Leukemia, Acute LymphoblasticNCT05440409
CAR-Multicenter Analysis (CAR-MA): Retrospective Study to Characterize CAR T-cell Outcomes and Related Toxicities in Children and Young Adults With B-ALL
An observational study of Leukemia, Acute Lymphoblastic and Lymphoblastic Leukemia, Acute, Childhood, run by National Cancer Institute (NCI) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 25 years
- Conditions the study is for
- Leukemia, Acute Lymphoblastic, Lymphoblastic Leukemia, Acute, Childhood
- How many people
- 57 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Oct 2021to 29 Oct 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Cancer Institute (NCI)
Bethesda, Maryland, 20892
About this study
Study Description:
This retrospective protocol focuses on characterizing clinical outcomes and toxicities following CAR T-cell therapy.
Read more
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.
Read the detailed description
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 Tcell 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 Tcell 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.
- Keywords
- Relapsed Disease, MRD-positive CD19+ ALL, Natural History
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Retrospective chart review of children and adults with cancer enrolled on immunotherapy treatment protocols |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response free survival (completed)
To evaluate the RFS at 6 months following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab.
Measured: 6 months
Outcome evaluation
To retrospectively evaluate outcomes following CAR T-cell therapy across children and young adults with B-ALL
Measured: 12 months
Secondary outcomes (14)
Complete Response Rate (completed)
To evaluate the CR rate following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab
Measured: 12 months
Relapse Rate (completed)
To evaluate the incidence of CD19 negative versus CD19 positive relapse following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab
Measured: 12 months
Response free survival (completed)
To evaluate the RFS at 12 months following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab
Measured: 12 months
Minimal Residual Disease detection (completed)
To evaluate the MRD negative remission rate following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab
Measured: 12 months
Impact of immunotherapies on CAR outcomes
To evaluate the impact of prior blinatumomab, prior inotuzumab, and other immunotherapies on subsequent CAR T cell outcomes
Measured: 12 months
Cytopenias after CAR
To determine the incidence, severity, resolution and risk factors for early and late cytopenias after CAR T cell outcomes
Measured: 12 months
Response of extramedullary disease
To evlauate the response of extramedullary disease following CAR T cell therapy
Measured: 12 months
Frequency of malignant neoplasms
To evaluate the response of extramedullary disease following CAR T cell therapy
Measured: 12 months
Response, survival and toxicities in subpopulations
To describe response, survival, and toxicities after CAR T-cell therapy in children with trisomy 21 and other important subpopulations.
Measured: 12 months
Impact of next generation sequencing MRD and duration of B cell aplasia
To determine the impact of next-generation sequencing MRD results and duration of B-cell aplasia on CAR T cell outcomes.
Measured: 12 months
Frequency of infections
To evaluate the frequency of infections and describe immune system function after CAR T-cell therapy.
Measured: 12 months
Response, survival and toxicities after CAR
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)
Measured: 12 months
Compare toxicities and outcomes across CAR constructs
To compare toxicities and outcomes across CAR T-cell constructs (e.g., different CD19 CAR constructs, dual-targeted CARs, CD22-targeted CARs, etc.)
Measured: 12 months
Impact of CAR on other health-related outcomes
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
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jun 2022
- Study start14 Oct 2021
- Primary completion29 Oct 2024
- Study completion29 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leukemia, Acute LymphoblasticStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Sara K Silbert, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05440409
- Sponsor's protocol ID
- 10000651
- Other IDs
- 000651-C
- Status last verified
- 22 Oct 2026
- First submitted
- 29 Jun 2022
- Last updated
- 26 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: All IPD recorded in the medical record will be shared with intramural investigators upon request.
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- CAR-Multicenter Analysis (CAR-MA): Retrospective Study to Characterize CAR T-cell Outcomes and Related Toxicities in Children and Young Adults With B-ALL
- ClinicalTrials.gov ID
- NCT05440409
- Acronym / sponsor ID
- 10000651
- Keywords
- Relapsed Disease, MRD-positive CD19+ ALL, Natural History
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 57 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 14 Oct 2021 (actual)
- Primary completion
- 29 Oct 2024 (actual)
- Study completion
- 29 Oct 2024 (actual)
- First posted
- 30 Jun 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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 Tcell 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 Tcell 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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | – | –Retrospective chart review of children and adults with cancer enrolled on immunotherapy treatment protocols |
Who may qualify
- Ages
- 3 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Response free survival (completed)
Time frame: 6 months
To evaluate the RFS at 6 months following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab.
Outcome evaluation
Time frame: 12 months
To retrospectively evaluate outcomes following CAR T-cell therapy across children and young adults with B-ALL
14 secondary outcomes
Complete Response Rate (completed)
Time frame: 12 months
Relapse Rate (completed)
Time frame: 12 months
Response free survival (completed)
Time frame: 12 months
Minimal Residual Disease detection (completed)
Time frame: 12 months
Impact of immunotherapies on CAR outcomes
Time frame: 12 months
Cytopenias after CAR
Time frame: 12 months
Response of extramedullary disease
Time frame: 12 months
Frequency of malignant neoplasms
Time frame: 12 months
Response, survival and toxicities in subpopulations
Time frame: 12 months
Impact of next generation sequencing MRD and duration of B cell aplasia
Time frame: 12 months
Frequency of infections
Time frame: 12 months
Response, survival and toxicities after CAR
Time frame: 12 months
Compare toxicities and outcomes across CAR constructs
Time frame: 12 months
Impact of CAR on other health-related outcomes
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2026.
- Individual participant dataSharing planned: Yes. All IPD recorded in the medical record will be shared with intramural investigators upon request.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sara K Silbert, M.D. principal investigator
National Cancer Institute (NCI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Leukemia, Acute Lymphoblastic and Lymphoblastic Leukemia, Acute, Childhood, run by National Cancer Institute (NCI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Response free survival (completed) (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2026.