Recurrent, Refractory, or High Risk LeukemiasNCT02670525
Matched Targeted Therapy For High-Risk Leukemias and Myelodysplastic Syndrome
A study testing Leukemia Profiling in people with Recurrent, Refractory, or High Risk Leukemias and Matched Targeted Therapy, run by Dana-Farber Cancer Institute at 15 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 30 years
- Conditions the study is for
- Recurrent, Refractory, or High Risk Leukemias, Matched Targeted Therapy
- What is being tested?
- Leukemia Profiling
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 338 participants
- Where
- 15 sitesUnited States
- Study dates
- 17 Aug 2016to 31 Jan 2028
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Show all 18Show fewer
- 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.
Who cannot join
- 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.
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
United States15 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Johns Hopkins Hospital
Baltimore, Maryland, 21287
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14263
The University of Chicago
Chicago, Illinois, 60637
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Children's Hospital's and Clinics of Minnesota
Minneapolis, Minnesota, 55404
Columbia University Medical Center
New York, New York, 10032
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19404
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, 94158
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University at St. Louis School of Medicine
St Louis, Missouri, 63110
The Children's Hospital at Montefiore
The Bronx, New York, 10467
About this study
Official title: Matched Targeted Therapy (MTT) Recommendation for Patients With Recurrent, Refractory, or High Risk Leukemias and Myelodysplastic Syndrome
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.
Read the detailed description
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.
- Being tested
- Leukemia Profiling
- Keywords
- Recurrent, Refractory, or High Risk Leukemias, Matched targeted therapy
- Medical subject headings
- Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Leukemia ProfilingGenetic
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.
| Study group | Role | What they receive |
|---|---|---|
| Relapsed/Refractory Leukemia | Experimental | 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. Receives: Genetic: Leukemia Profiling |
| New Diagnosis | Experimental | 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. Receives: Genetic: Leukemia Profiling |
What is being measured
The primary outcome is the main question the study is designed to answer.
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%.
Measured: 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 outcomes (4)
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.
Measured: 2 Years
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.
Measured: 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.
Measured: 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.
Measured: 2 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Feb 2016 (estimated)
- Study start17 Aug 2016
- Primary completion16 May 2022
- Study completion31 Jan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Recurrent, Refractory, or High Risk LeukemiasStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 338 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Yana Pikman, MD, Dana-Farber Cancer Institute
- Investigators
- Yana Pikman, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02670525
- Sponsor's protocol ID
- 15-384
- Status last verified
- Dec 2025
- First submitted
- 28 Jan 2016
- Last updated
- 6 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
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.
PubMed 37725576
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Matched Targeted Therapy (MTT) Recommendation for Patients With Recurrent, Refractory, or High Risk Leukemias and Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT02670525
- Acronym / sponsor ID
- 15-384
- Keywords
- Recurrent, Refractory, or High Risk Leukemias, Matched targeted therapy
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 338 participants (actual)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Study start
- 17 Aug 2016 (actual)
- Primary completion
- 16 May 2022 (actual)
- Study completion
- 31 Jan 2028 (estimated)
- First posted
- 1 Feb 2016 (estimated)
- Results
- Posted 18 Jul 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Relapsed/Refractory Leukemia | Experimental | Genetic: Leukemia ProfilingCohort 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. |
| New Diagnosis | Experimental | Genetic: Leukemia ProfilingCohort 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. |
Who may qualify
- Ages
- Up to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (18 listed)
- 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
Key exclusion criteria (1 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Leukemia ProfilingGenetic 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. | Genetic | New Diagnosis, Relapsed/Refractory Leukemia |
Primary outcome
Rate of Patients With Actionable Alterations
Time frame: 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.
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%.
4 secondary outcomes
Rate of Somatic Genomic Alterations
Time frame: 2 Years
Rate of Results Reporting
Time frame: 2 Years
Parent's Feelings and Understanding of Genomic Testing
Time frame: 2 Years
Analysis of Primary Leukemia Sensitivity Testing and Establishment of Xenograft Models
Time frame: 2 Years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37725576Paolino 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yana Pikman, MD principal investigator
Dana-Farber Cancer Institute
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Leukemia Profiling in people with Recurrent, Refractory, or High Risk Leukemias and Matched Targeted Therapy, run by Dana-Farber Cancer Institute at 15 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 6 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Jul 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of Patients With Actionable Alterations (time frame: 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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02670525, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Aug 2026.