Trial Outcomes & Findings for CPX-351 or CLAG-M Regimen for the Treatment of Acute Myeloid Leukemia or Other High-Grade Myeloid Neoplasms in Medically Less-Fit Patients (NCT NCT04195945)

NCT ID: NCT04195945

Last Updated: 2026-05-14

Results Overview

Will evaluate whether CPX-351 or cladribine, cytarabine, granulocyte colony-stimulating factor (G-CSF), and mitoxantrone (CLAG-M), at doses typically used for medically-fit adults with AML, improve 3-month overall survival in medically-unfit adults with AML compared to attenuated dose CPX-351 as used in our previous institutional trial (32 units/m2 per dose).

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

60 participants

Primary outcome timeframe

Up to 3 months from date of start of protocol therapy

Results posted on

2026-05-14

Participant Flow

Participants were recruited based on physician referral at University of Washington Medical Center between May 2020 and March 2025. The frist participant was enrolled on May 27, 2020 and the last participant was enrolled on March 3, 2025.

Participant milestones

Participant milestones
Measure	CPX-351 Induction Therapy Patients will receive CPX-351 at a dose of daunorubicin 44 mg/m\^2, cytarabine 100 mg/m\^2 on days 1, 3, and 5. CPX-351 should be administered IV over 90 minutes.	CLAG-M Induction Therapy The doses of the elements of CLAG-M will be as follows: cladribine 5 mg/m2 IV daily over 2 hours on Days 1-5; cytarabine 2,000 mg/m2 IV daily over 2 hours on Days 1-5; G-CSF 300 or 480 μcg/ (based on weight \<76 kg vs. ≥76kg) daily subcutaneously daily on Days 0-5; and Mitoxantrone 18 mg/m2 IV daily over 60 minutes on Days 1-3. Note that often day 0 G-CSF is given as outpatient and the remainder of therapy is given as inpatient. Thus, a \>24-hour delay between day 0 G-CSF and the start of day 1 therapy is allowed.
Overall Study STARTED	30	30
Overall Study Completed 1 Cycle of Therapy	23	27
Overall Study Completed 2 Cycles of Therapy	6	1
Overall Study Completed 3 Cycles of Therapy	0	1
Overall Study Completed 4 Cycles of Therapy	1	0
Overall Study Completed 5 Cycles of Therapy	0	1
Overall Study Completed 6 Cycles of Therapy	0	0
Overall Study COMPLETED	30	30
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

CPX-351 or CLAG-M Regimen for the Treatment of Acute Myeloid Leukemia or Other High-Grade Myeloid Neoplasms in Medically Less-Fit Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CLAG-M Induction Therapy n=30 Participants The doses of the elements of CLAG-M will be as follows: cladribine 5 mg/m\^2 IV	Total n=60 Participants Total of all reporting groups	CPX-351 Induction Therapy n=30 Participants Patients will receive CPX-351 at a dose of daunorubicin 44 mg/m\^2, cytarabine 100 mg/m\^2 on days 1, 3, and 5. CPX-351 should be administered IV over 90 minutes.
Age, Categorical <=18 years	0 Participants n=504 Participants	0 Participants n=2016 Participants	0 Participants n=1512 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=504 Participants	12 Participants n=2016 Participants	5 Participants n=1512 Participants
Age, Categorical >=65 years	23 Participants n=504 Participants	48 Participants n=2016 Participants	25 Participants n=1512 Participants
Age, Continuous	72 Years n=504 Participants	72 Years n=2016 Participants	72 Years n=1512 Participants
Sex: Female, Male Female	6 Participants n=504 Participants	17 Participants n=2016 Participants	11 Participants n=1512 Participants
Sex: Female, Male Male	24 Participants n=504 Participants	43 Participants n=2016 Participants	19 Participants n=1512 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=504 Participants	0 Participants n=2016 Participants	0 Participants n=1512 Participants
Race (NIH/OMB) Asian	0 Participants n=504 Participants	2 Participants n=2016 Participants	2 Participants n=1512 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=504 Participants	0 Participants n=2016 Participants	0 Participants n=1512 Participants
Race (NIH/OMB) Black or African American	2 Participants n=504 Participants	2 Participants n=2016 Participants	0 Participants n=1512 Participants
Race (NIH/OMB) White	26 Participants n=504 Participants	53 Participants n=2016 Participants	27 Participants n=1512 Participants
Race (NIH/OMB) More than one race	0 Participants n=504 Participants	0 Participants n=2016 Participants	0 Participants n=1512 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=504 Participants	3 Participants n=2016 Participants	1 Participants n=1512 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=504 Participants	3 Participants n=2016 Participants	1 Participants n=1512 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	27 Participants n=504 Participants	56 Participants n=2016 Participants	29 Participants n=1512 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=504 Participants	1 Participants n=2016 Participants	0 Participants n=1512 Participants
Region of Enrollment United States	30 Participants n=504 Participants	60 Participants n=2016 Participants	30 Participants n=1512 Participants

PRIMARY outcome

Timeframe: Up to 3 months from date of start of protocol therapy

Population: Count of participants (and percentage) alive, deceased, and unknown at 3-months after start of protocol therapy

Outcome measures

Outcome measures
Measure	CPX-351 Induction Therapy n=30 Participants Patients will receive CPX-351 at a dose of daunorubicin 44 mg/m\^2, cytarabine 100 mg/m\^2 on days 1, 3, and 5. CPX-351 should be administered IV over 90 minutes.	CLAG-M Induction Therapy n=30 Participants The doses of the elements of CLAG-M will be as follows: cladribine 5 mg/m\^2 IV
3-month Overall Survival (OS) Alive	17 Participants	21 Participants
3-month Overall Survival (OS) Deceased	12 Participants	9 Participants
3-month Overall Survival (OS) Unknown	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to 5 years post treatment

Population: Count of participants (and percentage) who achieved CR or CRi as a best response

Will compare CR rates between the study arms.

Outcome measures

Outcome measures
Measure	CPX-351 Induction Therapy n=30 Participants Patients will receive CPX-351 at a dose of daunorubicin 44 mg/m\^2, cytarabine 100 mg/m\^2 on days 1, 3, and 5. CPX-351 should be administered IV over 90 minutes.	CLAG-M Induction Therapy n=30 Participants The doses of the elements of CLAG-M will be as follows: cladribine 5 mg/m\^2 IV
Complete Remission (CR) Rates	14 Participants	22 Participants

SECONDARY outcome

Timeframe: Up to 5 years post treatment

Population: Count of participants (and percentage) who achieved CR (MRDNEG) or CRi(MRDNEG)

Will compare MRDneg CR rates between the study arms.

Outcome measures

Outcome measures
Measure	CPX-351 Induction Therapy n=30 Participants Patients will receive CPX-351 at a dose of daunorubicin 44 mg/m\^2, cytarabine 100 mg/m\^2 on days 1, 3, and 5. CPX-351 should be administered IV over 90 minutes.	CLAG-M Induction Therapy n=30 Participants The doses of the elements of CLAG-M will be as follows: cladribine 5 mg/m\^2 IV
MRDneg CR Rates	10 Participants	18 Participants

SECONDARY outcome

Timeframe: Up to 5 years post treatment

Population: Response duration was only analyzed for participants who relapsed, because response duration requires a relapse date to be calculated. The remaining participants did not relapse while data was collected and so their response duration is at least as long as the length of time that data was collected.

Will compare response duration between the study arms.

Outcome measures

Outcome measures
Measure	CPX-351 Induction Therapy n=10 Participants Patients will receive CPX-351 at a dose of daunorubicin 44 mg/m\^2, cytarabine 100 mg/m\^2 on days 1, 3, and 5. CPX-351 should be administered IV over 90 minutes.	CLAG-M Induction Therapy n=17 Participants The doses of the elements of CLAG-M will be as follows: cladribine 5 mg/m\^2 IV
Response Duration	4.7 Months Interval 1.09 to 9.53	7.06 Months Interval 2.53 to 14.65

SECONDARY outcome

Timeframe: Up to 5 years post treatment

Will compare RFS between the study arms.

Outcome measures

Outcome measures
Measure	CPX-351 Induction Therapy n=30 Participants Patients will receive CPX-351 at a dose of daunorubicin 44 mg/m\^2, cytarabine 100 mg/m\^2 on days 1, 3, and 5. CPX-351 should be administered IV over 90 minutes.	CLAG-M Induction Therapy n=30 Participants The doses of the elements of CLAG-M will be as follows: cladribine 5 mg/m\^2 IV
Relapse-free Survival	19.9 Months Interval 19.9 to Insufficient number of participants with events	37.6 Months Interval 37.6 to Insufficient number of participants with events

SECONDARY outcome

Timeframe: AEs were recorded from the time of first exposure to the therapy (start of the first drug administration infusion on day 1) until 4 weeks after the last dose. All-Cause Mortality was monitored from study start to data cut-off dates 5/27/2020 to 9/18/2025

Will use the CTCAE (National Cancer Institute \[NCI\] Common Terminology Criteria for Adverse Events) version 5.0 for toxicity and adverse event reporting. Will describe the toxicity profile and infectious complications of each study treatment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: At 30 days

30-day mortality rate from start of treatment

Outcome measures

Outcome measures
Measure	CPX-351 Induction Therapy n=30 Participants Patients will receive CPX-351 at a dose of daunorubicin 44 mg/m\^2, cytarabine 100 mg/m\^2 on days 1, 3, and 5. CPX-351 should be administered IV over 90 minutes.	CLAG-M Induction Therapy n=30 Participants The doses of the elements of CLAG-M will be as follows: cladribine 5 mg/m\^2 IV
30-day Mortality Rate Alive	22 Participants	24 Participants
30-day Mortality Rate Deceased	7 Participants	6 Participants
30-day Mortality Rate Unknown	1 Participants	0 Participants

SECONDARY outcome

Timeframe: At 60 days

60-day mortality rate from start of treatment

Outcome measures

Outcome measures
Measure	CPX-351 Induction Therapy n=30 Participants Patients will receive CPX-351 at a dose of daunorubicin 44 mg/m\^2, cytarabine 100 mg/m\^2 on days 1, 3, and 5. CPX-351 should be administered IV over 90 minutes.	CLAG-M Induction Therapy n=30 Participants The doses of the elements of CLAG-M will be as follows: cladribine 5 mg/m\^2 IV
60-day Mortality Alive	18 Participants	23 Participants
60-day Mortality Deceased	11 Participants	7 Participants
60-day Mortality Unknown	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to 5 years post treatment

Population: Only eight patients in the CPX-351 arm and ten in the CLAG-M arm completed both baseline/pre-treatment and post cycle 1 QOL, preventing an unbiased analysis of treatment effect on patient reported measures of QOL and functional status.

QOL of patients will be assessed longitudinally using the European Organization for Research and Treatment of Cancer (EORTC) core QOL questionnaire (QLQ-C30) and the newly developed acute myeloid leukemia-specific QOL instrument. QOL measures for Global Health Status (GHS), Functional Score, and Symptom Score were scored on a 0-100 scale with lower scores indicating worse outcomes.

Outcome measures

Outcome measures
Measure	CPX-351 Induction Therapy n=20 Participants Patients will receive CPX-351 at a dose of daunorubicin 44 mg/m\^2, cytarabine 100 mg/m\^2 on days 1, 3, and 5. CPX-351 should be administered IV over 90 minutes.	CLAG-M Induction Therapy n=22 Participants The doses of the elements of CLAG-M will be as follows: cladribine 5 mg/m\^2 IV
Quality of Life (QOL): Questionnaire Global Health Status	33 Patient-reported score Interval 0.0 to 75.0	29 Patient-reported score Interval 0.0 to 100.0
Quality of Life (QOL): Questionnaire Symptom score	44 Patient-reported score Interval 21.0 to 72.0	44 Patient-reported score Interval 10.0 to 82.0
Quality of Life (QOL): Questionnaire Functional score	51 Patient-reported score Interval 9.0 to 84.0	53 Patient-reported score Interval 0.0 to 91.0

SECONDARY outcome

Timeframe: Up to 5 years post treatment

Population: Number of units transfused for platelets and red blood cells for each arm

Electronic medical chart review to enumerate the number of platelet and red blood cell transfusions administered.

Outcome measures

Outcome measures
Measure	CPX-351 Induction Therapy n=30 Participants Patients will receive CPX-351 at a dose of daunorubicin 44 mg/m\^2, cytarabine 100 mg/m\^2 on days 1, 3, and 5. CPX-351 should be administered IV over 90 minutes.	CLAG-M Induction Therapy n=30 Participants The doses of the elements of CLAG-M will be as follows: cladribine 5 mg/m\^2 IV
Patient Use of Medical Resources (e.g. Transfusions) Number of units red blood cells transfused	9.00 Units Interval 6.0 to 12.75	10 Units Interval 7.0 to 14.0
Patient Use of Medical Resources (e.g. Transfusions) Number of units of platelet transfused	11.5 Units Interval 6.25 to 18.0	13.5 Units Interval 8.0 to 16.0

SECONDARY outcome

Timeframe: Up to 5 years post treatment

Population: Days

Electronic medical chart review to enumerate the number of days spent on IV antimicrobial therapy and the number of days spent in the ICU.

Outcome measures

Outcome measures
Measure	CPX-351 Induction Therapy n=30 Participants Patients will receive CPX-351 at a dose of daunorubicin 44 mg/m\^2, cytarabine 100 mg/m\^2 on days 1, 3, and 5. CPX-351 should be administered IV over 90 minutes.	CLAG-M Induction Therapy n=30 Participants The doses of the elements of CLAG-M will be as follows: cladribine 5 mg/m\^2 IV
Patient Use of Medical Resources (e.g. Transfusions) Number of days spent on IV antimicrobials	8.50 Days Interval 3.5 to 11.0	8 Days Interval 1.25 to 14.5
Patient Use of Medical Resources (e.g. Transfusions) Number of days spent in ICU	0 Days Interval 0.0 to 0.0	0 Days Interval 0.0 to 0.75

Adverse Events

CPX-351 Induction Therapy

Serious events: 2 serious events

Other events: 30 other events

Deaths: 22 deaths

CLAG-M Induction Therapy

Serious events: 6 serious events

Other events: 30 other events

Deaths: 25 deaths

Serious adverse events

Serious adverse events
Measure	CPX-351 Induction Therapy n=30 participants at risk Patients will receive CPX-351 at a dose of daunorubicin 44 mg/m\^2, cytarabine 100 mg/m\^2 on days 1, 3, and 5. CPX-351 should be administered IV over 90 minutes.	CLAG-M Induction Therapy n=30 participants at risk The doses of the elements of CLAG-M will be as follows: cladribine 5 mg/m\^2 IV
Nervous system disorders Stroke	0.00% 0/30 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.	3.3% 1/30 • Number of events 1 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.
Vascular disorders Hematoma	0.00% 0/30 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.	3.3% 1/30 • Number of events 1 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.
Respiratory, thoracic and mediastinal disorders Respiratory failure	6.7% 2/30 • Number of events 2 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.	10.0% 3/30 • Number of events 3 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.
Infections and infestations Bacteremia	0.00% 0/30 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.	3.3% 1/30 • Number of events 1 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.
Infections and infestations Lung infection	0.00% 0/30 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.	3.3% 1/30 • Number of events 1 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.
Infections and infestations Sepsis	0.00% 0/30 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.	3.3% 1/30 • Number of events 1 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.
Metabolism and nutrition disorders Tumor lysis syndrome	0.00% 0/30 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.	3.3% 1/30 • Number of events 1 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.
Nervous system disorders Encephalopathy	0.00% 0/30 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.	3.3% 1/30 • Number of events 1 • Serious and other AEs are monitored and recorded in the study database from the time of first exposure to the therapy in this study (i.e., the start of the first drug administration infusion on day 1) until 4 weeks after the last dose of study treatment, an average of 40.4 days. All-cause mortality is assessed for up to a maximum of 5 years post-transplant.

Other adverse events

Additional Information

Dr. Anna Halpern

Fred Hutchinson Cancer Center

Phone: (206) 606-1978

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place