CAR-Multicenter Analysis (CAR-MA): Retrospective Study to Characterize CAR T-cell Outcomes and Related Toxicities in Children and Young Adults With B-ALL

NCT05440409 | Completed

• 2 other identifiers: 10000651000651-C
• Study Type: observational
• Target: 57
• Locations: 1 country
• Sites: 1

Brief Summary

Study Description: This retrospective protocol focuses on characterizing clinical outcomes and toxicities following CAR T-cell therapy. Objectives: Primary To evaluate the Response Free Survival (RFS) at 6 months following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab. Completed To retrospectively evaluate outcomes following CAR T-cell therapy across children and young adults with B-ALL Secondary To evaluate the RFS at 12 months following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab and other immunotherapy. Completed To evaluate the incidence of CD19 negative versus CD19 positive relapse following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab. Completed To evaluate the Complete Response (CR) rate following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab. Completed To evaluate the Minimal Residual Disease (MRD) negative remission rate following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab. Completed To evaluate the impact of prior blinatumomab, prior inotuzumab, and other immunotherapies on subsequent CAR T cell outcomes. To determine the incidence, severity, resolution and risk factors for early and late cytopenias after CAR T-cell therapy. To evaluate the response of extramedullary disease following CAR T-cell therapy. To evaluate the frequency of subsequent malignant neoplasms after CAR T-cell therapy. To describe response, survival, and toxicities after CAR T-cell therapy in children with trisomy 21 and other important subpopulations. To determine the impact of next-generation sequencing MRD results and duration of B-cell aplasia on CAR T cell outcomes. To evaluate the frequency of infections and describe immune system function after CAR T-cell therapy. To describe response, survival, and toxicities after CAR T-cell therapy in children with leukemia containing specific cytogenetic lesions (e.gl. TP53, t(1;19), hypodiploidy). To compare toxicities and outcomes across CAR T-cell constructs (e.g., different CD19 CAR constructs, dual-targeted CARs, CD22-targeted CARs, etc.). To assess the impact of CAR T cells on other health-related outcomes, including, but not limited to, organ function, immune reconstitution, quality of life, and patient-reported outcomes. Study Population and Source of Data: Subjects who were less than \< 25 years of age at the time of diagnosis and received a CAR T-cell product for B-ALL.

Conditions

Leukemia, Acute Lymphoblastic; Lymphoblastic Leukemia, Acute, Childhood

Keywords

Relapsed Disease; MRD-positive CD19+ ALL; Natural History

Outcome Measures

Primary Outcomes (2)

• Response free survival (completed)

  To evaluate the RFS at 6 months following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab.

  6 months

• Outcome evaluation

  To retrospectively evaluate outcomes following CAR T-cell therapy across children and young adults with B-ALL

  12 months

Secondary Outcomes (14)

• Complete Response Rate (completed)

  12 months

• Relapse Rate (completed)

  12 months

• Response free survival (completed)

  12 months

• Minimal Residual Disease detection (completed)

  12 months

• Impact of immunotherapies on CAR outcomes

  12 months

Study Arms (1)

1

Retrospective chart review of children and adults with cancer enrolled on immunotherapy treatment protocols

Eligibility Criteria

• Age: 3 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

Subjects who were less than or equal to 25 years of age at the time of diagnosis and received a CAR T-cell product for B-ALL. This protocol will be amended to incorporate new research objectives and new data as necessary.

* Subjects will not be recruited for this study; however, up to 210 subjects records will be selected from treatment protocols who received CAR therapy for B-ALL. Subject who opted out of the future use of his/her data will be excluded. The subjects enrolled to a CAR T cell therapy treatment protocol within the Pediatric Oncology Branch unless, are \< 25 years of age at the time of diagnosis and must have received prior a CAR T-cell product.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (1)

National Cancer Institute (NCI)

Bethesda, Maryland, 20892, United States

Location

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma; Recurrence

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Sara K Silbert, M.D.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 29, 2022
• First Posted: June 30, 2022
• Study Start: October 14, 2021
• Primary Completion: October 29, 2024
• Study Completion: October 29, 2024
• Last Updated: June 25, 2026

Record last verified: 2026-06-23

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Clinical data available during the study and indefinitely.
• Access Criteria: Clinical data will be made available via subscription to BTRIS and with the permission of the study PI.

Locations

US (1)