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This research study is seeking to gain new knowledge about Recurrent, Refractory, or High Risk Leukemias in children and young adults.
This study is evaluating the use of specialized testing called leukemia profiling. Once the profiling is performed, the results are evaluated by an expert panel of physicians, scientists and pharmacists. This may result in a recommendation for a specific cancer therapy or a clinical trial called matched targeted therapy (MTT). The results of the leukemia profiling and, if applicable, the MTT recommendation will be communicated to the participant's primary oncologist.
Our tissues and organs are made up of cells. Cancer occurs when the molecules that normally control cell growth are damaged. The damage results in unchecked cell growth which causes a tumor, a collection of cancer cells. The damage is referred to as an alteration. There are different types of cancer-causing alterations. Genes are the part of cells that contain the instructions which tell our cells how to make the right proteins to grow and work. Genes are composed of Deoxyribonucleic Acid (DNA) letters that spell out these instructions.
By participating in this study, the participant's leukemia cells will be tested for cancer causing alterations. This testing is called leukemia profiling. The leukemia profiling will be performed using bone marrow or blood that has already been obtained during a clinical test. Alternately, the profiling may be done on leukemia cells that are planned to be obtained as part of routine clinical care.
This study will determine whether it is possible to use profiling results to determine a matched targeted therapy for patients with leukemia. It will describe the range of mutations found in patients with leukemia with this type of profiling, and describe the clinical outcomes of patients who receive a matched targeted therapy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Relapsed/Refractory Leukemia | Experimental | Cohort 1: Relapsed/Refractory Leukemia
After the screening procedures confirms patient eligibility:
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| New Diagnosis | Experimental | Cohort 2: New Diagnosis
After the screening procedures confirms eligibility:
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Leukemia Profiling | Genetic | Genetic profiling of leukemia cells will be performed and analyzed by an expert panel. Matched targeted therapy recommendation based on profiling results will be made if available. The recommendation, if available, will be communicated to the primary oncologist. |
| Measure | Description | Time Frame |
|---|---|---|
| Rate of Patients With Actionable Alterations | An actionable alteration was defined as a cancer-associated genomic event for which there was a targeted drug available. Actionable genetic alterations were identified by a combination of standard-of-care cytogenetics/fluorescence in situ hybridization (FISH) and sequencing performed as part of the study. This study is considered feasible if at least 13% of participants analyzed have profile data and identifiable actionable alterations. A 90% exact binomial confidence interval is presented for the rate of patients with actionable alterations to compare the observed rate against 13%. | Actionable alteration was identified based on a combination of fluorescence in situ hybridization (FISH)/cytogenetics and sequencing. Average time to full results was 2 weeks. |
| Measure | Description | Time Frame |
|---|---|---|
| Rate of Somatic Genomic Alterations | This Secondary Outcome will be addressed by describing the somatic genomic alterations in Cohort 1 (Relapsed/Refractory Leukemia arm) and Cohort 2 (New Diagnosis arm) that are discovered by genomic analyses. | 2 Years |
| Rate of Results Reporting |
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Inclusion Criteria:
Cohort 1: Relapsed/refractory leukemia
Cohort 2: New diagnosis
Pathologic Criteria
Specimen Samples
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Yana Pikman, MD | Dana-Farber Cancer Institute | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Helen Diller Family Comprehensive Cancer Center | San Francisco | California | 94158 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37725576 | Derived | Paolino J, Dimitrov B, Apsel Winger B, Sandoval-Perez A, Rangarajan AV, Ocasio-Martinez N, Tsai HK, Li Y, Robichaud AL, Khalid D, Hatton C, Gillani R, Polonen P, Dilig A, Gotti G, Kavanagh J, Adhav AA, Gow S, Tsai J, Li Y, Ebert BL, Van Allen EM, Bledsoe J, Kim AS, Tasian SK, Cooper SL, Cooper TM, Hijiya N, Sulis ML, Shukla NN, Magee JA, Mullighan CG, Burke MJ, Luskin MR, Mar BG, Jacobson MP, Harris MH, Stegmaier K, Place AE, Pikman Y. Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma. Clin Cancer Res. 2023 Nov 14;29(22):4613-4626. doi: 10.1158/1078-0432.CCR-22-2562. |
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Patients enrolled from August 17, 2016 to May 4, 2022.
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| ID | Title | Description |
|---|---|---|
| FG000 | Relapsed/Refractory Leukemia | Cohort 1: Relapsed/refractory leukemia
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| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Dec 1, 2021 |
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This Secondary Outcome will be addressed by a description of the time of results reporting. Documentation of the time of sample receipt and processing, resources and personnel utilized at each step of the process, time to results reporting, interpretation and review by Expert panel and communication of results to the primary oncologist will be captured. |
| 2 Years |
| Parent's Feelings and Understanding of Genomic Testing | This Secondary Outcome will be addressed by describing the hopes and concerns of parents of children with recurrent/refractory/high-risk de novo leukemia regarding genomic testing of their child's leukemia as well as their understanding of the testing and evaluate whether the hopes and concerns were realized following the return of results. | 2 Years |
| Analysis of Primary Leukemia Sensitivity Testing and Establishment of Xenograft Models | This Secondary Outcome will be addressed by completing analysis of the primary leukemia sensitivity testing to a panel of drugs or shRNA and establishing xenograft models in dedicated participating research laboratories and conducting co-clinical trials with the recommended matched therapy once the animal model is established. | 2 Years |
| Children's Hospital Colorado |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30322 | United States |
| The University of Chicago | Chicago | Illinois | 60637 | United States |
| Johns Hopkins Hospital | Baltimore | Maryland | 21287 | United States |
| Dana Farber Cancer Institute | Boston | Massachusetts | 02215 | United States |
| Children's Hospital's and Clinics of Minnesota | Minneapolis | Minnesota | 55404 | United States |
| Washington University at St. Louis School of Medicine | St Louis | Missouri | 63110 | United States |
| Roswell Park Comprehensive Cancer Center | Buffalo | New York | 14263 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| Memorial Sloan Kettering Cancer Center | New York | New York | 10065 | United States |
| The Children's Hospital at Montefiore | The Bronx | New York | 10467 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19404 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| FG001 |
| New Diagnosis |
Cohort 2: New diagnosis
|
| Matched targeted therapy (MTT) data available |
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| Evaluable for primary endpoint |
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| COMPLETED | A patient is completed if they completed the required follow-up and came off study. |
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| NOT COMPLETED |
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This analysis dataset is comprised of all enrolled patients.
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| ID | Title | Description |
|---|---|---|
| BG000 | Relapsed/Refractory Leukemia | Cohort 1: Relapsed/refractory leukemia
After the screening procedures confirms patient eligibility:
Leukemia Profiling: Genetic profiling of leukemia cells will be performed and analyzed by an expert panel. Matched targeted therapy recommendation based on profiling results will be made if available. The recommendation, if available, will be communicated to the primary oncologist. |
| BG001 | New Diagnosis | Cohort 2: New diagnosis
After the screening procedures confirms eligibility:
Leukemia Profiling: Genetic profiling of leukemia cells will be performed and analyzed by an expert panel. Matched targeted therapy recommendation based on profiling results will be made if available. The recommendation, if available, will be communicated to the primary oncologist. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median | Full Range | years |
| |||||||||||||||
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Disease | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Rate of Patients With Actionable Alterations | An actionable alteration was defined as a cancer-associated genomic event for which there was a targeted drug available. Actionable genetic alterations were identified by a combination of standard-of-care cytogenetics/fluorescence in situ hybridization (FISH) and sequencing performed as part of the study. This study is considered feasible if at least 13% of participants analyzed have profile data and identifiable actionable alterations. A 90% exact binomial confidence interval is presented for the rate of patients with actionable alterations to compare the observed rate against 13%. | The analysis dataset is comprised of the first 100 patients in Cohort 1, as defined per protocol. One patient in Cohort 2 was later identified to have relapsed/refractory disease, so therefore the analysis population contains 101 patients. The remainder of Cohort 1 patients and the entirety of Cohort 2 patients are represented in the analysis of secondary outcomes. | Posted | Number | 90% Confidence Interval | percentage of participants | Actionable alteration was identified based on a combination of fluorescence in situ hybridization (FISH)/cytogenetics and sequencing. Average time to full results was 2 weeks. |
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| ||||||||||||||||||||||||||||
| Secondary | Rate of Somatic Genomic Alterations | This Secondary Outcome will be addressed by describing the somatic genomic alterations in Cohort 1 (Relapsed/Refractory Leukemia arm) and Cohort 2 (New Diagnosis arm) that are discovered by genomic analyses. | Not Posted | May 2028 | 2 Years | Participants | |||||||||||||||||||||||||||||||||
| Secondary | Rate of Results Reporting | This Secondary Outcome will be addressed by a description of the time of results reporting. Documentation of the time of sample receipt and processing, resources and personnel utilized at each step of the process, time to results reporting, interpretation and review by Expert panel and communication of results to the primary oncologist will be captured. | Not Posted | May 2028 | 2 Years | Participants | |||||||||||||||||||||||||||||||||
| Secondary | Parent's Feelings and Understanding of Genomic Testing | This Secondary Outcome will be addressed by describing the hopes and concerns of parents of children with recurrent/refractory/high-risk de novo leukemia regarding genomic testing of their child's leukemia as well as their understanding of the testing and evaluate whether the hopes and concerns were realized following the return of results. | Not Posted | May 2028 | 2 Years | Participants | |||||||||||||||||||||||||||||||||
| Secondary | Analysis of Primary Leukemia Sensitivity Testing and Establishment of Xenograft Models | This Secondary Outcome will be addressed by completing analysis of the primary leukemia sensitivity testing to a panel of drugs or shRNA and establishing xenograft models in dedicated participating research laboratories and conducting co-clinical trials with the recommended matched therapy once the animal model is established. | Not Posted | May 2028 | 2 Years | Participants |
Adverse event data was not of interest or collected for this study since there was no treatment intervention involved. Mortality data was not monitored for the duration of study completion, and cause of death was not collected for patients for whom a status of death was provided by outside sites.
Adverse event data was not of interest or collected for this study since there was no treatment intervention involved. Mortality data was not monitored for the duration of study completion, and cause of death was not collected for patients for whom a status of death was provided by outside sites.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Relapsed/Refractory Leukemia | Cohort 1: Relapsed/refractory leukemia
After the screening procedures confirms patient eligibility:
Leukemia Profiling: Genetic profiling of leukemia cells will be performed and analyzed by an expert panel. Matched targeted therapy recommendation based on profiling results will be made if available. The recommendation, if available, will be communicated to the primary oncologist. | 0 | 0 | 0 | 0 | 0 | 0 |
| EG001 | New Diagnosis | Cohort 2: New diagnosis
After the screening procedures confirms eligibility:
Leukemia Profiling: Genetic profiling of leukemia cells will be performed and analyzed by an expert panel. Matched targeted therapy recommendation based on profiling results will be made if available. The recommendation, if available, will be communicated to the primary oncologist. | 0 | 0 | 0 | 0 | 0 | 0 |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Yana Pikman | Dana-Farber Cancer Institute | 6176324754 | yana_pikman@dfci.harvard.edu |
| May 9, 2023 |
| Prot_SAP_000.pdf |
| ID | Term |
|---|---|
| D012008 | Recurrence |
| ID | Term |
|---|---|
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| 1-9 years |
|
| 10-17 years |
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| >=18 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
|
| Unknown or Not Reported |
|
| MDS |
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| JMML |
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| B-cell ALL |
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| T-cell ALL |
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| Infant MLL rearranged ALL |
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| Leukemia of ambiguous lineage |
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| Other rare leukemia |
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| Secondary leukemia |
|