Clinical trial NCT06579469: Prospective Evaluation Of Delayed Effects Of Pediatric Car T Cell Therapy
Official title: Prospective Evaluation Of Delayed Effects Of Pediatric Car T Cell Therapy (PROSPER)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: B-ALL
Hematologic Malignancy
Solid Tumor
Keywords: CAR T cell infusion
Late Effects
Lead sponsor: St. Jude Children's Research Hospital
Sponsor class: OTHER
Collaborators: name: Rally Foundation for Childhood Cancer Research
First posted: 2024-08-30
Enrollment: 100
Sex: ALL
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants who have received CAR T cell therapy

Brief summary: 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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Presence of bone marrow dysfunction (BMD)
timeFrame: Within 6 months post CAR T-cell therapy
measure: Occurrence of clinically significant infections
timeFrame: Within 6 months post CAR T-cell therapy
measure: Presence of persistent ICANS
timeFrame: Within 6 months post CAR T-cell therapy
Secondary outcomes:
measure: Presence of bone marrow dysfunction (BMD)
timeFrame: Within 24 months post CAR T-cell therapy
measure: Severity of BMD
timeFrame: Within 24 months post CAR T-cell therapy
measure: Occurrence of clinically significant infections
timeFrame: Within 24 months post CAR T-cell therapy
measure: Time to the earliest clinically significant infection
timeFrame: Within 24 months post CAR T-cell therapy
measure: Severity of clinically significant infections
timeFrame: Within 24 months post CAR T-cell therapy
measure: Presence of persistent ICANS
timeFrame: Within 24 months post CAR T-cell therapy
measure: Severity of persistent ICANS
timeFrame: Within 24 months post CAR T-cell therapy
Other outcomes:
Locations:
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
Study officials:
affiliation: St. Jude Children's Research Hospital
name: Rebecca Epperly, MD
role: PRINCIPAL_INVESTIGATOR
References: