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

Phase 2

Terminated

Conditions: Appendix Cancer

Interventions: Drug: nintedanib

Registration Number: NCT03287947

Lead Sponsor: Wake Forest University Health Sciences

Brief Summary: The purpose of this trial is to evaluate the disease control rate of nintedanib in subjects with metastatic appendiceal cancer for whom initial fluoropyrimidine-based chemotherapy has failed. Based on previous studies, the anticancer activity of nintedanib in lung and ovarian cancer trials, along with the similarities between appendiceal and colorectal cancer and potentially ovarian cancer, warrant additional investigation for the optimal treatment of metastatic appendiceal carcinomas.

Detailed Description: The primary study objective is to evaluate the disease control rate. The secondary study objectives are to evaluate safety and toxicity, objective response rate, overall and 6-month progression free survival, and overall survival. Exploratory study objectives include evaluation of serum and ascites VEGF, hypertension, and paracentesis frequency in subjects with ascites at study entry.

Recruitment & Eligibility

Status: TERMINATED

Sex: All

Target Recruitment: 5

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
A	nintedanib	Nintedanib

Primary Outcome Measures

Name	Time	Method
Disease Control Rate	From first dose of study drug to date of progression as determined by RECIST 1.1, assessed up to 7.5 months.	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.

Secondary Outcome Measures

Name	Time	Method
Progression-free Survival	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.	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.
Overall Survival	From date of first dose of study treatment to the date of death from any cause, assessed up to 14.5 months.	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.
Treatment Administration of Nintedanib, as Measured by Average Daily Dose of Nintedanib.	From the first dose of study drug to the last dose, assessed up to 7.5 months.	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.