Trial Outcomes & Findings for A Safety and Efficacy Study of XERMELO® + First-line Chemotherapy in Patients With Advanced Biliary Tract Cancer (NCT NCT03790111)
NCT ID: NCT03790111
Last Updated: 2023-04-19
Results Overview
Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions", or similar definition as accurate and appropriate.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
53 participants
Primary outcome timeframe
Month 6
Results posted on
2023-04-19
Participant Flow
Participant milestones
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Overall Study
STARTED
|
53
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
53
|
Reasons for withdrawal
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Overall Study
Study did not meet its primary endpoint.
|
53
|
Baseline Characteristics
A Safety and Efficacy Study of XERMELO® + First-line Chemotherapy in Patients With Advanced Biliary Tract Cancer
Baseline characteristics by cohort
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
22 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
31 Participants
n=5 Participants
|
|
Age, Continuous
|
63.4 Years
STANDARD_DEVIATION 10.96 • n=5 Participants
|
|
Sex: Female, Male
Female
|
33 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
50 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
46 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
53 participants
n=5 Participants
|
|
Height
|
66.4 inches
STANDARD_DEVIATION 3.37 • n=5 Participants
|
|
Weight
|
81.9 kilogram
STANDARD_DEVIATION 17.40 • n=5 Participants
|
|
Body Mass Index (BMI)
|
28.85 kg/m2
STANDARD_DEVIATION 6.343 • n=5 Participants
|
|
Baseline Plasma 5-Hydroxyindoleacetic Acid (5-HIAA) Levels </= to ULN
</= ULN
|
31 Participants
n=5 Participants
|
|
Baseline Plasma 5-Hydroxyindoleacetic Acid (5-HIAA) Levels </= to ULN
>ULN
|
22 Participants
n=5 Participants
|
|
Serum CA19-9
Normal
|
19 Participants
n=5 Participants
|
|
Serum CA19-9
Abnormal
|
32 Participants
n=5 Participants
|
|
Serum CA19-9
Not Available
|
2 Participants
n=5 Participants
|
|
Serum CEA
Normal
|
25 Participants
n=5 Participants
|
|
Serum CEA
Abnormal
|
26 Participants
n=5 Participants
|
|
Serum CEA
Not Available
|
2 Participants
n=5 Participants
|
|
Serum Albumin
Normal
|
48 Participants
n=5 Participants
|
|
Serum Albumin
Abnormal
|
4 Participants
n=5 Participants
|
|
Serum Albumin
Not Available
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Month 6Population: Safety Population
Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions", or similar definition as accurate and appropriate.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment
Safety Population · Responders, Central Reviewer
|
12 Participants
|
|
Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment
Safety Population · Non-Responders, Central Reviewer
|
41 Participants
|
|
Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment
Per Protocol Population · Responders, Central Reviewer
|
12 Participants
|
|
Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment
Per Protocol Population · Non-Responders, Central Reviewer
|
30 Participants
|
|
Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment
By Treatment Cycle Population · Responders, Central Reviewer
|
12 Participants
|
|
Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment
By Treatment Cycle Population · Non-Responders, Central Reviewer
|
21 Participants
|
PRIMARY outcome
Timeframe: 6 MonthsPopulation: Safety Population
Overall Survival (OS) was defined as the time from the frist dose of study treatment until the date of death due to any cause. Duration of Overall Survival (OS) in days is defined as (Date of event/censoring- date of First dose +1). Use Kaplan Meier method to project survival rate at month 6.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Project Overall Survival Rate at Month 6
|
0.87 Proportion of participants
Interval 0.74 to 0.93
|
SECONDARY outcome
Timeframe: First dose of study treatment until the date of death due to any cause, whichever came first, a median of approximately 17.67 monthsPopulation: Safety Population
Overall Survival (OS) was defined as the time from first dose of study treatment until the date of death due to any cause.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Overall Survival (OS)
|
17.667 Months
Interval 11.567 to
Due to insufficient number of participants with events, the upper margin cannot be estimated.
|
SECONDARY outcome
Timeframe: 12 MonthsPopulation: Safety population
Overall Survival (OS) was defined as the time from the first dose of study treatment until the date of death due to any cause. Duration of Overall Survival (OS) in days is defined as (Date of event/censoring - date of first dose +1). Use Kaplan Meier method to project survival rate at month 12.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Project Overall Survival Rate at Month 12
|
0.60 Proportion of participants
Interval 0.45 to 0.72
|
SECONDARY outcome
Timeframe: Month 12Population: Safety Population
Scheduled disease assessment at Cycle 19 Day 1 was used to determine PFS response rate at Month 12.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Median Progression Free Survival
|
6.233 Months
Interval 4.067 to 7.467
|
SECONDARY outcome
Timeframe: Month 6Population: Safety Population
Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.".
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Disease Control Rate (DCR), Central Radiologist's Assessment
|
33 Participants
|
SECONDARY outcome
Timeframe: Month 12Population: Safety Population
Disease control rate (DCR) was defined as the proportion of patients with best overall response of SD longer than 6 weeks from first dosing, confirmed PR, or confirmed CR.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Disease Control Rate (DCR), Central Radiologist's Assessment
|
33 Participants
|
SECONDARY outcome
Timeframe: End of Study up to 24 monthsPopulation: Safety Population
Disease control rate (DCR) was defined as the proportion of patients with best overall response of SD longer than 6 weeks from first dosing, confirmed PR, or confirmed CR.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Disease Control Rate (DCR), Central Radiologist's Assessment
|
35 Participants
|
SECONDARY outcome
Timeframe: Month 6Population: Safety Population
Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment until the EOS considering any requirement for confirmation. (CR) + partial response (PR) at Months 6
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Overall (Objective) Response Rate (ORR), Central Radiologist's Assessment
|
7 Participants
|
SECONDARY outcome
Timeframe: Month 12Population: Safety Population
Overall response rate (ORR) was defined as the proportion of patients (Number of Responders) with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment until Month 12.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Overall (Objective) Response Rate (ORR), Central Radiologist's Assessment
|
7 Participants
|
SECONDARY outcome
Timeframe: End of Study as defined up to 24 monthsPopulation: Safety Population
Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment until the EOS considering any requirement for confirmation.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Overall (Objective) Response Rate, Central Radiologist's Assessment
|
7 Participants
|
SECONDARY outcome
Timeframe: up to 7 monthsPopulation: Safety Population
Summary of Duration of Median Progression Free Survival, Local Radiologist's Assessment. Patient progression was defined from date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 7 months.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Summary of Duration of Progression Free Survival, Local Radiologist's Assessment
|
7.000 Months
Interval 5.567 to
Due to insufficient number of participants with events, the upper margin cannot be estimated.
|
SECONDARY outcome
Timeframe: Month 12Population: Safety Population
Summary of Median Progression Free Survival, Local Radiologist's Assessment. Defined as the time from first dose of study treatment until the first date of either disease progression or death due to any cause. Scheduled disease assessment at Cycle 19 Day 1 was used to determine PFS response rate at Month 12.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Progression Free Survival, Local Radiologist's Assessment
|
7.467 Months
Interval 5.567 to 10.533
|
SECONDARY outcome
Timeframe: End of Study as defined up to 24 monthsPopulation: Safety Population
Summary of Median Progression Free Survival, Local Radiologist's Assessment, End of Study
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Progression Free Survival, Local Radiologist's Assessment
|
7.467 Months
Interval 5.567 to 10.533
|
SECONDARY outcome
Timeframe: 6 MonthsPopulation: Safety Population
Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment at Month 6.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=48 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Overall (Objective) Response Rate, Local Read
|
6 Participants
|
SECONDARY outcome
Timeframe: 12 MonthsPopulation: Safety Population
Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment at Month 12.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=48 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Overall (Objective) Response Rate, Local Reader's Assessment
|
8 Participants
|
SECONDARY outcome
Timeframe: End of Study as defined up to 24 monthsPopulation: Safety Population
Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment until the EOS considering any requirement for confirmation.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=48 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Overall (Objective) Response Rate (ORR), Local Reader's Assessment
|
8 Participants
|
SECONDARY outcome
Timeframe: Month 12 as defined by 1 yearPopulation: Safety Population
Disease control rate (DCR) was defined as the proportion of patients with best overall response of SD longer than 6 weeks from first dosing, confirmed PR, or confirmed CR.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=48 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Disease Control Rate (DCR), Local Reviewer
|
40 Participants
|
SECONDARY outcome
Timeframe: Month 6Population: Safety Population
Disease control rate (DCR), Local Reviewer, 6 Months
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=48 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Disease Control Rate (DCR), Local Reviewer
|
40 Participants
|
SECONDARY outcome
Timeframe: End of Study as defined up to 24 monthsPopulation: Safety Population
Disease control rate (DCR) was defined as the proportion of patients with best overall response of SD longer than 6 weeks from first dosing, confirmed PR, or confirmed CR.
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=48 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Disease Control Rate End of Study, Local Reviewer
|
40 Participants
|
SECONDARY outcome
Timeframe: Month 6Population: Safety Population
Mean change from Baseline to Month 6 plasma level 5-hydroxyindoleacetic acid (5-HIAA)
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=24 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Mean Change From Baseline in Plasma 5-hydroxyindoleacetic Acid (5-HIAA)
|
-1.735 micrograms/Liter
Standard Deviation 8.6933
|
SECONDARY outcome
Timeframe: Month 12Population: Safety Population
Mean change from Baseline to Month 12 in plasma level 5-hydroxyindoleacetic Acid (5-HIAA)
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=6 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Mean Change From Baseline in Plasma 5-hydroxyindoleacetic Acid (5-HIAA)
|
-5.608 micrograms/Liter
Standard Deviation 6.5192
|
SECONDARY outcome
Timeframe: End of Study as defined up to 24 monthsPopulation: Safety Population
Mean change from Baseline to End of Study in plasma 5-hydroxyindoleacetic acid (5-HIAA)
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=7 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Mean Change From Baseline in Plasma 5-hydroxyindoleacetic Acid (5-HIAA)
|
4.154 micrograms/Liter
Standard Deviation 5.7003
|
SECONDARY outcome
Timeframe: Month 6Population: Safety Population
Mean change from Baseline to month 6 in plasma carbohydrate antigen 19-9 (CA 19-9)
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=21 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Change From Baseline in Carbohydrate Antigen 19-9 (CA 19-9)
|
-229.82 U/mL
Standard Deviation 2484.902
|
SECONDARY outcome
Timeframe: Month 12Population: Safety Population
Mean change from Baseline to Month 12 in plasma carbohydrate antigen 19-9 (CA 19-9)
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=7 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Change From Baseline in Carbohydrate Antigen 19-9 (CA 19-9)
|
-18.04 U/mL
Standard Deviation 38.665
|
SECONDARY outcome
Timeframe: End of Study as defined up to 24 monthsPopulation: Safety Population
Mean change from Baseline to End of Study in plasma carbohydrate antigen 19-9 (CA 19-9)
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=8 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Change From Baseline in Plasma Carbohydrate Antigen 19-9 (CA 19-9)
|
-153.98 Units per milliliter
Standard Deviation 456.210
|
SECONDARY outcome
Timeframe: Month 6Population: Safety Population
Mean change in weight at Month 6 from baseline measurement
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=17 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Weight Change From Baseline
|
-0.69 kg
Standard Deviation 9.759
|
SECONDARY outcome
Timeframe: Month 12Population: Safety Population
Mean change in weight at Month 12 from baseline measurement
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=6 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Weight Change From Baseline
|
1.69 kg
Standard Deviation 7.473
|
SECONDARY outcome
Timeframe: End of Study as defined up to 24 monthsPopulation: Safety Population
Mean change in weight from baseline to End of Study
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=19 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Weight Change From Baseline
|
-3.28 kg
Standard Deviation 8.920
|
SECONDARY outcome
Timeframe: Month 6Population: Safety Population
Mean change from Baseline to Month 6 serum albumin levels
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=15 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Change From Baseline in Serum Albumin
|
-0.2 g/L
Standard Deviation 4.59
|
SECONDARY outcome
Timeframe: Month 12Population: Safety Population
Mean change from Baseline to Month 12 serum albumin levels
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=6 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Change From Baseline in Serum Albumin
|
-2.2 g/L
Standard Deviation 2.86
|
SECONDARY outcome
Timeframe: End of Study as defined up to 24 monthsPopulation: Safety Population
Mean change from Baseline to End of Study serum albumin levels
Outcome measures
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=9 Participants
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Change From Baseline in Serum Albumin
|
-1.2 g/L
Standard Deviation 4.99
|
Adverse Events
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
Serious events: 24 serious events
Other events: 53 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 participants at risk
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Gastrointestinal disorders
Gastrointestinal disorders
|
15.1%
8/53 • Number of events 8 • 2 years
|
|
Infections and infestations
Infections and infestations
|
9.4%
5/53 • Number of events 5 • 2 years
|
|
Hepatobiliary disorders
Hepatobiliary disorders
|
5.7%
3/53 • Number of events 3 • 2 years
|
|
Nervous system disorders
Nervous system disorders
|
5.7%
3/53 • Number of events 3 • 2 years
|
|
Cardiac disorders
Cardiac Disorders
|
3.8%
2/53 • Number of events 2 • 2 years
|
|
Investigations
Investigations
|
3.8%
2/53 • Number of events 2 • 2 years
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders
|
1.9%
1/53 • Number of events 1 • 2 years
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications
|
1.9%
1/53 • Number of events 1 • 2 years
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders
|
1.9%
1/53 • Number of events 1 • 2 years
|
|
Psychiatric disorders
Psychiatric disorders
|
1.9%
1/53 • Number of events 1 • 2 years
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders
|
1.9%
1/53 • Number of events 1 • 2 years
|
|
Vascular disorders
Vascular disorders
|
1.9%
1/53 • Number of events 1 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, Thoracic and mediastinal disorders
|
9.4%
5/53 • Number of events 5 • 2 years
|
Other adverse events
| Measure |
Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg
n=53 participants at risk
Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study
|
|---|---|
|
Gastrointestinal disorders
Constipation
|
64.2%
34/53 • Number of events 34 • 2 years
|
|
Gastrointestinal disorders
Nausea
|
62.3%
33/53 • Number of events 33 • 2 years
|
|
General disorders
Fatigue
|
60.4%
32/53 • Number of events 32 • 2 years
|
|
Blood and lymphatic system disorders
Anemia
|
50.9%
27/53 • Number of events 27 • 2 years
|
|
Blood and lymphatic system disorders
Neutrophil count decreased
|
41.5%
22/53 • Number of events 22 • 2 years
|
|
Blood and lymphatic system disorders
Platelet count decreased
|
35.8%
19/53 • Number of events 19 • 2 years
|
|
Gastrointestinal disorders
Diarrhea
|
34.0%
18/53 • Number of events 18 • 2 years
|
|
Gastrointestinal disorders
Vomiting
|
32.1%
17/53 • Number of events 17 • 2 years
|
|
Blood and lymphatic system disorders
White blood cell count decreased
|
26.4%
14/53 • Number of events 14 • 2 years
|
|
Metabolism and nutrition disorders
Decreased appetite
|
24.5%
13/53 • Number of events 13 • 2 years
|
|
General disorders
Oedema peripheral
|
22.6%
12/53 • Number of events 12 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
20.8%
11/53 • Number of events 11 • 2 years
|
|
Nervous system disorders
Headache
|
20.8%
11/53 • Number of events 11 • 2 years
|
|
Vascular disorders
Hypertension
|
20.8%
11/53 • Number of events 11 • 2 years
|
|
General disorders
Pyrexia
|
20.8%
11/53 • Number of events 11 • 2 years
|
|
Gastrointestinal disorders
Abdominal pain
|
26.4%
14/53 • Number of events 14 • 2 years
|
Additional Information
Janine North, Executive Director-Clinical Development
TerSera Therapeutics Inc.
Phone: 888 600 8116
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place