Trial Outcomes & Findings for Investigator Initiated Trial of CPX-351 for Untreated Acute Myeloid Leukemia (NCT NCT03335267)
NCT ID: NCT03335267
Last Updated: 2024-02-22
Results Overview
Overall survival is measured from the date of registration to death from any cause. Patients not known to have died will be censored on the date they were last known to be alive. Patients were followed for 2.5 years.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
30 participants
Primary outcome timeframe
2.5 Years
Results posted on
2024-02-22
Participant Flow
Participant milestones
| Measure |
CPX-351 (Cytarabine:Daunorubicin) Injection
Dosing for first induction: CPX-351
• CPX-351 at 100u/m2 will be administered on study days 1, 3 and 5
Dosing for second induction:
• CPX-351 at 100 u/m2 will be administered on days 1 and 3
Dosing for consolidation:
• CPX-351 at 65 u/m2 will be administered on days 1 and 3
CPX-351: Cytarabine:Daunorubicin Liposome Injection
|
|---|---|
|
Overall Study
STARTED
|
30
|
|
Overall Study
COMPLETED
|
30
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Investigator Initiated Trial of CPX-351 for Untreated Acute Myeloid Leukemia
Baseline characteristics by cohort
| Measure |
CPX-351 (Cytarabine:Daunorubicin) Injection
n=30 Participants
Dosing for first induction: CPX-351
• CPX-351 at 100u/m2 will be administered on study days 1, 3 and 5
Dosing for second induction:
• CPX-351 at 100 u/m2 will be administered on days 1 and 3
Dosing for consolidation:
• CPX-351 at 65 u/m2 will be administered on days 1 and 3
CPX-351: Cytarabine:Daunorubicin Liposome Injection
|
|---|---|
|
Age, Continuous
|
70.98 Years
n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
17 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
10 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
15 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
9 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
30 Participants
n=5 Participants
|
|
Newly Diagnosed AML Patients Aged ≥ 60 Years
|
30 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2.5 YearsOverall survival is measured from the date of registration to death from any cause. Patients not known to have died will be censored on the date they were last known to be alive. Patients were followed for 2.5 years.
Outcome measures
| Measure |
CPX-351 (Cytarabine:Daunorubicin) Injection
n=30 Participants
Dosing for first induction: CPX-351
• CPX-351 at 100u/m2 will be administered on study days 1, 3 and 5
Dosing for second induction:
• CPX-351 at 100 u/m2 will be administered on days 1 and 3
Dosing for consolidation:
• CPX-351 at 65 u/m2 will be administered on days 1 and 3
CPX-351: Cytarabine:Daunorubicin Liposome Injection
|
|---|---|
|
Primary Efficacy
|
7 Participants
|
PRIMARY outcome
Timeframe: 30 DaysMortality rate at Day 30
Outcome measures
| Measure |
CPX-351 (Cytarabine:Daunorubicin) Injection
n=30 Participants
Dosing for first induction: CPX-351
• CPX-351 at 100u/m2 will be administered on study days 1, 3 and 5
Dosing for second induction:
• CPX-351 at 100 u/m2 will be administered on days 1 and 3
Dosing for consolidation:
• CPX-351 at 65 u/m2 will be administered on days 1 and 3
CPX-351: Cytarabine:Daunorubicin Liposome Injection
|
|---|---|
|
30-Day Mortality Rate
|
2 Participants
|
SECONDARY outcome
Timeframe: 30 days post-treatment, up to 3 months post-baselinePopulation: 14 subjects were excluded from analysis for this measure due to treatment failure (progressive disease or non-evaluable).
The number of patients who achieve response will be divided by the number of patients in the efficacy population to determine response rate.
Outcome measures
| Measure |
CPX-351 (Cytarabine:Daunorubicin) Injection
n=16 Participants
Dosing for first induction: CPX-351
• CPX-351 at 100u/m2 will be administered on study days 1, 3 and 5
Dosing for second induction:
• CPX-351 at 100 u/m2 will be administered on days 1 and 3
Dosing for consolidation:
• CPX-351 at 65 u/m2 will be administered on days 1 and 3
CPX-351: Cytarabine:Daunorubicin Liposome Injection
|
|---|---|
|
Complete Response Rate (CR, CRp, CRi, and CR+CRp+CRi)
Complete remission (CR)
|
11 Participants
|
|
Complete Response Rate (CR, CRp, CRi, and CR+CRp+CRi)
Complete remission with incomplete recovery (CRi)
|
5 Participants
|
SECONDARY outcome
Timeframe: Screening through 30 days post-treatment, up to 3 months post-baselinePopulation: 9 subjects did not complete the MOCA and they were excluded from analysis for this measure.
The Montreal Cognitive Assessment (MOCA) is a 30-point test which assesses several cognitive domains. Possible total scores range from 0 to 30. The results can be interpreted as follows: normal cognition: 26-30 points, mild cognitive impairment: 18-25 points, moderate cognitive impairment: 10-17 points, and severe cognitive impairment: under 10 points.
Outcome measures
| Measure |
CPX-351 (Cytarabine:Daunorubicin) Injection
n=21 Participants
Dosing for first induction: CPX-351
• CPX-351 at 100u/m2 will be administered on study days 1, 3 and 5
Dosing for second induction:
• CPX-351 at 100 u/m2 will be administered on days 1 and 3
Dosing for consolidation:
• CPX-351 at 65 u/m2 will be administered on days 1 and 3
CPX-351: Cytarabine:Daunorubicin Liposome Injection
|
|---|---|
|
Change in Relationship of Cognitive Function to Treatment Response and OS, Event-free Survival and Morphologic Leukemia Free State as Measured by the MOCA
Montreal Cognitive Assessment (baseline)
|
24.72 score on a scale
Standard Deviation 2.85
|
|
Change in Relationship of Cognitive Function to Treatment Response and OS, Event-free Survival and Morphologic Leukemia Free State as Measured by the MOCA
Montreal Cognitive Assessment (30 days post-treatment)
|
25.79 score on a scale
Standard Deviation 2.99
|
SECONDARY outcome
Timeframe: Screening through 30 days post-treatment, up to 3 months post-baselinePopulation: 9 subjects did not complete the Blessed Orientation-Memory-Concentration Test and they were excluded from analysis for this measure.
The Blessed Orientation-Memory-Concentration Test is designed to evaluate older patients for early dementia. Possible total scores range from 0 (all items answered correctly) to 28 (all items answered incorrectly).
Outcome measures
| Measure |
CPX-351 (Cytarabine:Daunorubicin) Injection
n=21 Participants
Dosing for first induction: CPX-351
• CPX-351 at 100u/m2 will be administered on study days 1, 3 and 5
Dosing for second induction:
• CPX-351 at 100 u/m2 will be administered on days 1 and 3
Dosing for consolidation:
• CPX-351 at 65 u/m2 will be administered on days 1 and 3
CPX-351: Cytarabine:Daunorubicin Liposome Injection
|
|---|---|
|
Change in Relationship of Cognitive Function to Treatment Response and OS, Event-free Survival and Morphologic Leukemia Free State as Measured by the Blessed Orientation-Memory-Concentration Test
Blessed Orientation-Memory-Concentration Test (baseline)
|
3.45 score on a scale
Standard Deviation 2.87
|
|
Change in Relationship of Cognitive Function to Treatment Response and OS, Event-free Survival and Morphologic Leukemia Free State as Measured by the Blessed Orientation-Memory-Concentration Test
Blessed Orientation-Memory-Concentration Test (30 days post-treatment)
|
2 score on a scale
Standard Deviation 2.27
|
SECONDARY outcome
Timeframe: Through treatment completion, an average of 1 yearAdverse events included neutropenic fever, transient episodes of pericarditis, febrile neutropenia, streptococcus bacteremia, hypotensive episode, severe diffuse cerebral dysfunction, UTI (klebsiella pneumonia), anorexia with malnutrition, hypophosphatemia, hypokalemia, purple macules, elevated ALT, hypoalbuminemia, hyperbilirubinemia, syncope, joint pain, transaminitis, bacteremia, typhlitis, VRE bacteremia, Osteomyelitis, Decreased LVEF, GI adenovirus, Acute kidney injury, pulmonary edema, neutropenia, bronchospasm, bone pain, urinary incontinence, c. difficile infection, mucositis, and vaginal pain.
Outcome measures
| Measure |
CPX-351 (Cytarabine:Daunorubicin) Injection
n=30 Participants
Dosing for first induction: CPX-351
• CPX-351 at 100u/m2 will be administered on study days 1, 3 and 5
Dosing for second induction:
• CPX-351 at 100 u/m2 will be administered on days 1 and 3
Dosing for consolidation:
• CPX-351 at 65 u/m2 will be administered on days 1 and 3
CPX-351: Cytarabine:Daunorubicin Liposome Injection
|
|---|---|
|
Incidence of Adverse Events
|
56 events
|
Adverse Events
CPX-351 (Cytarabine:Daunorubicin) Injection
Serious events: 14 serious events
Other events: 30 other events
Deaths: 23 deaths
Serious adverse events
| Measure |
CPX-351 (Cytarabine:Daunorubicin) Injection
n=30 participants at risk
Dosing for first induction: CPX-351
• CPX-351 at 100u/m2 will be administered on study days 1, 3 and 5
Dosing for second induction:
• CPX-351 at 100 u/m2 will be administered on days 1 and 3
Dosing for consolidation:
• CPX-351 at 65 u/m2 will be administered on days 1 and 3
CPX-351: Cytarabine:Daunorubicin Liposome Injection
|
|---|---|
|
General disorders
Neutropenic fever
|
6.7%
2/30 • 2.5 Years
|
|
Vascular disorders
Hemorrhagic cerebrovascular accident
|
3.3%
1/30 • 2.5 Years
|
|
Infections and infestations
Septic shock
|
3.3%
1/30 • 2.5 Years
|
|
Cardiac disorders
Volume overload
|
3.3%
1/30 • 2.5 Years
|
|
Nervous system disorders
Intracranial hemorrhage
|
6.7%
2/30 • 2.5 Years
|
|
Respiratory, thoracic and mediastinal disorders
Massive hemoptysis
|
3.3%
1/30 • 2.5 Years
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
3.3%
1/30 • 2.5 Years
|
|
Cardiac disorders
Ejection fraction decreased
|
20.0%
6/30 • 2.5 Years
|
|
General disorders
Failure to thrive
|
3.3%
1/30 • 2.5 Years
|
|
Cardiac disorders
Cardiopulmonary arrest
|
3.3%
1/30 • 2.5 Years
|
Other adverse events
| Measure |
CPX-351 (Cytarabine:Daunorubicin) Injection
n=30 participants at risk
Dosing for first induction: CPX-351
• CPX-351 at 100u/m2 will be administered on study days 1, 3 and 5
Dosing for second induction:
• CPX-351 at 100 u/m2 will be administered on days 1 and 3
Dosing for consolidation:
• CPX-351 at 65 u/m2 will be administered on days 1 and 3
CPX-351: Cytarabine:Daunorubicin Liposome Injection
|
|---|---|
|
General disorders
Neutropenic fever
|
23.3%
7/30 • 2.5 Years
|
|
Cardiac disorders
Transient episodes of pericarditis
|
3.3%
1/30 • 2.5 Years
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
40.0%
12/30 • 2.5 Years
|
|
Infections and infestations
Streptococcus bacteremia
|
3.3%
1/30 • 2.5 Years
|
|
Vascular disorders
Hypotensive episode
|
3.3%
1/30 • 2.5 Years
|
|
Nervous system disorders
Severe diffuse cerebral dysfunction
|
3.3%
1/30 • 2.5 Years
|
|
Infections and infestations
UTI (klebsiella pneumonia)
|
3.3%
1/30 • 2.5 Years
|
|
Metabolism and nutrition disorders
Anorexia with malnutrition
|
3.3%
1/30 • 2.5 Years
|
|
Investigations
Hypophosphatemia
|
6.7%
2/30 • 2.5 Years
|
|
Investigations
Hypokalemia
|
10.0%
3/30 • 2.5 Years
|
|
Skin and subcutaneous tissue disorders
Purple macules
|
3.3%
1/30 • 2.5 Years
|
|
Investigations
Elevated ALT
|
3.3%
1/30 • 2.5 Years
|
|
Investigations
Hypoalbuminemia
|
3.3%
1/30 • 2.5 Years
|
|
Investigations
Hyperbilirubinemia
|
10.0%
3/30 • 2.5 Years
|
|
Nervous system disorders
Syncope
|
3.3%
1/30 • 2.5 Years
|
|
Musculoskeletal and connective tissue disorders
Joint pain
|
3.3%
1/30 • 2.5 Years
|
|
Investigations
Transaminitis
|
3.3%
1/30 • 2.5 Years
|
|
Infections and infestations
Bacteremia
|
3.3%
1/30 • 2.5 Years
|
|
Infections and infestations
Typhlitis
|
3.3%
1/30 • 2.5 Years
|
|
Infections and infestations
VRE bacteremia
|
3.3%
1/30 • 2.5 Years
|
|
Infections and infestations
Osteomyelitis
|
3.3%
1/30 • 2.5 Years
|
|
Cardiac disorders
Decreased LVEF
|
6.7%
2/30 • 2.5 Years
|
|
Infections and infestations
GI adenovirus
|
3.3%
1/30 • 2.5 Years
|
|
Renal and urinary disorders
Acute kidney injury
|
6.7%
2/30 • 2.5 Years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
3.3%
1/30 • 2.5 Years
|
|
Blood and lymphatic system disorders
Neutropenia
|
3.3%
1/30 • 2.5 Years
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
3.3%
1/30 • 2.5 Years
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
3.3%
1/30 • 2.5 Years
|
|
Renal and urinary disorders
Urinary incontinence
|
3.3%
1/30 • 2.5 Years
|
|
Infections and infestations
C. difficile infection
|
3.3%
1/30 • 2.5 Years
|
|
Gastrointestinal disorders
Mucositis
|
3.3%
1/30 • 2.5 Years
|
|
Reproductive system and breast disorders
Vaginal pain
|
3.3%
1/30 • 2.5 Years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place