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Matched Targeted Therapy (MTT) Recommendation for Patients with Recurrent, Refractory, or High Risk Leukemias

Cancer Type
Acute Lymphoblastic Leukemia
Acute Myelogenous Leukemia
Myelodysplastic Syndromes (MDS)
Trial Phase
0 - 30 Years, Male and Female
Study Type
Protocol IDs
15-384 (primary)
Study Sponsor
Dana-Farber Harvard Cancer Center


This clinical trial studies matched targeted therapy recommendation in identifying genetic changes in patients with leukemia that has come back after a period of improvement (recurrent), has not responded to previous treatment (refractory) or it is likely to come back or spread (high risk). Studying samples from patients with leukemia in the laboratory may help doctors learn more about biomarkers related to cancer and may help receive matched therapy based on the identified changes in the genes.


I. To determine whether it is feasible to identify actionable alterations with a matched targeted therapy for pediatric participants with recurrent/refractory/high-risk de novo leukemia.

I. Describe the range of somatic genomic alterations identified using a leukemia-focused sequencing panel in pediatric patients with recurrent/refractory/high-risk de novo leukemia.
II. Describe the time between receipt of sample and results reporting and interpretation of genomic data when performed in a research context and explore the factors that impact this time interval.
III. Analyze 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 return of results.
IV. Study the feasibility and utility of pre-clinical studies including testing primary leukemia sensitivity testing to a panel of drugs or short hairpin ribonucleic acid (shRNA) and the creation of xenograft models of leukemia and measure response to genomic-based recommendations in these models of leukemia.

I. Explore whether patients received targeted therapy matched to an actionable alteration recommended by the expert review panel and explore reasons that a drug was not received.
II. Explore whether actionable mutations can be identified using technology beyond the targeted cancer gene panel(s).
III. Explore the different sample types (e.g., buccal swab, saliva, nail clippings, bone marrow- and skin-derived fibroblasts, remission bone marrow) that may be used to assess germline status in patients with relapsed/refractory/high-risk leukemia.
IV. Evaluate whether relapsed/refractory/high-risk pediatric leukemias acquire altered clonal predominance and additional actionable alterations during disease progression or during treatment with a matched therapy.
V. To store leukemia material, derived xenografts, deoxyribonucleic acid (DNA) derived from buccal swabs/saliva/nail clippings/bone marrow- and skin-derived fibroblasts, and remission bone marrow samples for potential future research.

Previously collected samples are analyzed using tumor profiling and sequencing. Participants may also undergo collection of saliva, buccal swab, tissue, finger nail clippings, bone marrow or blood samples for genetic analysis via sequencing. Patients may receive a recommendation for an individualized cancer therapy.

After completion of study, patients are followed for 5 years.

Treatment Sites in Georgia

Aflac Cancer and Blood Disorders Center of Children’s at Egleston

1405 Clifton Road NE
3rd Floor
Atlanta, GA 30322

**Clinical trials are research studies that involve people. These studies test new ways to prevent, detect, diagnose, or treat diseases. People who take part in cancer clinical trials have an opportunity to contribute to scientists’ knowledge about cancer and to help in the development of improved cancer treatments. They also receive state-of-the-art care from cancer experts... Click here to learn more about clinical trials.
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