Evaluating blood tests to monitor recovery after CAR T-cell therapy in young patients with leukemia
A Pilot Trial to Evaluate Next-Generation Sequencing (NGS) Testing and Monitoring of B-Cell Recovery to Guide Management Following CAR T-cell Induced Remission in Pediatric Patients With B Lineage Acute Lymphoblastic Leukemia
This study is testing new blood tests to help doctors keep an eye on young patients with leukemia after their CAR T-cell therapy to see who might need extra treatment and who can stay healthy without it.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 1 Year to 25 Years |
| Sex | All |
| Sponsor | National Institutes of Health Clinical Center (CC) NIH |
| Drugs / interventions | CART, Chimeric antigen receptor, immunotherapy |
| Locations | 8 sites (Los Angeles, California and 7 other locations) |
| Trial ID | NCT05621291 on ClinicalTrials.gov |
What this trial studies
This study aims to improve outcomes for children and young adults with B-lineage acute lymphoblastic leukemia (B-ALL) who have undergone chimeric antigen receptor T-cell (CART) therapy. It will utilize next-generation sequencing (NGS) testing and regular blood and bone marrow tests to monitor for potential relapse after CART treatment. The goal is to identify patients at high risk of relapse who may benefit from a stem cell transplant, while also distinguishing those who can maintain remission without additional interventions. Participants will be closely monitored for their disease status within 42 days post-CART infusion.
Who should consider this trial
Good fit: Ideal candidates are children and young adults aged 1 to 25 years who have received CART therapy for B-ALL and are currently in remission.
Not a fit: Patients who have previously undergone a stem cell transplant or those who are not in remission after CART therapy may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could lead to better management of B-ALL, reducing the need for risky stem cell transplants in patients who can maintain remission.
How similar studies have performed: Other studies have shown promise in using biomarker-guided strategies for monitoring remission in leukemia, making this approach both relevant and potentially impactful.
Eligibility criteria
Show full inclusion / exclusion criteria
* INCLUSION CRITERIA: * Age \>=1 year and \<= 25 years old at the time of CD19 CART infusion * Confirmed diagnosis of CD19+ B-ALL with an informative NGS clonality sample --Have an informative NGS clonality sample for MRD assessment based on immunoglobulin rearrangement in bone marrow or blood at any time of active disease between diagnosis and CD19 CART infusion and any time prior to the first on-study intervention confirmed by NGS MRD testing. * Post-CD19 CART infusion disease status: * Are in bone marrow morphologic complete remission and are flow cytometry measurable residual disease (MRD) negative within 42 days post CD19 CART infusion. * Are NGS MRD negative by tracking sample in the bone marrow within 42 days post CD19 CART infusion confirmed by NGS MRD testing. * Received first CD19 (4-1BB) CART within 42 days prior to enrollment. Note: Eligible CART including FDA approved Kymriah (tisagenlecleucel) infused on a treatment plan, research study, or other comparable 4-1BB based constructs. Study chairs will determine whether other 4-1BB CART are considered comparable. * All participants must have an allogeneic HCT donor identified for potential HCT. Note: Donor identification and selection will be according to institutional practice. * Have B-cell aplasia (BCA) post CD19 CART persisting within 42 days post CD19 CART infusion. Note: BCA persisting is defined as \<1% B cells lymphocytes or \<50 B cells/microliter in the peripheral blood * Performance of all screening tests prior to day 42 post CD19 CART. * The ability of participant or parent/guardian to understand and the willingness to sign a written consent document or participants unable to consent if they are represented by a Legally Authorized Representative (LAR). EXCLUSION CRITERIA: * Prior hematopoietic stem cell transplantation (HCT) * Recent history of the extramedullary disease (EMD) that requires ongoing radiographic surveillance (e.g., participants with active EMD at CD19 CART infusion that requires monitoring by imaging without the ability to more precisely assess disease status will be ineligible). A remote history of EMD does not exclude the participant. * Active and/or residual central nervous system (CNS) disease that requires ongoing therapy or monitoring. * Co-morbidities precluding myeloablative HCT. Note: Determination of co-morbidities precluding myeloablative HCT will be made by the treating transplant (HCT) physician and documented in the research record. This does not require that the participant is immediately fully eligible for HCT, only that there are no long-term comorbidities that would preclude a myeloablative approach (e.g., renal failure, severe cardiac failure, long-term oxygen requirement). * Uncontrolled, symptomatic, intercurrent illness or social situations that would limit compliance with study requirements. Note: Determination of uncontrolled, symptomatic illness or social situation that would limit compliance with the study requirements will be made by the site-PI and documented in the research record.
Where this trial is running
Los Angeles, California and 7 other locations
- Children's Hospital of Los Angeles — Los Angeles, California, United States (Recruiting)
- Children's National Medical Center — Washington D.C., District of Columbia, United States (Recruiting)
- Children's Healthcare of Atlanta — Atlanta, Georgia, United States (Not_yet_recruiting)
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States (Recruiting)
- Dana-Farber/Boston Children s Hospital — Boston, Massachusetts, United States (Not_yet_recruiting)
- Huntsman Cancer Institute, University of Utah — Salt Lake City, Utah, United States (Recruiting)
- Seattle Children's, University of Washington — Seattle, Washington, United States (Recruiting)
- Fred Hutchinson Cancer Research Center — Seattle, Washington, United States (Recruiting)
Study contacts
- Principal investigator: Nirali N Shah, M.D. — National Cancer Institute (NCI)
- Study coordinator: NCI Pediatric Leukemia, Lymphoma Transpl
- Email: ncilltct@mail.nih.gov
- Phone: (240) 760-6970
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.