Prospective Evaluation Of Delayed Effects Of Pediatric Car T Cell Therapy

St. Jude Children's Research Hospital

Status: Enrolling

Conditions: Solid Tumor; Hematologic Malignancy; B-ALL

Study type: Observational

Funder types: Other

Identifiers

NCT06579469

PROSPER

NCI-2024-06836 (Registry Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 100 estimated patients

Sex: All

Ages: Under 30 years old

Volunteers: No Healthy Volunteers

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.

Trial design

100 participants in 3 patient groups

B cell acute lymphoblastic leukemia (B cell acute lymphoblastic leukemia (B-ALL) ) cohort

Description:

B-ALL participants who have received initial CAR T cell therapy within the last 1-3 months.

Other hematologic malignancy

Description:

Other hematologic malignancy participants who have received initial CAR T cell therapy within the last 1-3 months.

Solid tumor (ST)

Description:

ST participants who have received initial CAR T cell therapy within the last 1-3 months.