Trial Outcomes & Findings for CPX-351 Versus Immediate Stem Cell Transplantation for the Treatment of High-Grade Myeloid Cancers With Measurable Residual Disease (NCT NCT04526288)

NCT ID: NCT04526288

Last Updated: 2024-05-23

Results Overview

The survival (in days) of subjects in the two arms will be compared using the log-rank test.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

1 participants

Primary outcome timeframe

Up to 730 days post-randomization.

Results posted on

2024-05-23

Participant Flow

Patients arrived to Fred Hutchinson Cancer Center, Transplant Clinic, for transplant screening and disease staging. Upon confirmation of ability to proceed to transplant and concurrent enrollment in transplant protocol, recruitment to this study was offered. The dates of the study's recruitment period were 3/16/2021-12/07/2022.

Participant milestones

Participant milestones
Measure
Arm A (alloHCT)
Patients undergo allogeneic transplant (alloHCT) and do not receive investigational product. Allogeneic Hematopoietic Stem Cell Transplantation: Undergo alloHCT
Arm B (CPX-351, alloHCT)
Patients receive CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment may repeat for an additional cycle for a total of 2 cycles (on days 1 and 3 only of cycle 2) in the absence of disease progression or unacceptable toxicity. Within 60 days after completion of CPX-351, patients undergo alloHCT. Allogeneic Hematopoietic Stem Cell Transplantation: Undergo alloHCT Liposome-encapsulated Daunorubicin-Cytarabine: Given IV
Overall Study
STARTED
1
0
Overall Study
COMPLETED
0
0
Overall Study
NOT COMPLETED
1
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Arm A (alloHCT)
Patients undergo allogeneic transplant (alloHCT) and do not receive investigational product. Allogeneic Hematopoietic Stem Cell Transplantation: Undergo alloHCT
Arm B (CPX-351, alloHCT)
Patients receive CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment may repeat for an additional cycle for a total of 2 cycles (on days 1 and 3 only of cycle 2) in the absence of disease progression or unacceptable toxicity. Within 60 days after completion of CPX-351, patients undergo alloHCT. Allogeneic Hematopoietic Stem Cell Transplantation: Undergo alloHCT Liposome-encapsulated Daunorubicin-Cytarabine: Given IV
Overall Study
Disease Progression
1
0

Baseline Characteristics

CPX-351 Versus Immediate Stem Cell Transplantation for the Treatment of High-Grade Myeloid Cancers With Measurable Residual Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Arm A (alloHCT)
n=1 Participants
Patients undergo alloHCT. Allogeneic Hematopoietic Stem Cell Transplantation: Undergo alloHCT
Arm B (CPX-351, alloHCT)
Patients receive CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment may repeat for an additional cycle for a total of 2 cycles (on days 1 and 3 only of cycle 2) in the absence of disease progression or unacceptable toxicity. Within 60 days after completion of CPX-351, patients undergo alloHCT. Allogeneic Hematopoietic Stem Cell Transplantation: Undergo alloHCT Liposome-encapsulated Daunorubicin-Cytarabine: Given IV
Total
n=1 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
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Age, Categorical
>=65 years
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Sex: Female, Male
Female
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Sex: Female, Male
Male
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 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
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 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
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 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
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 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
1 participants
n=5 Participants
1 participants
n=5 Participants

PRIMARY outcome

Timeframe: Up to 730 days post-randomization.

Population: We did not enroll sufficiently to allow for analysis.

The survival (in days) of subjects in the two arms will be compared using the log-rank test.

Outcome measures

Outcome measures
Measure
Arm A (alloHCT)
n=1 Participants
Patients undergo alloHCT. Allogeneic Hematopoietic Stem Cell Transplantation: Undergo alloHCT
Arm B (CPX-351, alloHCT)
Patients receive CPX-351 IV over 90 minutes on days 1, 3, and 5. Treatment may repeat for an additional cycle for a total of 2 cycles (on days 1 and 3 only of cycle 2) in the absence of disease progression or unacceptable toxicity. Within 60 days after completion of CPX-351, patients undergo alloHCT. Allogeneic Hematopoietic Stem Cell Transplantation: Undergo alloHCT Liposome-encapsulated Daunorubicin-Cytarabine: Given IV
Overall Survival
730 Days
Standard Deviation 0

SECONDARY outcome

Timeframe: From the time of randomization up to 2 years post-randomization. For descriptive purposes, we shall also report survival and relapse-free survival from time of transplant up to 2 years post-transplant among patients who receive a transplant.

Population: Data was not collected for this outcome measure.

The following comparisons will be made: (1) number of days elapsing between the date of minimal residual disease (MRD) identification and the date of death or relapse, (2) among transplanted patients, the number of days elapsing between the date of transplantation and the date of death or relapse.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 2 years post-randomization

Population: Data was not collected for this outcome measure because the one participant did not start transplantation conditioning.

This outcome will be assessed among patients who have begun transplantation conditioning 60 days and 180 days following enrollment will be compared using the chi-squared test. The time to transplantation among the two arms will be compared by comparing the number of days elapsed between enrollment and the initiation of transplantation conditions by the log-rank test.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 2 years post-randomization

Population: The one participant enrolled did not proceed with transplant on the study.

Among the patients receiving transplantation, the proportion of patients in each arm receiving myeloablative transplantation conditioning will be compared using the chi-squared or Fisher's exact test. Among patients receiving transplantation, the proportion of patients in each arm receiving donor stem cells from a haploidentical donor, an unrelated donor, or from cord blood unit will each be compared using the chi-squared or Fisher's exact test.

Outcome measures

Outcome data not reported

Adverse Events

Arm A (alloHCT)

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Arm B (CPX-351, alloHCT)

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Filippo Milano, Director of FHCC Cord Blood Transplant Program

Fred Hutchinson Cancer Center

Phone: 206.667.5925

Results disclosure agreements

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