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Trial Outcomes & Findings for Ixazomib (MLN9708) in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia (NCT NCT02030405)

NCT ID: NCT02030405

Last Updated: 2019-04-05

Results Overview

Overall response rate after 3 cycles of treatment (9 weeks) was assessed as complete remission (CR); CR with incomplete recovery (CRi); and partial remission (PR) with MLN9708, in participants with NPM1-mutated AML by LeukemiaNet1 guidelines: Although achievement of complete remission (CR) has unique clinical significance for improved overall survival (OS) and relapse-free survival (RFS) compared to achievement of CRi with incomplete platelet recovery, the latter is still a clinically meaningful response, as it is independently-superior to resistant disease. Partial remission (PR) is defined as meeting all hematologic criteria for CR with an allowance for 5% to 25% bone marrow blasts or decrease of pre-treatment bone marrow blast percentage by at least 50%. Stable disease is defined as a change in bone marrow aspirate blast count within 10% of baseline. Relapsed disease is defined as reappearance of blasts in the blood or bone marrow blasts

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

9 weeks

Results posted on

2019-04-05

Participant Flow

Participant milestones

Participant milestones
Measure
Ixazomib (MLN9708)
Participants receive ixazomib (orally) PO on days 1, 4, 8, and 11. Treatment repeats every 21 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.
Overall Study
STARTED
4
Overall Study
COMPLETED
2
Overall Study
NOT COMPLETED
2

Reasons for withdrawal

Reasons for withdrawal
Measure
Ixazomib (MLN9708)
Participants receive ixazomib (orally) PO on days 1, 4, 8, and 11. Treatment repeats every 21 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.
Overall Study
Withdrawal by Subject
1
Overall Study
Lost to Follow-up
1

Baseline Characteristics

Ixazomib (MLN9708) in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Ixazomib (MLN9708)
n=4 Participants
Participants receive ixazomib (orally) PO on days 1, 4, 8, and 11. Treatment repeats every 21 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.
Age, Categorical
<=18 years
0 Participants
n=93 Participants
Age, Categorical
Between 18 and 65 years
1 Participants
n=93 Participants
Age, Categorical
>=65 years
3 Participants
n=93 Participants
Sex: Female, Male
Female
4 Participants
n=93 Participants
Sex: Female, Male
Male
0 Participants
n=93 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=93 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
4 Participants
n=93 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=93 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=93 Participants
Race (NIH/OMB)
Asian
0 Participants
n=93 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=93 Participants
Race (NIH/OMB)
Black or African American
1 Participants
n=93 Participants
Race (NIH/OMB)
White
2 Participants
n=93 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=93 Participants
Race (NIH/OMB)
Unknown or Not Reported
1 Participants
n=93 Participants

PRIMARY outcome

Timeframe: 9 weeks

Overall response rate after 3 cycles of treatment (9 weeks) was assessed as complete remission (CR); CR with incomplete recovery (CRi); and partial remission (PR) with MLN9708, in participants with NPM1-mutated AML by LeukemiaNet1 guidelines: Although achievement of complete remission (CR) has unique clinical significance for improved overall survival (OS) and relapse-free survival (RFS) compared to achievement of CRi with incomplete platelet recovery, the latter is still a clinically meaningful response, as it is independently-superior to resistant disease. Partial remission (PR) is defined as meeting all hematologic criteria for CR with an allowance for 5% to 25% bone marrow blasts or decrease of pre-treatment bone marrow blast percentage by at least 50%. Stable disease is defined as a change in bone marrow aspirate blast count within 10% of baseline. Relapsed disease is defined as reappearance of blasts in the blood or bone marrow blasts

Outcome measures

Outcome measures
Measure
Ixazomib (MLN9708)
n=2 Participants
Participants receive ixazomib (orally) PO on days 1, 4, 8, and 11. Treatment repeats every 21 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.
Overall Response Rate (ORR)
Complete Remission (CR)
0 Participants
Overall Response Rate (ORR)
Complete Remission with incomplete recovery (CRi)
0 Participants
Overall Response Rate (ORR)
Partial Remission (PR)
0 Participants
Overall Response Rate (ORR)
Stable Disease (SD)
0 Participants
Overall Response Rate (ORR)
Relapsed Disease (RD)
0 Participants
Overall Response Rate (ORR)
Progressive Disease (PD)
2 Participants

SECONDARY outcome

Timeframe: 1 year

Population: No participants met the criteria of complete remission (CR), and so duration of response (DOR) in those participants could not be assessed.

Duration of response (DOR) in participants with complete remission (CR) was defined as the period of time from documented complete remission through relapse or death, with relapse defined as reappearance of blasts in the blood or bone marrow blasts, after documented CR. DOR was to be assessed through at least 1 year follow-up.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Overall survival (OS) from time of study entry to the earlier of death from any cause or end of follow up at 1 year

Outcome measures

Outcome measures
Measure
Ixazomib (MLN9708)
n=4 Participants
Participants receive ixazomib (orally) PO on days 1, 4, 8, and 11. Treatment repeats every 21 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.
Overall Survival (OS)
0 Participants

SECONDARY outcome

Timeframe: 1 year

Population: All treated subjects are included.

Ixazomib toxicity and tolerability were assessed based on the non-hematologic toxicities ≥ Grade 3 determined to be possibly, probably, or definitely related to the study agent Ixazomib. Adverse events that are possibly, probably, or definitely related to the study agent are considered "toxicities." The outcome is reported as the overall number of non-hematologic toxicities ≥ Grade 3.

Outcome measures

Outcome measures
Measure
Ixazomib (MLN9708)
n=4 Participants
Participants receive ixazomib (orally) PO on days 1, 4, 8, and 11. Treatment repeats every 21 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.
Serious Adverse Events Related to Ixazomib
3 Related adverse events

Adverse Events

Ixazomib (MLN9708)

Serious events: 4 serious events
Other events: 4 other events
Deaths: 4 deaths

Serious adverse events

Serious adverse events
Measure
Ixazomib (MLN9708)
n=4 participants at risk
Participants receive ixazomib (orally) PO on days 1, 4, 8, and 11. Treatment repeats every 21 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.
Infections and infestations
Febrile Neutropenia
25.0%
1/4 • Number of events 1 • 1 year
Blood and lymphatic system disorders
AML Disease progression
50.0%
2/4 • Number of events 2 • 1 year
Blood and lymphatic system disorders
Pancytopenia
25.0%
1/4 • Number of events 1 • 1 year
Renal and urinary disorders
Acute kidney injury
25.0%
1/4 • Number of events 1 • 1 year
Gastrointestinal disorders
Diarrhea
50.0%
2/4 • Number of events 2 • 1 year
Gastrointestinal disorders
Nausea
25.0%
1/4 • Number of events 1 • 1 year
General disorders
Dehydration
25.0%
1/4 • Number of events 1 • 1 year
Respiratory, thoracic and mediastinal disorders
Acute Respiratory Distress Syndrome
25.0%
1/4 • Number of events 1 • 1 year
Cardiac disorders
Cardiopulmonary Arrest
25.0%
1/4 • Number of events 1 • 1 year

Other adverse events

Other adverse events
Measure
Ixazomib (MLN9708)
n=4 participants at risk
Participants receive ixazomib (orally) PO on days 1, 4, 8, and 11. Treatment repeats every 21 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.
Respiratory, thoracic and mediastinal disorders
Acute Pulmonary Edema
25.0%
1/4 • Number of events 1 • 1 year
Skin and subcutaneous tissue disorders
Skin Rash
50.0%
2/4 • Number of events 2 • 1 year
Gastrointestinal disorders
Vomiting
25.0%
1/4 • Number of events 1 • 1 year
Gastrointestinal disorders
Diarrhea
50.0%
2/4 • Number of events 2 • 1 year
Respiratory, thoracic and mediastinal disorders
Dysphagia
25.0%
1/4 • Number of events 1 • 1 year
Gastrointestinal disorders
Nausea
50.0%
2/4 • Number of events 2 • 1 year
Renal and urinary disorders
Rectal Pain
25.0%
1/4 • Number of events 1 • 1 year

Additional Information

Bruno Carneiro de Medeiros, MD

Stanford University Medical Center

Phone: 650-498-6000

Results disclosure agreements

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