Trial Outcomes & Findings for Trametinib in Patients With Advanced Neurofibromatosis Type 1 (NF1)-Mutant Non-small Cell Lung Cancer (NCT NCT03232892)
NCT ID: NCT03232892
Last Updated: 2019-12-03
Results Overview
For participants receiving at least one dose of study treatment, the ORR is defined as the best overall response recorded from the start of the treatment until disease progression or recurrence as assessed over a 1-year period from the start of treatment. The frequency and percentages of patients with a best overall response rate of complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD) will be determined. We will test the hypothesis that the ORR is greater than the null hypothesis of 10% using the Fisher's exact test.
TERMINATED
PHASE2
1 participants
Up to 1 year
2019-12-03
Participant Flow
Participant milestones
| Measure |
Trametinib 2.0mg PO Daily
Trametinib 2.0mg PO daily in 28-day cycles. A maximum of two trametinib dose level reductions are allowed (1.5mg and 1mg) in the case of adverse reactions.
Trametinib: Trametinib 2mg, once daily, PO, 28-day cycles
|
|---|---|
|
Overall Study
STARTED
|
1
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Trametinib in Patients With Advanced Neurofibromatosis Type 1 (NF1)-Mutant Non-small Cell Lung Cancer
Baseline characteristics by cohort
| Measure |
Trametinib 2.0mg PO Daily
n=1 Participants
Trametinib 2.0mg PO daily in 28-day cycles. A maximum of two trametinib dose level reductions are allowed (1.5mg and 1mg) in the case of adverse reactions.
Trametinib: Trametinib 2mg, once daily, PO, 28-day cycles
|
|---|---|
|
Age, Customized
60-69 years old
|
1 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Up to 1 yearPopulation: The single participant did not allow for a sufficient data collection of endpoint analysis
For participants receiving at least one dose of study treatment, the ORR is defined as the best overall response recorded from the start of the treatment until disease progression or recurrence as assessed over a 1-year period from the start of treatment. The frequency and percentages of patients with a best overall response rate of complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD) will be determined. We will test the hypothesis that the ORR is greater than the null hypothesis of 10% using the Fisher's exact test.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 4 yearsPopulation: The single participant did not allow for a sufficient data collection of endpoint analysis
The DR for Complete Response (CR) and Partial Response (PR) will be measured from the date that the best response is first recorded until the date that PD is documented. For patients who continue treatment post progression, the date of Disease Progression (PD) documentation will be used for analysis. The DR will be summarized using descriptive statistics (N, mean, standard deviation, minimum, and maximum).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 4 yearsPopulation: The single participant did not allow for a sufficient data collection of endpoint analysis
DCR will be defined as the percentage of patients who have achieved CR, PR, or SD for at least 12 weeks. The DCR will be summarized using descriptive statistics (N, mean, standard deviation, minimum, and maximum).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 1 yearPopulation: The single participant did not allow for a sufficient data collection of endpoint analysis
PFS will be calculated as 1+ the number of days from the first dose of study drugs to documented radiographic progression or death due to any cause over a period of 1 year. For patients who continue treatment post-progression, the date of radiographic progression will be used for PFS analysis. For patients who continue treatment post-progression, the date of radiographic progression will be used for PFS analysis. The Kaplan-Meier analysis will be used to calculate the median PFS with 95% confidence interval.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 1 yearPopulation: The single participant did not allow for a sufficient data collection of endpoint analysis
OS will be calculated as 1+ the number of days from the first dose of study drugs to death due to any cause over a period of 1 year. The Kaplan-Meier analysis will be used to calculate the median OS with 95% confidence interval.
Outcome measures
Outcome data not reported
Adverse Events
Trametinib 2.0mg PO Daily
Serious adverse events
| Measure |
Trametinib 2.0mg PO Daily
n=1 participants at risk
Trametinib 2.0mg PO daily in 28-day cycles. A maximum of two trametinib dose level reductions are allowed (1.5mg and 1mg) in the case of adverse reactions.
Trametinib: Trametinib 2mg, once daily, PO, 28-day cycles
|
|---|---|
|
Cardiac disorders
shortness of breath
|
100.0%
1/1 • Number of events 1 • Up to 1 year
The safety population will consist of all subjects who receive any amount of study treatment which for this protocol is a total of 1 participant. Adverse event terms recorded on the CRFs were be mapped to preferred terms using the Medical Dictionary for Regulatory Activities (MedDRA). Seriousness, severity grade and relationship to study treatment will be assessed by the investigator. Severity grade will be defined by the National Cancer Institute (NCI) CTCAE v4.03
|
|
Respiratory, thoracic and mediastinal disorders
hypoxic respiratory failure
|
100.0%
1/1 • Number of events 1 • Up to 1 year
The safety population will consist of all subjects who receive any amount of study treatment which for this protocol is a total of 1 participant. Adverse event terms recorded on the CRFs were be mapped to preferred terms using the Medical Dictionary for Regulatory Activities (MedDRA). Seriousness, severity grade and relationship to study treatment will be assessed by the investigator. Severity grade will be defined by the National Cancer Institute (NCI) CTCAE v4.03
|
Other adverse events
| Measure |
Trametinib 2.0mg PO Daily
n=1 participants at risk
Trametinib 2.0mg PO daily in 28-day cycles. A maximum of two trametinib dose level reductions are allowed (1.5mg and 1mg) in the case of adverse reactions.
Trametinib: Trametinib 2mg, once daily, PO, 28-day cycles
|
|---|---|
|
Gastrointestinal disorders
Nausea
|
100.0%
1/1 • Number of events 1 • Up to 1 year
The safety population will consist of all subjects who receive any amount of study treatment which for this protocol is a total of 1 participant. Adverse event terms recorded on the CRFs were be mapped to preferred terms using the Medical Dictionary for Regulatory Activities (MedDRA). Seriousness, severity grade and relationship to study treatment will be assessed by the investigator. Severity grade will be defined by the National Cancer Institute (NCI) CTCAE v4.03
|
Additional Information
Dr. Collin Blakely, MD PhD.
University of California, San Francisco
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place