Trial Outcomes & Findings for Matched Targeted Therapy For High-Risk Leukemias and Myelodysplastic Syndrome (NCT NCT02670525)
NCT ID: NCT02670525
Last Updated: 2026-01-09
Results Overview
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%.
ACTIVE_NOT_RECRUITING
NA
338 participants
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.
2026-01-09
Participant Flow
Patients enrolled from August 17, 2016 to May 4, 2022.
Participant milestones
| Measure |
Relapsed/Refractory Leukemia
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
|
New Diagnosis
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
|
|---|---|---|
|
Overall Study
STARTED
|
236
|
102
|
|
Overall Study
Matched targeted therapy (MTT) data available
|
157
|
74
|
|
Overall Study
Evaluable for primary endpoint
|
101
|
0
|
|
Overall Study
COMPLETED
|
14
|
9
|
|
Overall Study
NOT COMPLETED
|
222
|
93
|
Reasons for withdrawal
| Measure |
Relapsed/Refractory Leukemia
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
|
New Diagnosis
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
|
|---|---|---|
|
Overall Study
Death
|
94
|
14
|
|
Overall Study
Lost to Follow-up
|
4
|
1
|
|
Overall Study
Subject withdrew consent
|
1
|
0
|
|
Overall Study
Unknown, data entry delayed
|
123
|
78
|
Baseline Characteristics
Matched Targeted Therapy For High-Risk Leukemias and Myelodysplastic Syndrome
Baseline characteristics by cohort
| Measure |
Relapsed/Refractory Leukemia
n=236 Participants
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.
Leukemia Profiling: 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.
|
New Diagnosis
n=102 Participants
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.
Leukemia Profiling: 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.
|
Total
n=338 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
11.3 years
n=8 Participants
|
10.2 years
n=7 Participants
|
11.1 years
n=15 Participants
|
|
Age, Customized
Age (years) at sample · <1 years
|
7 Participants
n=8 Participants
|
10 Participants
n=7 Participants
|
17 Participants
n=15 Participants
|
|
Age, Customized
Age (years) at sample · 1-9 years
|
91 Participants
n=8 Participants
|
40 Participants
n=7 Participants
|
131 Participants
n=15 Participants
|
|
Age, Customized
Age (years) at sample · 10-17 years
|
94 Participants
n=8 Participants
|
33 Participants
n=7 Participants
|
127 Participants
n=15 Participants
|
|
Age, Customized
Age (years) at sample · >=18 years
|
44 Participants
n=8 Participants
|
19 Participants
n=7 Participants
|
63 Participants
n=15 Participants
|
|
Sex: Female, Male
Female
|
113 Participants
n=8 Participants
|
51 Participants
n=7 Participants
|
164 Participants
n=15 Participants
|
|
Sex: Female, Male
Male
|
123 Participants
n=8 Participants
|
51 Participants
n=7 Participants
|
174 Participants
n=15 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
43 Participants
n=8 Participants
|
16 Participants
n=7 Participants
|
59 Participants
n=15 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
144 Participants
n=8 Participants
|
75 Participants
n=7 Participants
|
219 Participants
n=15 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
49 Participants
n=8 Participants
|
11 Participants
n=7 Participants
|
60 Participants
n=15 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=8 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=15 Participants
|
|
Race (NIH/OMB)
Asian
|
15 Participants
n=8 Participants
|
9 Participants
n=7 Participants
|
24 Participants
n=15 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=8 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=15 Participants
|
|
Race (NIH/OMB)
Black or African American
|
19 Participants
n=8 Participants
|
14 Participants
n=7 Participants
|
33 Participants
n=15 Participants
|
|
Race (NIH/OMB)
White
|
127 Participants
n=8 Participants
|
60 Participants
n=7 Participants
|
187 Participants
n=15 Participants
|
|
Race (NIH/OMB)
More than one race
|
24 Participants
n=8 Participants
|
15 Participants
n=7 Participants
|
39 Participants
n=15 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
51 Participants
n=8 Participants
|
4 Participants
n=7 Participants
|
55 Participants
n=15 Participants
|
|
Disease
AML
|
89 Participants
n=8 Participants
|
70 Participants
n=7 Participants
|
159 Participants
n=15 Participants
|
|
Disease
MDS
|
4 Participants
n=8 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=15 Participants
|
|
Disease
JMML
|
2 Participants
n=8 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=15 Participants
|
|
Disease
B-cell ALL
|
111 Participants
n=8 Participants
|
1 Participants
n=7 Participants
|
112 Participants
n=15 Participants
|
|
Disease
T-cell ALL
|
14 Participants
n=8 Participants
|
2 Participants
n=7 Participants
|
16 Participants
n=15 Participants
|
|
Disease
Infant MLL rearranged ALL
|
2 Participants
n=8 Participants
|
4 Participants
n=7 Participants
|
6 Participants
n=15 Participants
|
|
Disease
Leukemia of ambiguous lineage
|
8 Participants
n=8 Participants
|
8 Participants
n=7 Participants
|
16 Participants
n=15 Participants
|
|
Disease
Other rare leukemia
|
2 Participants
n=8 Participants
|
4 Participants
n=7 Participants
|
6 Participants
n=15 Participants
|
|
Disease
Secondary leukemia
|
4 Participants
n=8 Participants
|
9 Participants
n=7 Participants
|
13 Participants
n=15 Participants
|
PRIMARY outcome
Timeframe: 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.Population: The analysis dataset is comprised of the first 100 patients in Cohort 1, as defined per protocol. One patient in Cohort 2 was later identified to have relapsed/refractory disease, so therefore the analysis population contains 101 patients. The remainder of Cohort 1 patients and the entirety of Cohort 2 patients are represented in the analysis of secondary outcomes.
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%.
Outcome measures
| Measure |
Relapsed/Refractory Leukemia
n=101 Participants
Cohort 1: Relapsed/refractory leukemia
* Acute lymphoblastic leukemia, first or greater relapse
* Acute myeloid leukemia, 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.
Leukemia Profiling: 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.
|
New Diagnosis
Cohort 2: New diagnosis
* Acute myeloid leukemia, new diagnosis (excluding acute promyelocytic leukemia (APL))
* New diagnosis infant MLL-rearranged ALL or low hypodiploid (\<40 chromosomes) ALL
* Rare leukemia- e.g., 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.
Leukemia Profiling: 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.
|
|---|---|---|
|
Rate of Patients With Actionable Alterations
|
77 percentage of participants
Interval 69.0 to 84.0
|
—
|
SECONDARY outcome
Timeframe: 2 YearsThis 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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 YearsThis 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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 YearsThis 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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 YearsThis 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.
Outcome measures
Outcome data not reported
Adverse Events
Relapsed/Refractory Leukemia
New Diagnosis
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place