Trial Outcomes & Findings for CPX-351 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome (NCT NCT02019069)

NCT ID: NCT02019069

Last Updated: 2019-01-22

Results Overview

The response rate was determined as the sum of complete response calculated by adding the total complete response (CR) and complete response with incomplete count recovery (CRi). The outcome is reported as the total number without dispersion. * CR = less than 5% blasts; no blasts with auer rods; and no persistence of extramedullary disease, with blood count recovery to platelets ≥ 100,000/uL and ANC \> 1000/uL, with transfusion independence. * CRi = all the parameters for CR, but platelets \< 100,000/uL and/or ANC ≤ 1000/uL.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

11 participants

Primary outcome timeframe

Day 42

Results posted on

2019-01-22

Participant Flow

Participant milestones

Participant milestones
Measure
Liposomal Cytarabine-daunorubicin CPX-351
* 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5. * 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. * CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. liposomal cytarabine-daunorubicin CPX-351: Given IV
Overall Study
STARTED
11
Overall Study
COMPLETED
11
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

CPX-351 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Liposomal Cytarabine-daunorubicin CPX-351
n=11 Participants
* 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5. * 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. * CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. liposomal cytarabine-daunorubicin CPX-351: Given IV
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
0 Participants
n=5 Participants
Age, Categorical
>=65 years
11 Participants
n=5 Participants
Age, Continuous
74 years
STANDARD_DEVIATION 4.9 • 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
2 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
9 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
1 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
0 Participants
n=5 Participants
Race (NIH/OMB)
White
8 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: Day 42

The response rate was determined as the sum of complete response calculated by adding the total complete response (CR) and complete response with incomplete count recovery (CRi). The outcome is reported as the total number without dispersion. * CR = less than 5% blasts; no blasts with auer rods; and no persistence of extramedullary disease, with blood count recovery to platelets ≥ 100,000/uL and ANC \> 1000/uL, with transfusion independence. * CRi = all the parameters for CR, but platelets \< 100,000/uL and/or ANC ≤ 1000/uL.

Outcome measures

Outcome measures
Measure
Liposomal Cytarabine-daunorubicin CPX-351
n=11 Participants
* 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5. * 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. * CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. liposomal cytarabine-daunorubicin CPX-351: Given IV
Response Rate (RR)
3 Participants

SECONDARY outcome

Timeframe: Day 42

Complete response (CR) with incomplete count recovery (CRi) was determined as the number of participants who achieved CRi after induction therapy. The outcome is reported as the total number or participants without dispersion. * CR = less than 5% blasts; no blasts with auer rods; and no persistence of extramedullary disease, with blood count recovery to platelets ≥ 100,000/uL and ANC \> 1000/uL, with transfusion independence. * CRi = all the parameters for CR, but platelets \< 100,000/uL and/or ANC ≤ 1000/uL.

Outcome measures

Outcome measures
Measure
Liposomal Cytarabine-daunorubicin CPX-351
n=11 Participants
* 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5. * 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. * CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. liposomal cytarabine-daunorubicin CPX-351: Given IV
Complete Response With Incomplete Count Recovery (CRi)
1 Participants

SECONDARY outcome

Timeframe: Day 42

Complete response (CR) was determined the number of participants who achieved CR by Day 42 after induction treatment. The outcome is reported as the total number of participants without dispersion. • CR = less than 5% blasts; no blasts with auer rods; and no persistence of extramedullary disease, with blood count recovery to platelets ≥ 100,000/uL and ANC \> 1000/uL, with transfusion independence.

Outcome measures

Outcome measures
Measure
Liposomal Cytarabine-daunorubicin CPX-351
n=11 Participants
* 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5. * 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. * CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. liposomal cytarabine-daunorubicin CPX-351: Given IV
Complete Response (CR)
2 Participants

SECONDARY outcome

Timeframe: Up to 1 year

Duration of remission (DOR) was assessed as the length of time from documented complete response (CR) or complete response with incomplete count recovery (CRi) until documented lost of response, relapse, or death. The outcome is reported as the median with full range. * CR = less than 5% blasts; no blasts with auer rods; and no persistence of extramedullary disease, with blood count recovery to platelets ≥ 100,000/uL and ANC \> 1000/uL, with transfusion independence. * CRi = all the parameters for CR, but platelets \< 100,000/uL and/or ANC ≤ 1000/uL. For patients remaining alive, duration of remission (DOR) is reported as the length of time from documented complete response (CR) or complete response with incomplete count recovery (CRi) until the most recent assessment.

Outcome measures

Outcome measures
Measure
Liposomal Cytarabine-daunorubicin CPX-351
n=11 Participants
* 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5. * 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. * CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. liposomal cytarabine-daunorubicin CPX-351: Given IV
Duration of Remission (DOR) Following Induction With CPX-351
185 days
Interval 25.0 to 329.0

SECONDARY outcome

Timeframe: At 12 months

Overall survival (OS) was assessed as the number of participants remaining alive 12 months, starting from date of entry into trial. The outcome is reported as the number of participants (without dispersion).

Outcome measures

Outcome measures
Measure
Liposomal Cytarabine-daunorubicin CPX-351
n=11 Participants
* 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5. * 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. * CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. liposomal cytarabine-daunorubicin CPX-351: Given IV
Overall Survival (OS)
1 Participants

SECONDARY outcome

Timeframe: 30 days

Early induction mortality was assessed as the number of participants who died within 30 days of completing the 1st cycle of CPX-351 (1st induction). The outcome is reported as the number of participants without dispersion.

Outcome measures

Outcome measures
Measure
Liposomal Cytarabine-daunorubicin CPX-351
n=11 Participants
* 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5. * 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. * CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. liposomal cytarabine-daunorubicin CPX-351: Given IV
Early Induction Mortality (Day 30 After 1st Induction)
2 Participants

SECONDARY outcome

Timeframe: 60 days

Mortality at Day 60 after 1st induction was assessed as the number of participants who died within 60 days of completing the 1st cycle of CPX-351 (1st induction). The outcome is reported as the number of participants without dispersion.

Outcome measures

Outcome measures
Measure
Liposomal Cytarabine-daunorubicin CPX-351
n=11 Participants
* 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5. * 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. * CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. liposomal cytarabine-daunorubicin CPX-351: Given IV
Mortality at Day 60 After 1st Induction
3 Participants

SECONDARY outcome

Timeframe: Up to 4 weeks after completion of treatment

Serious adverse events per participant were assessed as serious adverse events per 21CFR§312.32 that were Grade 3 or greater, and independent of relationship to CPX-351. The outcome is reported as the number of participants that experienced any defined SAE, a number without dispersion.

Outcome measures

Outcome measures
Measure
Liposomal Cytarabine-daunorubicin CPX-351
n=11 Participants
* 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5. * 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. * CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. liposomal cytarabine-daunorubicin CPX-351: Given IV
Participants Experiencing of Serious Adverse Events
5 Participants

SECONDARY outcome

Timeframe: Up to 4 weeks after completion of treatment

Population: Per protocol, the outcome only includes SAEs that are Grade 3 or greater. Adverse events that were Grade 2 or less, (eg, Grade 2 event with hospitalization) are not included.

Serious adverse events were assessed as serious adverse events per 21CFR§312.32 that were Grade 3 or greater, and independent of relationship to CPX-351. The outcome is reported as the total number of the defined SAEs, a number without dispersion.

Outcome measures

Outcome measures
Measure
Liposomal Cytarabine-daunorubicin CPX-351
n=11 Participants
* 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5. * 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. * CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. liposomal cytarabine-daunorubicin CPX-351: Given IV
Serious Adverse Events
8 Adverse events

Adverse Events

Liposomal Cytarabine-daunorubicin CPX-351

Serious events: 5 serious events
Other events: 11 other events
Deaths: 10 deaths

Serious adverse events

Serious adverse events
Measure
Liposomal Cytarabine-daunorubicin CPX-351
n=11 participants at risk
* 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5. * 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. * CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. liposomal cytarabine-daunorubicin CPX-351: Given IV
Respiratory, thoracic and mediastinal disorders
Pneumonia
18.2%
2/11 • Number of events 2 • Up to 4 weeks after completion of treatment]
Respiratory, thoracic and mediastinal disorders
Adult repiratory distress syndrome (ARDS)
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Cardiac disorders
Cardio-pulmonary arrest
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Blood and lymphatic system disorders
Febrile neutropenia
36.4%
4/11 • Number of events 5 • Up to 4 weeks after completion of treatment]
Respiratory, thoracic and mediastinal disorders
Respiratory failure
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
General disorders
Chills
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Blood and lymphatic system disorders
Anemia
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms - Other, Acute myelogenous leukemia
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]

Other adverse events

Other adverse events
Measure
Liposomal Cytarabine-daunorubicin CPX-351
n=11 participants at risk
* 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5. * 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. * CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3. liposomal cytarabine-daunorubicin CPX-351: Given IV
Blood and lymphatic system disorders
Blood and lymphatic system disorders, other - clot in cheek
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Blood and lymphatic system disorders
Febrile neutropenia
72.7%
8/11 • Number of events 8 • Up to 4 weeks after completion of treatment]
Blood and lymphatic system disorders
Thrombotic thrombocytopenic purpura
18.2%
2/11 • Number of events 2 • Up to 4 weeks after completion of treatment]
Cardiac disorders
Atrial fibrillation
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Cardiac disorders
Cardiac disorders, other - tachycardia
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Cardiac disorders
Sinus tachycardia
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Eye disorders
Eye disorders, other - eye itching
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Eye disorders
Papilledema
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Gastrointestinal disorders
Abdominal pain
27.3%
3/11 • Number of events 3 • Up to 4 weeks after completion of treatment]
Gastrointestinal disorders
Colitis
27.3%
3/11 • Number of events 3 • Up to 4 weeks after completion of treatment]
Gastrointestinal disorders
Constipation
36.4%
4/11 • Number of events 4 • Up to 4 weeks after completion of treatment]
Gastrointestinal disorders
Diarrhea
63.6%
7/11 • Number of events 7 • Up to 4 weeks after completion of treatment]
Gastrointestinal disorders
Mucositis oral
54.5%
6/11 • Number of events 6 • Up to 4 weeks after completion of treatment]
Gastrointestinal disorders
Nausea
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Gastrointestinal disorders
Rectal hemorrhage
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
General disorders
Pain, hip
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
General disorders
Pain, leg
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
General disorders
Fatigue
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
General disorders
Fever
36.4%
4/11 • Number of events 4 • Up to 4 weeks after completion of treatment]
Infections and infestations
Lung infection
18.2%
2/11 • Number of events 2 • Up to 4 weeks after completion of treatment]
Investigations
Alanine aminotransferase increased
18.2%
2/11 • Number of events 2 • Up to 4 weeks after completion of treatment]
Investigations
Investigations, other - fluid overload
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Metabolism and nutrition disorders
Hepatobiliary disorders, other - hyperbilirubinemia
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Metabolism and nutrition disorders
Anorexia (decreased food consumption)
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Metabolism and nutrition disorders
Hypertriglyceridemia
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Musculoskeletal and connective tissue disorders
Bone pain
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder, other - broken leg
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Nervous system disorders
Brachial plexopathy
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Nervous system disorders
Dizziness
18.2%
2/11 • Number of events 2 • Up to 4 weeks after completion of treatment]
Nervous system disorders
Headache
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Nervous system disorders
Peripheral motor neuropathy
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Psychiatric disorders
Hallucinations
36.4%
4/11 • Number of events 4 • Up to 4 weeks after completion of treatment]
Renal and urinary disorders
Hematuria
27.3%
3/11 • Number of events 3 • Up to 4 weeks after completion of treatment]
Renal and urinary disorders
Renal and urinary disorders, other - urinary discomfort
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Respiratory, thoracic and mediastinal disorders
Aspiration
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Respiratory, thoracic and mediastinal disorders
Cough
27.3%
3/11 • Number of events 3 • Up to 4 weeks after completion of treatment]
Respiratory, thoracic and mediastinal disorders
Epistaxis (nose bleed)
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Respiratory, thoracic and mediastinal disorders
Hypoxia
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders, other - chest tightness
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Respiratory, thoracic and mediastinal disorders
Pneumothorax
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Skin and subcutaneous tissue disorders
Rash
27.3%
3/11 • Number of events 3 • Up to 4 weeks after completion of treatment]
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders, other - multiple subcutaneous nodules
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]
Vascular disorders
Thromboembolic event
9.1%
1/11 • Number of events 1 • Up to 4 weeks after completion of treatment]

Additional Information

Rondeep Singh Brar

Stanford University

Phone: (650) 498-6000

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place