Clinical trial · Interventional
Matched Targeted Therapy For High-Risk Leukemias and Myelodysplastic Syndrome
Matched Targeted Therapy (MTT) Recommendation for Patients With Recurrent, Refractory, or High Risk Leukemias and Myelodysplastic Syndrome
NCT02670525CI-TRIAL-00120855active not recruitingN/AResults postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Matched Targeted Therapy | — | UNRESOLVED | — |
| Recurrent, Refractory, or High Risk Leukemias | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Leukemia Profiling | Genetic | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Relapsed/Refractory Leukemia
- description
- 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 After the screening procedures confirms patient eligibility: * Leukemia Profiling will be performed * Identifying an actionable genomic alteration and making a matched targeted therapy treatment recommendation.
- interventionNames
- Genetic: Leukemia Profiling
- type
- EXPERIMENTAL
- label
- New Diagnosis
- description
- Cohort 2: New Diagnosis * Acute myeloid leukemia (AML), new diagnosis (excluding acute promyelocytic leukemia (APL)) * 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 After the screening procedures confirms eligibility: * Leukemia Profiling will be performed * Identifying an actionable genomic alteration and making a matched targeted therapy treatment recommendation.
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 30 Years
Show eligibility criteria text
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.
References
Publications (1)
- DERIVEDPaolino 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. PMID 37725576