Trial Outcomes & Findings for CPX-351 Therapy for MDS After Hypomethylating Agent Failure (NCT NCT03957876)
NCT ID: NCT03957876
Last Updated: 2024-08-15
Results Overview
Efficacy of CPX as measured by ORR as defined by IWG 2006 criteria for MDS participants at end of induction. IWG 2006 responses that must be for at least 4 weeks include Complete Remission (CR), Partial Remission (PR), Marrow CR, Stable Disease (SD), Failure, Relapse after CR or PR (PD), or cytogenetic response. Hematologic Improvement (HI), which must be for at least 8 weeks, includes erythroid response (pretreatment, \< 11 g/dL), Platelet response (pretreatment, \< 100x109/L), Neutrophil response (pretreatment, \< 1 x109/L), or Progression or relapse after HI.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
4 participants
Primary outcome timeframe
day 28 +/- 7 days of induction
Results posted on
2024-08-15
Participant Flow
Participant milestones
| Measure |
Intravenous CPX-351 With Potential Maintenance Therapy
Single agent CPX-351 administered at the standard FDA approved dose of 44 mg/m2 intravenously on days 1, 3, 5 of the induction cycle. If participants achieve complete remission (CR), complete remission with incomplete count recovery (CRi) or partial remission (PR), they will be eligible to continue on to maintenance therapy, which will consist of CPX351 at a dose of 15.4 mg/m2 every 28 days. Participants can receive up to 4 cycles of maintenance therapy.
CPX-351: CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin.
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
CPX-351 Therapy for MDS After Hypomethylating Agent Failure
Baseline characteristics by cohort
| Measure |
Intravenous CPX-351 With Potential Maintenance Therapy
n=4 Participants
Single agent CPX-351 administered at the standard FDA approved dose of 44 mg/m2 intravenously on days 1, 3, 5 of the induction cycle. If participants achieve complete remission (CR), complete remission with incomplete count recovery (CRi) or partial remission (PR), they will be eligible to continue on to maintenance therapy, which will consist of CPX351 at a dose of 15.4 mg/m2 every 28 days. Participants can receive up to 4 cycles of maintenance therapy.
CPX-351: CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: day 28 +/- 7 days of inductionPopulation: 3 of the 4 participants did not meet evaluable criteria as they did not survive to C1D1 of study.
Efficacy of CPX as measured by ORR as defined by IWG 2006 criteria for MDS participants at end of induction. IWG 2006 responses that must be for at least 4 weeks include Complete Remission (CR), Partial Remission (PR), Marrow CR, Stable Disease (SD), Failure, Relapse after CR or PR (PD), or cytogenetic response. Hematologic Improvement (HI), which must be for at least 8 weeks, includes erythroid response (pretreatment, \< 11 g/dL), Platelet response (pretreatment, \< 100x109/L), Neutrophil response (pretreatment, \< 1 x109/L), or Progression or relapse after HI.
Outcome measures
| Measure |
Intravenous CPX-351 With Potential Maintenance Therapy
n=1 Participants
Single agent CPX-351 administered at the standard FDA approved dose of 44 mg/m2 intravenously on days 1, 3, 5 of the induction cycle. If participants achieve complete remission (CR), complete remission with incomplete count recovery (CRi) or partial remission (PR), they will be eligible to continue on to maintenance therapy, which will consist of CPX351 at a dose of 15.4 mg/m2 every 28 days. Participants can receive up to 4 cycles of maintenance therapy.
CPX-351: CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin.
|
|---|---|
|
Efficacy of CPX-351 as Measured by Overall Response Rate (ORR)
Complete remission (CR)
|
1 Participants
|
|
Efficacy of CPX-351 as Measured by Overall Response Rate (ORR)
Partial remission (PR)
|
0 Participants
|
|
Efficacy of CPX-351 as Measured by Overall Response Rate (ORR)
Marrow CR
|
0 Participants
|
|
Efficacy of CPX-351 as Measured by Overall Response Rate (ORR)
Stable disease (SD)
|
0 Participants
|
|
Efficacy of CPX-351 as Measured by Overall Response Rate (ORR)
Failure
|
0 Participants
|
|
Efficacy of CPX-351 as Measured by Overall Response Rate (ORR)
Relapse after CR or PR (PD)
|
0 Participants
|
|
Efficacy of CPX-351 as Measured by Overall Response Rate (ORR)
Cytogenetic response
|
0 Participants
|
SECONDARY outcome
Timeframe: day 28 +/- 7 days of inductionPopulation: 3 additional subjects did not receive study drug (only completed induction phase).
TTR associated with CPX-351 in participant with MDS at the end of induction. TTR defined by the time between starting the treatment and the time of achieving best response.
Outcome measures
| Measure |
Intravenous CPX-351 With Potential Maintenance Therapy
n=1 Participants
Single agent CPX-351 administered at the standard FDA approved dose of 44 mg/m2 intravenously on days 1, 3, 5 of the induction cycle. If participants achieve complete remission (CR), complete remission with incomplete count recovery (CRi) or partial remission (PR), they will be eligible to continue on to maintenance therapy, which will consist of CPX351 at a dose of 15.4 mg/m2 every 28 days. Participants can receive up to 4 cycles of maintenance therapy.
CPX-351: CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin.
|
|---|---|
|
Time to Response (TTR) Associated With CPX-351
|
28 Days
|
SECONDARY outcome
Timeframe: up to 1 year after end of treatmentPopulation: 3 of the 4 participants did not meet evaluable criteria as they did not survive to C1D1 of study.
DOR in participants achieving a response defined by the time between first response (day C1 D28 +/-7 days from induction) and the day of loss of response
Outcome measures
| Measure |
Intravenous CPX-351 With Potential Maintenance Therapy
n=1 Participants
Single agent CPX-351 administered at the standard FDA approved dose of 44 mg/m2 intravenously on days 1, 3, 5 of the induction cycle. If participants achieve complete remission (CR), complete remission with incomplete count recovery (CRi) or partial remission (PR), they will be eligible to continue on to maintenance therapy, which will consist of CPX351 at a dose of 15.4 mg/m2 every 28 days. Participants can receive up to 4 cycles of maintenance therapy.
CPX-351: CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin.
|
|---|---|
|
Duration of Response (DOR) in Participants Achieving a Response
|
458 days
|
SECONDARY outcome
Timeframe: up to 1 year after end of treatmentEFS probability of all participants enrolled in this trial from start of treatment and up to 1 year after the end of treatment.
Outcome measures
| Measure |
Intravenous CPX-351 With Potential Maintenance Therapy
n=4 Participants
Single agent CPX-351 administered at the standard FDA approved dose of 44 mg/m2 intravenously on days 1, 3, 5 of the induction cycle. If participants achieve complete remission (CR), complete remission with incomplete count recovery (CRi) or partial remission (PR), they will be eligible to continue on to maintenance therapy, which will consist of CPX351 at a dose of 15.4 mg/m2 every 28 days. Participants can receive up to 4 cycles of maintenance therapy.
CPX-351: CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin.
|
|---|---|
|
Event-free Survival (EFS)
|
1 Participants
|
SECONDARY outcome
Timeframe: up to 1 year after end of treatmentOS probability of all participants enrolled in this trial from start of treatment and up to 1 year after the end of treatment.
Outcome measures
| Measure |
Intravenous CPX-351 With Potential Maintenance Therapy
n=4 Participants
Single agent CPX-351 administered at the standard FDA approved dose of 44 mg/m2 intravenously on days 1, 3, 5 of the induction cycle. If participants achieve complete remission (CR), complete remission with incomplete count recovery (CRi) or partial remission (PR), they will be eligible to continue on to maintenance therapy, which will consist of CPX351 at a dose of 15.4 mg/m2 every 28 days. Participants can receive up to 4 cycles of maintenance therapy.
CPX-351: CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin.
|
|---|---|
|
Overall Survival (OS)
|
1 Participants
|
Adverse Events
Intravenous CPX-351 With Potential Maintenance Therapy
Serious events: 2 serious events
Other events: 4 other events
Deaths: 4 deaths
Serious adverse events
| Measure |
Intravenous CPX-351 With Potential Maintenance Therapy
n=4 participants at risk
Single agent CPX-351 administered at the standard FDA approved dose of 44 mg/m2 intravenously on days 1, 3, 5 of the induction cycle. If participants achieve complete remission (CR), complete remission with incomplete count recovery (CRi) or partial remission (PR), they will be eligible to continue on to maintenance therapy, which will consist of CPX351 at a dose of 15.4 mg/m2 every 28 days. Participants can receive up to 4 cycles of maintenance therapy.
CPX-351: CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Anemia
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
Other adverse events
| Measure |
Intravenous CPX-351 With Potential Maintenance Therapy
n=4 participants at risk
Single agent CPX-351 administered at the standard FDA approved dose of 44 mg/m2 intravenously on days 1, 3, 5 of the induction cycle. If participants achieve complete remission (CR), complete remission with incomplete count recovery (CRi) or partial remission (PR), they will be eligible to continue on to maintenance therapy, which will consist of CPX351 at a dose of 15.4 mg/m2 every 28 days. Participants can receive up to 4 cycles of maintenance therapy.
CPX-351: CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin.
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
100.0%
4/4 • 2.5 years or until death, whichever occurs first.
|
|
Blood and lymphatic system disorders
Cardiac disorders
|
50.0%
2/4 • 2.5 years or until death, whichever occurs first.
|
|
Blood and lymphatic system disorders
Chest pain - cardiac
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Blood and lymphatic system disorders
Febrile neutropenoa
|
75.0%
3/4 • 2.5 years or until death, whichever occurs first.
|
|
Blood and lymphatic system disorders
Sinus Tachycardia
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Gastrointestinal disorders
Abdominal pain
|
50.0%
2/4 • 2.5 years or until death, whichever occurs first.
|
|
Gastrointestinal disorders
Constipation
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Gastrointestinal disorders
Diarrhea
|
75.0%
3/4 • 2.5 years or until death, whichever occurs first.
|
|
Gastrointestinal disorders
Fecal incontinence
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Gastrointestinal disorders
Flatulence
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Gastrointestinal disorders
Gum bleeding
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Gastrointestinal disorders
Hemorrhoids
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Gastrointestinal disorders
Oral hemorrhage
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Gastrointestinal disorders
Lip pain
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Gastrointestinal disorders
Nausea
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
General disorders
Chills
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
General disorders
Edema face
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
General disorders
Edema limbs
|
50.0%
2/4 • 2.5 years or until death, whichever occurs first.
|
|
General disorders
Fatigue
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Infections and infestations
Lung infection
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Infections and infestations
Other
|
100.0%
4/4 • 2.5 years or until death, whichever occurs first.
|
|
Infections and infestations
Sepsis
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Infections and infestations
Skin infection
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Infections and infestations
Sputum pseudomonas
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Injury, poisoning and procedural complications
Fall
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Investigations
Alanine aminotransferase increased
|
50.0%
2/4 • 2.5 years or until death, whichever occurs first.
|
|
Investigations
Alkaline phosphatase increased
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Investigations
Aspartate aminotransferase increased
|
50.0%
2/4 • 2.5 years or until death, whichever occurs first.
|
|
Investigations
Blood biliruib increased
|
50.0%
2/4 • 2.5 years or until death, whichever occurs first.
|
|
Investigations
Blood lactate dehydrogenase increased
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Investigations
Creatinine increased
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Investigations
Electrocardiogram QT corrected interval prolonged
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Investigations
Lymphocyte count decreased
|
50.0%
2/4 • 2.5 years or until death, whichever occurs first.
|
|
Investigations
Neutrophil count decrease
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Investigations
Platelet count decreased
|
75.0%
3/4 • 2.5 years or until death, whichever occurs first.
|
|
Investigations
Weight gain
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Investigations
White blood cell descreased
|
75.0%
3/4 • 2.5 years or until death, whichever occurs first.
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
50.0%
2/4 • 2.5 years or until death, whichever occurs first.
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
75.0%
3/4 • 2.5 years or until death, whichever occurs first.
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Metabolism and nutrition disorders
Hypomagnesmia
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Metabolism and nutrition disorders
Hyponatremia
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
50.0%
2/4 • 2.5 years or until death, whichever occurs first.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Nervous system disorders
Dizziness
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Nervous system disorders
Headache
|
75.0%
3/4 • 2.5 years or until death, whichever occurs first.
|
|
Nervous system disorders
Memory Impairment
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Psychiatric disorders
Confusion
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Psychiatric disorders
Delirium
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Psychiatric disorders
Insomnia
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Renal and urinary disorders
Dysuria
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Renal and urinary disorders
Urinary incontinence
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
50.0%
2/4 • 2.5 years or until death, whichever occurs first.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Respiratory, thoracic and mediastinal disorders
Hemoptysis
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
50.0%
2/4 • 2.5 years or until death, whichever occurs first.
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Skin and subcutaneous tissue disorders
Erythema multiforme
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Skin and subcutaneous tissue disorders
Night sweats
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
50.0%
2/4 • 2.5 years or until death, whichever occurs first.
|
|
Vascular disorders
Hypertension
|
50.0%
2/4 • 2.5 years or until death, whichever occurs first.
|
|
Vascular disorders
Thromboembolic event
|
25.0%
1/4 • 2.5 years or until death, whichever occurs first.
|
Additional Information
Sudipto Mukherjee, MD, PhD, MPH
Cleveland Clinic Foundation, Case Comprehensive Cancer Center
Phone: 216-444-0506
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place