 months post CAR T-cell therapy
Presence of persistent ICANS
Time Frame: Within 6 months post CAR T-cell therapy
We will summarize the rates of persistent ICANS at 3- and 6-months post-infusion and estimate the cumulative incidence and timing of new ICANS and ICANS recovery for patients with persistent ICANS at 3- and 6-months in the B-ALL cohort.
Within 6 months post CAR T-cell therapy

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Presence of bone marrow dysfunction (BMD)
Time Frame: Within 24 months post CAR T-cell therapy
Among patients in remission or without bone marrow involvement of disease in the B-ALL cohort, we will summarize the rates of prevalent BMD at 12- and 24-months post-infusion and estimate the cumulative incidence of new BMD and BMD recovery for patients with prevalent BMD at 12- and 24-months. Analyses will be replicated in the other hematologic malignancies and solid tumor cohorts.
Within 24 months post CAR T-cell therapy
Severity of BMD
Time Frame: Within 24 months post CAR T-cell therapy
The highest severity BMD for each patient will be recorded. The severity of BMD in the B-ALL cohort will be described using descriptive statistics. Analyses will be replicated in the other hematologic malignancies and solid tumor cohorts.
Within 24 months post CAR T-cell therapy
Occurrence of clinically significant infections
Time Frame: Within 24 months post CAR T-cell therapy
The infection density of clinically significant infections within 24 months will be summarized in the B-ALL cohort. Analyses will be replicated in the other hematologic malignancies and solid tumor cohorts.
Within 24 months post CAR T-cell therapy
Time to the earliest clinically significant infection
Time Frame: Within 24 months post CAR T-cell therapy
The time from 3 months post-infusion to the first clinically significant post-infusion within 24 months post-infusion will be summarized in the B-ALL cohort. The cumulative incidence of the first clinically significant infection post-infusion within 24 months will be estimated in B-ALL cohort. Analyses will be replicated in the other hematologic malignancies and solid tumor cohorts.
Within 24 months post CAR T-cell therapy
Severity of clinically significant infections
Time Frame: Within 24 months post CAR T-cell therapy
The highest severity among all clinically significant infections for each patient will be summarized. The severity of clinically significant infections in the B-ALL cohort will be described. Analyses will be replicated in the other hematologic malignancies and solid tumor cohorts.
Within 24 months post CAR T-cell therapy
Presence of persistent ICANS
Time Frame: Within 24 months post CAR T-cell therapy
We will summarize the rates of persistent ICANS at 12- and 24-months post-infusion and estimate the cumulative incidence and timing of new ICANS and ICANS recovery for patients with persistent ICANS at 12- and 24-months in B-ALL cohort. Analyses will be replicated in the other hematologic malignancies and solid tumor cohorts.
Within 24 months post CAR T-cell therapy
Severity of persistent ICANS
Time Frame: Within 24 months post CAR T-cell therapy
The highest severity of ICANS for each patient will be recorded. The severity of ICANS in the B-ALL cohort will be described using descriptive statistics. Analyses will be replicated in the other hematologic malignancies and solid tumor cohorts.
Within 24 months post CAR T-cell therapy

Collaborators and Investigators

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

Sponsor

St. Jude Children's Research Hospital

Collaborators

Rally Foundation for Childhood Cancer Research

Investigators

  • Principal Investigator: Rebecca Epperly, MD, St. Jude Children's Research Hospital

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)

January 20, 2026

Primary Completion (Estimated)

March 1, 2027

Study Completion (Estimated)

March 1, 2029

Study Registration Dates

First Submitted

August 28, 2024

First Submitted That Met QC Criteria

August 28, 2024

First Posted (Actual)

August 30, 2024

Study Record Updates

Last Update Posted (Actual)

June 18, 2026

Last Update Submitted That Met QC Criteria

June 16, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PROSPER
  • NCI-2024-06836 (Registry Identifier: NCI Clinical Trial Registration Program)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

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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