Matched Targeted Therapy Clinical Trial
Official title:
Matched Targeted Therapy (MTT) Recommendation for Patients With Recurrent, Refractory, or High Risk Leukemias and Myelodysplastic Syndrome
| Verified date | October 2023 |
| Source | Dana-Farber Cancer Institute |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Active, not recruiting |
| Enrollment | 338 |
| Est. completion date | May 2027 |
| Est. primary completion date | May 16, 2022 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | N/A to 30 Years |
| Eligibility | Inclusion Criteria: - Birth to = 30 years at study entry - Diagnosis: Patients will be enrolled in one of the two cohorts based on diagnosis: Cohort 1: Relapsed/refractory leukemia - Acute lymphoblastic leukemia (ALL), first or greater relapse - Acute myeloid leukemia (AML), first or greater relapse - Leukemia refractory to induction chemotherapy - Other recurrent leukemia - Myelodysplastic syndrome (MDS), first or greater relapse, or refractory to initial therapy Cohort 2: New diagnosis - Acute myeloid leukemia (AML), new diagnosis - New diagnosis infant mixed-lineage leukemia (MLL)-rearranged ALL or low hypodiploid (<40 chromosomes) ALL - Rare leukemia- e.g., juvenile myelomonocytic leukemia (JMML), leukemia of ambiguous lineage - Secondary leukemia - Myelodysplastic syndrome (MDS) not eligible for stem cell transplant Pathologic Criteria - Histologic confirmation of leukemia at the time of diagnosis or recurrence Specimen Samples - Sufficient leukemia specimen available for profiling from diagnosis or recurrence OR bone marrow aspirate/blood draw/pheresis/other fresh sample of patient leukemia cells planned for clinical care anticipated to allow collection of minimum specimen for testing. Exclusion Criteria: - Insufficient leukemia specimen available for profiling from diagnosis or recurrence; or bone marrow evaluation/blood draw/other leukemia cell sample NOT planned to be obtained for clinical care; or peripheral blast percentage <20% AND clinical blood draw not planned. |
| Country | Name | City | State |
|---|---|---|---|
| United States | Children's Healthcare of Atlanta | Atlanta | Georgia |
| United States | Children's Hospital Colorado | Aurora | Colorado |
| United States | Johns Hopkins Hospital | Baltimore | Maryland |
| United States | Dana Farber Cancer Institute | Boston | Massachusetts |
| United States | The Children's Hospital at Montefiore | Bronx | New York |
| United States | Roswell Park Comprehensive Cancer Center | Buffalo | New York |
| United States | The University of Chicago | Chicago | Illinois |
| United States | Children's Hospital of Wisconsin | Milwaukee | Wisconsin |
| United States | Children's Hospital's and Clinics of Minnesota | Minneapolis | Minnesota |
| United States | Columbia University Medical Center | New York | New York |
| United States | Memorial Sloan Kettering Cancer Center | New York | New York |
| United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
| United States | Washington University at St. Louis School of Medicine | Saint Louis | Missouri |
| United States | UCSF Helen Diller Family Comprehensive Cancer Center | San Francisco | California |
| United States | Seattle Children's Hospital | Seattle | Washington |
| Lead Sponsor | Collaborator |
|---|---|
| Dana-Farber Cancer Institute |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| 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%. | 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. | |
| 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. | 2 Years | |
| 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. | 2 Years | |
| 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. | 2 Years | |
| 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. | 2 Years |