Trial Outcomes & Findings for LCI-GI-APX-NIN-001: Nintedanib in Metastatic Appendiceal Carcinoma (NCT NCT03287947)

Last Updated: 2022-08-09

Results Overview

The disease control rate is the proportion of those subjects with complete response, partial response, or stable disease, as defined by Response Evaluation Criteria in Solid Tumors Criteria (RECIST 1.1). Per RECIST 1.1 criteria for target lesions assessed by radiologic evaluation of CT and tumor measurements: Complete Response (CR), Disappearance of all target and non-target lesions, any pathological lymph nodes reduced in short axis to \<10 mm; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Stable Disease (SD), Neither sufficient shrinkage to qualify for PR nor PD; Disease Control Rate (DCR) = CR + PR + SD.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

5 participants

Primary outcome timeframe

From first dose of study drug to date of progression as determined by RECIST 1.1, assessed up to 7.5 months.

Results posted on

2022-08-09

Participant Flow

Participant milestones

Participant milestones
Measure	Nintedanib Participants received 200 mg oral nintedanib, taken twice daily.
Overall Study STARTED	5
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

LCI-GI-APX-NIN-001: Nintedanib in Metastatic Appendiceal Carcinoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Nintedanib n=5 Participants Subjects received 200 mg oral nintedanib, taken twice daily.
Age, Continuous	62.8 years STANDARD_DEVIATION 15.8 • n=99 Participants
Sex: Female, Male Female	5 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	5 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	4 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
Ascites present at baseline	1 Participants n=99 Participants

PRIMARY outcome

Timeframe: From first dose of study drug to date of progression as determined by RECIST 1.1, assessed up to 7.5 months.

Population: Subjects enrolled to the study at their initiations of nintedanib therapy and had measurable disease at baseline; four of five enrolled subjects had measurable disease at baseline.

Outcome measures

Outcome measures
Measure	Nintedanib n=4 Participants Subjects received 200 mg oral nintedanib, taken twice daily.
Disease Control Rate	.25 Proportion of participants Interval 0.0064 to 0.8058

SECONDARY outcome

Timeframe: From date of first dose of study treatment to the date of death from any cause, assessed up to 14.5 months.

Population: All five enrolled subjects are evaluable for the analysis of overall survival.

Overall survival was defined as the duration from the start of nintedanib treatment to the date of death from any cause; subjects who are alive or lost to follow-up at the time of the analysis were censored at the last known date they were alive. Median overall survival was estimated using Kaplan-Meier methods. No formal comparative statistical analysis of overall survival was performed due to low accrual.

Outcome measures

Outcome measures
Measure	Nintedanib n=5 Participants Subjects received 200 mg oral nintedanib, taken twice daily.
Overall Survival	2.62 months Interval 0.78 to 14.32

SECONDARY outcome

Timeframe: From date of first dose of study treatment to the date of progressive disease or death from any cause, whichever occurred first, assessed up to 7.5 months.

Population: All five enrolled subjects are evaluable for the analysis of progression-free survival.

Progression-free survival was defined as the duration from the start of nintedanib treatment to the first occurrence of either progressive disease or death; disease progression was objectively determined per Response Evaluation Criteria in Solid Tumors Criteria (RECIST 1.1) or subjectively determined by the investigator. Per RECIST 1.1 criteria for target lesions assessed by radiologic evaluation of CT and tumor measurements: Progressive Disease (PD), \>= 20% increase in the sum of the longest diameter of target lesions (at least 5 mm), or a measurable increase or progression in a non-target lesion, or the appearance of new lesions. Median progression-free survival was estimated using Kaplan-Meier methods. No formal comparative statistical analysis of progression-free survival was performed due to low accrual.

Outcome measures

Outcome measures
Measure	Nintedanib n=5 Participants Subjects received 200 mg oral nintedanib, taken twice daily.
Progression-free Survival	1.34 months Interval 0.75 to 7.29

SECONDARY outcome

Timeframe: From the first dose of study drug to the last dose, assessed up to 7.5 months.

Population: Enrolled subjects returning bottles for drug accountability are included in the reporting of average daily dose of nintedanib.

The average daily dose of nintedanib is calculated as the total cumulative dose (in mg) of nintedanib administered divided by the number of 28-day cycles on nintedanib treatment. Prescribed daily dose of nintedanib is 400 mg.

Outcome measures

Outcome measures
Measure	Nintedanib n=3 Participants Subjects received 200 mg oral nintedanib, taken twice daily.
Treatment Administration of Nintedanib, as Measured by Average Daily Dose of Nintedanib.	350.4 mg per cycle day Standard Deviation 48.4

Adverse Events

Nintedanib

Serious events: 3 serious events

Other events: 4 other events

Deaths: 5 deaths

Serious adverse events

Serious adverse events
Measure	Nintedanib n=5 participants at risk Subjects received 200 mg oral nintedanib, taken twice daily.
General disorders Death NOS	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Cardiac disorders Cardiac arrest	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Gastrointestinal disorders Duodenal obstruction	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.

Other adverse events

Other adverse events
Measure	Nintedanib n=5 participants at risk Subjects received 200 mg oral nintedanib, taken twice daily.
Gastrointestinal disorders Abdominal distension	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Gastrointestinal disorders Abdominal pain	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Metabolism and nutrition disorders Anorexia	40.0% 2/5 • Number of events 2 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Cardiac disorders Cardiac disorders - Other, Bradycardia	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Metabolism and nutrition disorders Dehydration	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Gastrointestinal disorders Diarrhea	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Skin and subcutaneous tissue disorders Dry skin	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Eye disorders Eye pain	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
General disorders Fatigue	40.0% 2/5 • Number of events 2 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Metabolism and nutrition disorders Hypokalemia	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
General disorders Localized edema	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Musculoskeletal and connective tissue disorders Myalgia	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Gastrointestinal disorders Nausea	40.0% 2/5 • Number of events 3 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Skin and subcutaneous tissue disorders Scalp pain	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Infections and infestations Urinary tract infection	20.0% 1/5 • Number of events 1 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Gastrointestinal disorders Vomiting	40.0% 2/5 • Number of events 3 • Adverse event data was collected for subjects from enrollment until 30 days after last dose of study drug, assessed up to 8.5 months. All-Cause Mortality data was collected from enrollment until death, assessed up to 14.5 months. The attribution and severity of adverse events were classified and recorded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.

Additional Information

Danielle M Boselli

Atrium Health/Levine Cancer Institute, Department of Cancer Biostatistics

Phone: 12017903385

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place