Trial Outcomes & Findings for Midostaurin and Decitabine in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia and FLT3 Mutation (NCT NCT02634827)

NCT ID: NCT02634827

Last Updated: 2019-08-07

Results Overview

The proportion of successes will be estimated by the number of successes divided by the total number of evaluable patients. Exact binomial confidence intervals for the true success proportion will be calculated.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

1 participants

Primary outcome timeframe

Up to 2 years

Results posted on

2019-08-07

Participant Flow

One (1) patient was accrued from December 2015 to June 2018 at Mayo Clinic. Since only one patient was accrued, patient confidentiality prevents the reporting of this patient.

Participant milestones

Participant milestones
Measure
Treatment (Decitabine, Midostaurin)
Patients receive decitabine intravenously (IV) over 1 hour on days 1-5 and midostaurin orally (PO) twice daily (BID) on days 8-21 of courses 1 and 2, and on days 1-28 of each subsequent course. Patients failing to achieve complete response (CR)/complete response with incomplete recovery (CRi)/partial response (PR)/morphologic leukemia-free state by end of course 2 receive midostaurin PO BID on days 1-28. Patients achieving CR/CRi/PR/morphologic leukemia-free state by end of course 8 may continue on current regimen. Patients failing to achieve a CR/CRi/PR/ morphologic leukemia-free state in bone marrow blasts by end of course 8 go to event monitoring. Treatment repeats every 28 days for up to 18 courses in the absence of disease progression or unacceptable toxicity.
Overall Study
STARTED
1
Overall Study
COMPLETED
1
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Midostaurin and Decitabine in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia and FLT3 Mutation

Baseline characteristics by cohort

Baseline data not reported

PRIMARY outcome

Timeframe: Up to 2 years

Population: Since only one patient was accrued, patient confidentiality prevents the reporting of this patient.

The proportion of successes will be estimated by the number of successes divided by the total number of evaluable patients. Exact binomial confidence intervals for the true success proportion will be calculated.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 2 year

Population: Since only one patient was accrued, patient confidentiality prevents the reporting of this patient.

The distribution of duration of complete response will be estimated using the method of Kaplan-Meier. If there are a sufficient number of CR/CRi, a landmark analyses may be performed to compare duration of CR/CRi in patients who first achieved a CR/CRi at 2 months vs those who first achieved a CR/CRi at 8 months.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 2 years

Population: Since only one patient was accrued, patient confidentiality prevents the reporting of this patient.

The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Time from registration to death due to any cause, assessed up to 2 years

Population: Since only one patient was accrued, patient confidentiality prevents the reporting of this patient.

Estimated using the method of Kaplan-Meier. In addition, the overall survival rate at 1 year after registration will be reported.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 2 years

Population: Since only one patient was accrued, patient confidentiality prevents the reporting of this patient.

Overall response rate, estimated by the total number of complete or partial responses (CR, CRi, morphologic leukemia-free state, or PR) divided by the total number of evaluable patients Exact binomial 95% confidence intervals for the true overall response rate will be calculated.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Time from registration to the time of relapse or death due to any cause, assessed up to 2 years

Population: Since only one patient was accrued, patient confidentiality prevents the reporting of this patient.

Estimated using the method of Kaplan-Meier. In addition, the progression-free survival rate at 1 year after registration will be reported.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to day 1 of course 9

Population: Since only one patient was accrued, patient confidentiality prevents the reporting of this patient.

Prognostic and predictive factors including age and FLT3 mutation by ITD vs. TKD vs. both will be assessed. These factors will be summarized and used to help characterize the types of patients accrued to this trial. In addition, differences in the distributions of these risk factors by clinical outcome will be explored (CR/CRi vs. not, OS and disease-free survival). Nonparametric quantitative comparisons by group will be made as appropriate (Fisher's exact or Wilcoxon rank sum). Kaplan-Meier methods and log rank statistics will be used to compare between groups for time to- event measures.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to day 1 of course 9

Population: Since only one patient was accrued, patient confidentiality prevents the reporting of this patient.

MRD status will be correlated with response using Fisher's exact test. In addition, the relationship between MRD status (positive vs. negative) and disease-free survival will be evaluated using landmark analyses.

Outcome measures

Outcome data not reported

Adverse Events

Treatment (Decitabine, Midostaurin)

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

Aref Al-Kali, MD

Mayo Clinic

Phone: 507-538-0871

Results disclosure agreements

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