Trial Outcomes & Findings for Liposome-encapsulated Daunorubicin-Cytarabine, Fludarabine Phosphate, Cytarabine, and Filgrastim in Treating Younger Patients With Relapsed or Refractory Acute Myeloid Leukemia (NCT NCT02642965)
NCT ID: NCT02642965
Last Updated: 2023-09-08
Results Overview
Number of patients in the dose-finding phase with a dose-limiting toxicity, graded using the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
38 participants
Primary outcome timeframe
28 days
Results posted on
2023-09-08
Participant Flow
Participant milestones
| Measure |
Treatment (CPX-351 and FLAG)
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
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|---|---|
|
Overall Study
STARTED
|
38
|
|
Overall Study
COMPLETED
|
25
|
|
Overall Study
NOT COMPLETED
|
13
|
Reasons for withdrawal
| Measure |
Treatment (CPX-351 and FLAG)
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
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|---|---|
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Overall Study
Lack of Efficacy
|
4
|
|
Overall Study
Physician Decision
|
9
|
Baseline Characteristics
Liposome-encapsulated Daunorubicin-Cytarabine, Fludarabine Phosphate, Cytarabine, and Filgrastim in Treating Younger Patients With Relapsed or Refractory Acute Myeloid Leukemia
Baseline characteristics by cohort
| Measure |
Treatment (CPX-351 and FLAG)
n=38 Participants
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
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|---|---|
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Age, Categorical
<=18 years
|
35 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
20 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
29 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
26 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
36 participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 28 daysPopulation: First 6 patients evaluable for dose limiting toxicity (DLT).
Number of patients in the dose-finding phase with a dose-limiting toxicity, graded using the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0.
Outcome measures
| Measure |
Treatment (CPX-351 and FLAG)
n=6 Participants
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
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|---|---|
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Number of Participants With a Dose-limiting Toxicity
|
1 Participants
|
PRIMARY outcome
Timeframe: Up to 8 weeksPopulation: 1 patient among the 38 eligible patients was not evaluable for response.
Best response (complete response or complete remission with partial platelet recovery) after up to 2 cycles of therapy, where response is assessed using the revised Acute Myeloid Leukemia International Working Group Criteria. Percentage of responders is calculated using the methods of Jung and Kim. 90% confidence interval is determined using the methods of Koyama and Chen.
Outcome measures
| Measure |
Treatment (CPX-351 and FLAG)
n=37 Participants
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
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|---|---|
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Percentage of Responders (Complete Response or Complete Remission With Partial Platelet Recovery) After up to 2 Cycles
|
68.30 Percantage of best responders
Interval 52.89 to 78.02
|
SECONDARY outcome
Timeframe: Up to 4 weeksPopulation: 1 patient among the 38 eligible patients was not evaluable for response.
Response (complete response or complete remission with partial or incomplete platelet recovery) after first cycle of therapy, where response is assessed using the revised Acute Myeloid Leukemia International Working Group Criteria. Percentage of responders is calculated by the total of number of patients with complete response or complete remission with partial or incomplete platelet recovery divided by the number of patients evaluable for response after the first cycle. 95% confidence interval is determined using a binomial exact method.
Outcome measures
| Measure |
Treatment (CPX-351 and FLAG)
n=37 Participants
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
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|---|---|
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Percentage of Responders (Complete Response or Complete Remission With Partial or Incomplete Platelet Recovery) After First Cycle of Therapy
|
75.68 Percentage of responders
Interval 58.8 to 88.23
|
SECONDARY outcome
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1Population: Patients in the efficacy phase excluded (n=32). 1 dose finding patient excluded because dose record was not consistent with treatment plan.
Geometric mean liposome-encapsulated daunorubicin clearance following IV infusion will be determined for patients in the dose-finding phase.
Outcome measures
| Measure |
Treatment (CPX-351 and FLAG)
n=5 Participants
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
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|---|---|
|
Liposome-encapsulated Daunorubicin Clearance
|
94.7 MILLILITER / HOUR
Interval 47.5 to 193.0
|
SECONDARY outcome
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1Population: Patients in the efficacy phase excluded (n=32). 1 dose finding patient excluded because dose record was not consistent with treatment plan.
Geometric mean liposome-encapsulated daunorubicin volume of distribution following IV infusion will be determined for patients in the dose-finding phase.
Outcome measures
| Measure |
Treatment (CPX-351 and FLAG)
n=5 Participants
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
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|---|---|
|
Liposome-encapsulated Daunorubicin Volume of Distribution
|
3827.7 MILLILITER
Interval 2165.0 to 6596.0
|
SECONDARY outcome
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1Population: Patients in the efficacy phase excluded (n=32). 1 dose finding patient excluded because dose record was not consistent with treatment plan.
Median liposome-encapsulated daunorubicin time of maximum observed plasma concentration will be determined for patients in the dose-finding phase.
Outcome measures
| Measure |
Treatment (CPX-351 and FLAG)
n=5 Participants
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
|
|---|---|
|
Liposome-encapsulated Daunorubicin Time of Maximum Concentration
|
2 HOUR
Interval 1.42 to 2.07
|
SECONDARY outcome
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1Population: Patients in the efficacy phase excluded (n=32). 1 dose finding patient excluded because dose record was not consistent with treatment plan.
Geometric mean liposome-encapsulated daunorubicin area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration will be determined for patients in the dose-finding phase.
Outcome measures
| Measure |
Treatment (CPX-351 and FLAG)
n=5 Participants
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
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|---|---|
|
Liposome-encapsulated Daunorubicin Area Under the Curve
|
1288010.3 (NANOGRAM x HOUR) / MILLILITER
Interval 761251.0 to 1715266.0
|
SECONDARY outcome
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1Population: Patients in the efficacy phase excluded (n=32). 1 dose finding patient excluded because dose record was not consistent with treatment plan.
Geometric mean liposome-encapsulated cytarabine clearance following IV infusion will be determined for patients in the dose-finding phase.
Outcome measures
| Measure |
Treatment (CPX-351 and FLAG)
n=5 Participants
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
|
|---|---|
|
Liposome-encapsulated Cytarabine Clearance
|
71.76 MILLILITER / HOUR
Interval 37.5 to 151.0
|
SECONDARY outcome
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1Population: Patients in the efficacy phase excluded (n=32). 1 dose finding patient excluded because dose record was not consistent with treatment plan.
Geometric mean liposome-encapsulated cytarabine volume of distribution following IV infusion will be determined for patients in the dose-finding phase.
Outcome measures
| Measure |
Treatment (CPX-351 and FLAG)
n=5 Participants
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
|
|---|---|
|
Liposome-encapsulated Cytarabine Volume of Distribution
|
4158.0 MILLILITER
Interval 2642.0 to 7081.0
|
SECONDARY outcome
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1Population: Patients in the efficacy phase excluded (n=32). 1 dose finding patient excluded because dose record was not consistent with treatment plan.
Median liposome-encapsulated cytarabine time of maximum observed plasma concentration will be determined for patients in the dose-finding phase.
Outcome measures
| Measure |
Treatment (CPX-351 and FLAG)
n=5 Participants
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
|
|---|---|
|
Liposome-encapsulated Cytarabine Time of Maximum Concentration
|
5 HOUR
Interval 1.92 to 5.07
|
SECONDARY outcome
Timeframe: Prior to infusion on day 5 and, 45 and 90 minutes post day 5 infusion of cycle 1Population: Patients in the efficacy phase excluded (n=32). 1 dose finding patient excluded because dose record was not consistent with treatment plan.
Geometric mean liposome-encapsulated cytarabine area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration will be determined for patients in the dose-finding phase.
Outcome measures
| Measure |
Treatment (CPX-351 and FLAG)
n=5 Participants
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
|
|---|---|
|
Liposome-encapsulated Cytarabine Area Under the Curve
|
4418582.5 (NANOGRAM x HOUR) / MILLILITER
Interval 2765108.0 to 6382600.0
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 1 yearDescriptive statistics will be used to summarize length of hospitalization time.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 1 yearDescriptive statistics will be used to summarize bone marrow count recovery.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 1 yearDescriptive statistics will be used to summarize peripheral blood cell count recovery.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 8 weeks post-treatmentProportion of patients experiencing \>= grade 3 non-hematologic toxicities, cardiac toxicities, and infections by National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline up to day 30Spearman correlation coefficients will be used to correlate the post-treatment values of troponin with previous cumulative anthracycline dose prior to liposome-encapsulated daunorubicin-cytarabine, change in ejection fraction between pre- and post-liposome-encapsulated daunorubicin-cytarabine baseline assessed by echocardiogram before and after course 1, and change in global longitudinal strain.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline up to day 30Spearman correlation coefficients will be used to correlate the post-treatment values of NT-BNP with previous cumulative anthracycline dose prior to liposome-encapsulated daunorubicin-cytarabine, change in ejection fraction between pre- and post-liposome-encapsulated daunorubicin-cytarabine baseline assessed by echocardiogram before and after course 1, and change in in global longitudinal strain.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline up to day 30Spearman correlation coefficients will be used to correlate the post-treatment values of HS-CRP with previous cumulative anthracycline dose prior to liposome-encapsulated daunorubicin-cytarabine, change in ejection fraction between pre- and post-liposome-encapsulated daunorubicin-cytarabine baseline assessed by echocardiogram before and after course 1, and change in in global longitudinal strain.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline up to 28 daysA paired t-test will be used to compare the mean global longitudinal strain between the pre-treatment (at relapse) baseline and post-course 1 echocardiogram.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline up to day 30Plasma miR-29b and -499 fold change from pre-treatment baseline will be determined at each post-treatment time point using the delta-delta-Ct method. The relationship between 6-hour miR-29b and -499 expression and change in left ventricular global longitudinal strain between the pre-cycle and end of cycle 1 echocardiograms will be determined by calculating a Spearman correlation coefficient.
Outcome measures
Outcome data not reported
Adverse Events
Treatment (CPX-351 and FLAG)
Serious events: 21 serious events
Other events: 34 other events
Deaths: 14 deaths
Serious adverse events
| Measure |
Treatment (CPX-351 and FLAG)
n=38 participants at risk
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
|
|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
28.9%
11/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Gastrointestinal disorders
Dysphagia
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Gastrointestinal disorders
Mucositis oral
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Gastrointestinal disorders
Typhlitis
|
5.3%
2/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
General disorders
Death NOS
|
13.2%
5/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
General disorders
Fever
|
5.3%
2/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Anorectal infection
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Catheter related infection
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Infections and infestations - Other, specify
|
15.8%
6/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Kidney infection
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Lung infection
|
5.3%
2/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Skin infection
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Small intestine infection
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Soft tissue infection
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Investigations
Blood bilirubin increased
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Investigations
Ejection fraction decreased
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Investigations
Lymphocyte count decreased
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Investigations
White blood cell decreased
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Metabolism and nutrition disorders
Anorexia
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
5.3%
2/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Nervous system disorders
Headache
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Nervous system disorders
Nervous system disorders - Other, specify
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Nervous system disorders
Syncope
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Renal and urinary disorders
Urinary frequency
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Reproductive system and breast disorders
Reproductive system and breast disorders - Other, specify
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Respiratory, thoracic and mediastinal disorders
Stridor
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
15.8%
6/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
Other adverse events
| Measure |
Treatment (CPX-351 and FLAG)
n=38 participants at risk
COURSE 1: Patients receive cytarabine IT on day 0 and at day 28-30 or up to 7 days prior to day 1 of course 2, and liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5. Patients with CNS1 receive no further CNS-directed therapy in course 1. Patients with CNS2 disease may receive additional 4-6 doses of cytarabine IT twice weekly starting 48 hours after the third dose of liposome-encapsulated daunorubicin-cytarabine until CNS is clear at the discretion of the investigator. Patients meeting criteria for CR, CRp, and CRi may proceed to course 2.
COURSE 2: Patients receive filgrastim on days 1-5 and then on day 15 until blood count recovery, and fludarabine phosphate IV over 30 minutes and high-dose cytarabine IV over 1-3 hours QD on days 1-5.
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders - Other, specify
|
5.3%
2/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
18.4%
7/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Cardiac disorders
Ventricular arrhythmia
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Gastrointestinal disorders
Rectal pain
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
General disorders
General disorders and administration site conditions - Other, specify
|
10.5%
4/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
General disorders
Multi-organ failure
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Device related infection
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Enterocolitis infectious
|
5.3%
2/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Infections and infestations - Other, specify
|
7.9%
3/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Mucosal infection
|
5.3%
2/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Periorbital infection
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Skin infection
|
7.9%
3/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Infections and infestations
Upper respiratory infection
|
5.3%
2/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Investigations
Alanine aminotransferase increased
|
10.5%
4/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Investigations
Aspartate aminotransferase increased
|
7.9%
3/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Investigations
Ejection fraction decreased
|
5.3%
2/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Investigations
Electrocardiogram QT corrected interval prolonged
|
36.8%
14/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Investigations
GGT increased
|
5.3%
2/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Investigations
Investigations - Other, specify
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Investigations
Platelet count decreased
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Metabolism and nutrition disorders
Anorexia
|
5.3%
2/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
13.2%
5/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
5.3%
2/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
28.9%
11/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
|
Vascular disorders
Hypotension
|
2.6%
1/38 • While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).
Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution, via expedited reporting (NCI AdEERs / CAeRs). The "AE Other" table reflects all CTCAEs collected excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events. All-Cause Mortality includes all deaths collected on the study.
|
Additional Information
Results Reporting Coordinator
Children's Oncology Group
Phone: 626-447-0064
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Must obtain prior sponsor approval
- Publication restrictions are in place
Restriction type: OTHER