Trial Outcomes & Findings for Vyxeos(CPX-351) in Adults w R/R Acute Lymphoblastic Leukemia (NCT NCT03575325)

Last Updated: 2024-12-12

Results Overview

Expansion from phase II pilot to a phase II trial will depend on demonstration of CR/CRi amongst 4 of the initial 10 treated patients. Investigators will measure remission rate at day 28 to address the primary endpoint of complete remission (with or without complete hematologic recovery), as defined by Cheson Criteria (ref 27). For those with extramedullary disease, Lugano criteria will be used to assess response. This is a standard assessment of drug efficacy for phase 2 clinical trial design in acute leukemias, as response correlates closely with progression free- and overall survival (PFS and OS).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

11 participants

Primary outcome timeframe

At day 28

Results posted on

2024-12-12

Participant Flow

Participant milestones

Participant milestones
Measure	CPX-351 Treatment Participants will receive induction with CPX-351 at a dose of 100 u/m\^2 administered intravenously over 90 minutes on days 1, 3 and 5 of a 28 day cycle. This may be followed by consolidation with CPX-351 at a dose of 65 u/m\^2 administered intravenously over 90 minutes on days 1 and 3 of a 28 day cycle (up to 3 cycles). CPX-351: The infusion of CPX-351 (cytarabine:daunorubicin) Liposome Injection will be performed through a central venous catheter, using an infusion pump to ensure that the drug is infused over the specified time period.
Overall Study STARTED	11
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	CPX-351 Treatment Participants will receive induction with CPX-351 at a dose of 100 u/m\^2 administered intravenously over 90 minutes on days 1, 3 and 5 of a 28 day cycle. This may be followed by consolidation with CPX-351 at a dose of 65 u/m\^2 administered intravenously over 90 minutes on days 1 and 3 of a 28 day cycle (up to 3 cycles). CPX-351: The infusion of CPX-351 (cytarabine:daunorubicin) Liposome Injection will be performed through a central venous catheter, using an infusion pump to ensure that the drug is infused over the specified time period.
Overall Study Death	1

Baseline Characteristics

Vyxeos(CPX-351) in Adults w R/R Acute Lymphoblastic Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CPX-351 Treatment n=11 Participants Participants will receive induction with CPX-351 at a dose of 100 u/m\^2 administered intravenously over 90 minutes on days 1, 3 and 5 of a 28 day cycle. This may be followed by consolidation with CPX-351 at a dose of 65 u/m\^2 administered intravenously over 90 minutes on days 1 and 3 of a 28 day cycle (up to 3 cycles). CPX-351: The infusion of CPX-351 (cytarabine:daunorubicin) Liposome Injection will be performed through a central venous catheter, using an infusion pump to ensure that the drug is infused over the specified time period.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Region of Enrollment United States	11 participants n=5 Participants

PRIMARY outcome

Timeframe: At day 28

Population: Evaluable participants

Outcome measures

Outcome measures
Measure	CPX-351 Treatment n=10 Participants Participants will receive induction with CPX-351 at a dose of 100 u/m\^2 administered intravenously over 90 minutes on days 1, 3 and 5 of a 28 day cycle. This may be followed by consolidation with CPX-351 at a dose of 65 u/m\^2 administered intravenously over 90 minutes on days 1 and 3 of a 28 day cycle (up to 3 cycles). CPX-351: The infusion of CPX-351 (cytarabine:daunorubicin) Liposome Injection will be performed through a central venous catheter, using an infusion pump to ensure that the drug is infused over the specified time period.
Complete Remission Rate (CR) + CR With Incomplete Recovery (CRi)	30 Percent of participants

SECONDARY outcome

Timeframe: 12 months

Population: Evaluable participants

Progression Free Survival as defined by Cheson and Lugano Criteria, namely progression, failure to respond, or death, as assessed from time of first treatment. Progression Free Survival will be reported as per Cheson Criteria, and analyzed using a standard Kaplan-Meier approach. Patients will undergo bone marrow biopsy evaluation after each cycle of therapy per standard of care to facilitated assessment.

Outcome measures

Outcome measures
Measure	CPX-351 Treatment n=10 Participants Participants will receive induction with CPX-351 at a dose of 100 u/m\^2 administered intravenously over 90 minutes on days 1, 3 and 5 of a 28 day cycle. This may be followed by consolidation with CPX-351 at a dose of 65 u/m\^2 administered intravenously over 90 minutes on days 1 and 3 of a 28 day cycle (up to 3 cycles). CPX-351: The infusion of CPX-351 (cytarabine:daunorubicin) Liposome Injection will be performed through a central venous catheter, using an infusion pump to ensure that the drug is infused over the specified time period.
Progression Free Survival (PFS)	57 Days Interval 10.0 to 105.0

SECONDARY outcome

Timeframe: 12 months

Population: Evaluable participants

Overall Survival as defined by Cheson and Lygano Criteria, namely death due to any cause as assessed from time of first treatment. Overall Survival will be reported as per Cheson Criteria, and analyzed using a standard Kaplan-Meier approach. Patients will undergo bone marrow biopsy evaluation after each cycle of therapy per standard of care to facilitated assessment.

Outcome measures

Outcome measures
Measure	CPX-351 Treatment n=10 Participants Participants will receive induction with CPX-351 at a dose of 100 u/m\^2 administered intravenously over 90 minutes on days 1, 3 and 5 of a 28 day cycle. This may be followed by consolidation with CPX-351 at a dose of 65 u/m\^2 administered intravenously over 90 minutes on days 1 and 3 of a 28 day cycle (up to 3 cycles). CPX-351: The infusion of CPX-351 (cytarabine:daunorubicin) Liposome Injection will be performed through a central venous catheter, using an infusion pump to ensure that the drug is infused over the specified time period.
Overall Survival (OS)	218 Days Interval 161.0 to 275.0

SECONDARY outcome

Timeframe: At day 28

Population: Evaluable participants - Participants who achieved CR or CRi

MRD assessment will be provided using ClonoSeq test result. ClonoSEQ® is an FDA-cleared test used to detect minimal residual disease (MRD) in bone marrow from patients with multiple myeloma or B-cell acute lymphoblastic leukemia (B-ALL) and blood or bone marrow from patients with chronic lymphocytic leukemia (CLL).

Outcome measures

Outcome measures
Measure	CPX-351 Treatment n=3 Participants Participants will receive induction with CPX-351 at a dose of 100 u/m\^2 administered intravenously over 90 minutes on days 1, 3 and 5 of a 28 day cycle. This may be followed by consolidation with CPX-351 at a dose of 65 u/m\^2 administered intravenously over 90 minutes on days 1 and 3 of a 28 day cycle (up to 3 cycles). CPX-351: The infusion of CPX-351 (cytarabine:daunorubicin) Liposome Injection will be performed through a central venous catheter, using an infusion pump to ensure that the drug is infused over the specified time period.
Minimal Residual Disease (MRD) No trackable clone by ClonoSeq	2 Participants
Minimal Residual Disease (MRD) Complete Molecular Remission by ClonoSeq	1 Participants

Adverse Events

CPX-351 Treatment Induction Dose

Serious events: 2 serious events

Other events: 7 other events

Deaths: 1 deaths

CPX-351 Treatment Induction Dose + 1 Consolidation Dose

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

CPX-351 Treatment +2 Consolidation Doses

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	CPX-351 Treatment Induction Dose n=7 participants at risk Participants who received induction with CPX-351 at a dose of 100 u/m\^2 administered intravenously over 90 minutes on days 1, 3 and 5 of a 28 day cycle.	CPX-351 Treatment Induction Dose + 1 Consolidation Dose n=3 participants at risk Participants who received induction with CPX-351 at a dose of 100 u/m\^2 administered intravenously over 90 minutes on days 1, 3 and 5 of a 28 day cycle. Participants then received one consolidation dose of CPX-351 at a dose of 65 u/m\^2 administered intravenously over 90 minutes on days 1 and 3 of a 28 day cycle	CPX-351 Treatment +2 Consolidation Doses n=1 participants at risk Participants who received induction with CPX-351 at a dose of 100 u/m\^2 administered intravenously over 90 minutes on days 1, 3 and 5 of a 28 day cycle. Participants then received two consolidation doses of CPX-351 at a dose of 65 u/m\^2 administered intravenously over 90 minutes on days 1 and 3 of a 28 day cycle
Respiratory, thoracic and mediastinal disorders Respiratory failure	14.3% 1/7 • Number of events 1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	0.00% 0/3 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	0.00% 0/1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.
Infections and infestations Lung Infection	14.3% 1/7 • Number of events 1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	0.00% 0/3 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	0.00% 0/1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.
Infections and infestations Soft tissue infection	0.00% 0/7 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	0.00% 0/3 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	100.0% 1/1 • Number of events 1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.
Gastrointestinal disorders Gastric hemorrhage	14.3% 1/7 • Number of events 1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	0.00% 0/3 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	0.00% 0/1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.
Cardiac disorders Myopericarditis	0.00% 0/7 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	0.00% 0/1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.
Investigations Alanine aminotransferase increased	0.00% 0/7 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	0.00% 0/1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.
Investigations Aspartate aminotransferase increased	0.00% 0/7 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	0.00% 0/1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.
Investigations Blood bilirubin increased	0.00% 0/7 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.	0.00% 0/1 • Adverse events were collected from the time of consent until 30 days after the last dose of CPX-351, approximately 34 months.

Other adverse events

Additional Information

Bijal D. Shah, MD, MS

Moffitt Cancer Center

Phone: 813-745-4294

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place