Trial Outcomes & Findings for Liposomal Cytarabine-Daunorubicin CPX-351 in Treating Patients With Untreated Myelodysplastic Syndrome or Acute Myeloid Leukemia (NCT NCT01804101)

Last Updated: 2018-05-25

Results Overview

Estimate whether the 32 units/m2 or the 64 units/m2 or both dose levels of CPX-351 are likely to improve treatment-related mortality (TRM) rate while keeping the CR rate constant in patients with untreated high-risk MDS or non-APL AML at high risk of TRM.

Recruitment status

COMPLETED

Study phase

Target enrollment

48 participants

Primary outcome timeframe

Up to 1 month after completion of study treatment

Results posted on

2018-05-25

Participant Flow

Participant milestones

Participant milestones
Measure	Arm I (Lower-dose CPX-351) INDUCTION/RE-INDUCTION: Patients receive lower-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving CR or CRp continue on to consolidation. CONSOLIDATION: Patients receive lower-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1 and 3. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Liposomal Cytarabine-Daunorubicin CPX-351: Given IV	Arm II (Higher Dose COX-351) INDUCTION/RE-INDUCTION: Patients receive higher-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving CR or CRp continue on to consolidation. CONSOLIDATION: Patients receive higher-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1 and 3. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Liposomal Cytarabine-Daunorubicin CPX-351: Given IV
Overall Study STARTED	38	10
Overall Study COMPLETED	38	10
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Liposomal Cytarabine-Daunorubicin CPX-351 in Treating Patients With Untreated Myelodysplastic Syndrome or Acute Myeloid Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arm I (Lower-dose Liposomal Cytarabine-daunorubicin CPX-351) n=38 Participants INDUCTION/RE-INDUCTION: Patients receive lower-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving CR or CRp continue on to consolidation. CONSOLIDATION: Patients receive lower-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1 and 3. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Liposomal Cytarabine-Daunorubicin CPX-351: Given IV	Arm II (Closed to Accrual Effective 4/21/14) n=10 Participants INDUCTION/RE-INDUCTION: Patients receive higher-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving CR or CRp continue on to consolidation. CONSOLIDATION: Patients receive higher-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1 and 3. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Liposomal Cytarabine-Daunorubicin CPX-351: Given IV	Total n=48 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants	2 Participants n=7 Participants	12 Participants n=5 Participants
Age, Categorical >=65 years	28 Participants n=5 Participants	8 Participants n=7 Participants	36 Participants n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants	3 Participants n=7 Participants	16 Participants n=5 Participants
Sex: Female, Male Male	25 Participants n=5 Participants	7 Participants n=7 Participants	32 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	38 Participants n=5 Participants	10 Participants n=7 Participants	48 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	3 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	35 Participants n=5 Participants	10 Participants n=7 Participants	45 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	38 Participants n=5 Participants	10 Participants n=7 Participants	48 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 1 month after completion of study treatment

Outcome measures

Outcome measures
Measure	Arm I (Lower-dose Liposomal Cytarabine-daunorubicin CPX-351) n=38 Participants INDUCTION/RE-INDUCTION: Patients receive lower-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving CR or CRp continue on to consolidation. CONSOLIDATION: Patients receive lower-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1 and 3. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Liposomal Cytarabine-Daunorubicin CPX-351: Given IV	Arm II (Closed to Accrual Effective 4/21/14) n=10 Participants INDUCTION/RE-INDUCTION: Patients receive higher-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving CR or CRp continue on to consolidation. CONSOLIDATION: Patients receive higher-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1 and 3. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Liposomal Cytarabine-Daunorubicin CPX-351: Given IV
Treatment-related Mortality Rate. (TRM)	6 Participants	2 Participants

SECONDARY outcome

Timeframe: Up to day 28

Overall Remission Rate for the CR/CR with incomplete platelet count recovery (CRp) rate after up to 4 cycles of induction/re-induction therapy.

Outcome measures

Outcome measures
Measure	Arm I (Lower-dose Liposomal Cytarabine-daunorubicin CPX-351) n=38 Participants INDUCTION/RE-INDUCTION: Patients receive lower-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving CR or CRp continue on to consolidation. CONSOLIDATION: Patients receive lower-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1 and 3. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Liposomal Cytarabine-Daunorubicin CPX-351: Given IV	Arm II (Closed to Accrual Effective 4/21/14) n=10 Participants INDUCTION/RE-INDUCTION: Patients receive higher-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving CR or CRp continue on to consolidation. CONSOLIDATION: Patients receive higher-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1 and 3. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Liposomal Cytarabine-Daunorubicin CPX-351: Given IV
Overall Remission Rate (CR+CRp)	10 Participants	1 Participants

Adverse Events

Arm I (Lower-dose Liposomal Cytarabine-daunorubicin CPX-351)

Serious events: 0 serious events

Other events: 38 other events

Deaths: 10 deaths

Arm II (Closed to Accrual Effective 4/21/14)

Serious events: 0 serious events

Other events: 10 other events

Deaths: 5 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Arm I (Lower-dose Liposomal Cytarabine-daunorubicin CPX-351) n=38 participants at risk INDUCTION/RE-INDUCTION: Patients receive lower-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving CR or CRp continue on to consolidation. CONSOLIDATION: Patients receive lower-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1 and 3. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Liposomal Cytarabine-Daunorubicin CPX-351: Given IV	Arm II (Closed to Accrual Effective 4/21/14) n=10 participants at risk INDUCTION/RE-INDUCTION: Patients receive higher-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving CR or CRp continue on to consolidation. CONSOLIDATION: Patients receive higher-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1 and 3. Treatment repeats every 40 days for 4 courses in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Liposomal Cytarabine-Daunorubicin CPX-351: Given IV
Infections and infestations Neutroprenic Fever	13.2% 5/38 • Number of events 5 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.	20.0% 2/10 • Number of events 2 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.
Endocrine disorders Increased Creatinine	7.9% 3/38 • Number of events 3 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.	0.00% 0/10 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.
Blood and lymphatic system disorders epistaxis	5.3% 2/38 • Number of events 2 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.	0.00% 0/10 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.
Infections and infestations PICC Line infection	2.6% 1/38 • Number of events 1 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.	10.0% 1/10 • Number of events 1 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.
Gastrointestinal disorders Constipation	13.2% 5/38 • Number of events 5 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.	0.00% 0/10 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.
General disorders Fatigue	21.1% 8/38 • Number of events 8 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.	20.0% 2/10 • Number of events 2 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.
Infections and infestations Neutropenia	23.7% 9/38 • Number of events 9 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.	30.0% 3/10 • Number of events 3 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.
Blood and lymphatic system disorders Thrombocytopenia	18.4% 7/38 • Number of events 7 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.	20.0% 2/10 • Number of events 2 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.
Blood and lymphatic system disorders Leukopenia	7.9% 3/38 • Number of events 3 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.	10.0% 1/10 • Number of events 1 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.
Gastrointestinal disorders Diarrhea	7.9% 3/38 • Number of events 3 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.	0.00% 0/10 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.
Metabolism and nutrition disorders Nausea	10.5% 4/38 • Number of events 4 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.	10.0% 1/10 • Number of events 1 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.
Infections and infestations hypoxemia	5.3% 2/38 • Number of events 2 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.	10.0% 1/10 • Number of events 1 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.
General disorders Edema	13.2% 5/38 • Number of events 5 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.	0.00% 0/10 • 3 years SAE reporting per our institution, is if it's related and unexpected. None of our AE's met this criteria per protocol. AE's are were review by study coordinator and PI threw medical records and outside records from patients other physicians.

Additional Information

Dr. Roland B. Walter

Fred Hutchinson Cancer Center

Phone: 206-667-3599

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place