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Targeted Therapy

Matched Targeted Therapy for High-Risk Leukemia

N/A

Waitlist Available

Led By Yana Pikman, MD

Research Sponsored by Dana-Farber Cancer Institute

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Must not have

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.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is seeking to find new ways to treat leukemia in children and young adults who have either not responded to treatment, had their cancer come back after treatment, or have a high risk of the cancer coming back.

Who is the study for?

This trial is for children and young adults up to 30 years old with high-risk leukemias or myelodysplastic syndrome (MDS), including those who have relapsed, are refractory to treatment, or newly diagnosed with certain types. Participants must have a confirmed diagnosis and sufficient leukemia samples available for profiling.

What is being tested?

The study focuses on leukemia profiling to identify specific cancer therapies tailored to the patient's unique condition. An expert panel reviews the results, which may lead to recommendations for matched targeted therapy (MTT) that will be shared with the participant's primary oncologist.

What are the potential side effects?

Since this trial involves testing and personalized therapy recommendations rather than direct administration of drugs, side effects would depend on the individual treatments chosen based on profiling results.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My leukemia has returned or didn't respond to initial treatment.

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I have a new diagnosis of a rare or specific type of leukemia and am not eligible for a stem cell transplant.

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My leukemia was confirmed through lab tests.

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I am 30 years old or younger.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I don't have enough leukemia samples for testing or my blood tests show less than 20% cancer cells and no further tests are planned.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Rate of Patients With Actionable Alterations

Secondary study objectives

Analysis of Primary Leukemia Sensitivity Testing and Establishment of Xenograft Models

Parent's Feelings and Understanding of Genomic Testing

Rate of Results Reporting

+1 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Relapsed/Refractory LeukemiaExperimental Treatment1 Intervention

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.

Group II: New DiagnosisExperimental Treatment1 Intervention

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.

0 / 5Eligibility criteria met