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Trial record 34 of 34 for:    Lanreotide | Neuroendocrine Tumors

TELESTAR (Telotristat Etiprate for Somatostatin Analogue Not Adequately Controlled Carcinoid Syndrome)

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ClinicalTrials.gov Identifier: NCT01677910
Recruitment Status : Completed
First Posted : September 3, 2012
Results First Posted : September 18, 2017
Last Update Posted : February 27, 2018
Sponsor:
Information provided by (Responsible Party):
Lexicon Pharmaceuticals

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Carcinoid Syndrome
Interventions Drug: Telotristat etiprate
Drug: Placebo-matching telotristat etiprate
Enrollment 135
Recruitment Details Participants took part in the study at 48 investigative sites in Australia, Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Spain, Sweden, United Kingdom, and the United States from 08 January 2013 to 21 March 2016.
Pre-assignment Details Patients with Carcinoid Syndrome not adequately controlled by somatostatin analog (SSA) therapy were assigned in a 1:1:1 ratio to receive placebo, 250 mg or 500 mg telotristat etiprate (LX1606) in the double-blind period and were eligible to receive 500 mg telotristat etiprate in the 36 week open-label extension. 136 randomized;1 patient twice.
Arm/Group Title Placebo 250 mg Telotristat Etiprate 500 mg Telotristat Etiprate Telotristat Etiprate Open-Label Extension
Hide Arm/Group Description Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive two placebo-matching telotristat etiprate tablets administered three times daily for 12 weeks in the double-blind treatment period, followed by a 36 week open-label extension period. Following a 3 to 4-week run-in period on stable-dose somatostatin analog (SSA) therapy (octreotide or lanreotide) participants were randomized to receive one 250 mg telotristat etiprate tablet plus one placebo-matching telotristat etiprate tablet administered three times daily for 12 Weeks in the double-blind treatment period, followed by a 36 week open-label extension period. Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive, one telotristat etiprate 250 mg plus one placebo-matching telotristat etiprate tablet administered 3 times daily for 1 week, followed by two telotristat etiprate (250 mg) tablets administered three times daily for 11 weeks in the double-blind treatment period, followed by a 36 week open-label extension period. Patients previously assigned to 250 mg or 500 mg three times daily of telotristat etiprate were administered two 250 mg telotristat etiprate tablets three times daily in a 36 week open-label extension (OLE) period. Patients previously assigned to placebo were administered one 250 mg telotristat etiprate tablet plus one placebo-matching tablet three times daily for one week, followed by two 250 mg telotristat etiprate tablets three times daily for 35 weeks.
Period Title: Double-Blind Treatment Period
Started 45 45 45 0
Completed 38 42 38 0
Not Completed 7 3 7 0
Reason Not Completed
Adverse Event             6             2             3             0
Physician Decision             0             0             1             0
Withdrawal of consent             1             0             3             0
Reason Not Specified             0             1             0             0
Period Title: Open-Label Extension Period (OLE)
Started 0 0 0 115
Completed 0 0 0 79
Not Completed 0 0 0 36
Reason Not Completed
Physician Decision             0             0             0             4
Reason not Specified             0             0             0             2
Withdrawal of Consent             0             0             0             9
Lack of Efficacy             0             0             0             5
Adverse Event             0             0             0             15
Lost to Follow-up             0             0             0             1
Arm/Group Title Placebo 250 mg Telotristat Etiprate 500 mg Telotristat Etiprate Total
Hide Arm/Group Description Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive two placebo-matching telotristat etiprate tablets administered three times daily for 12 weeks in the double-blind treatment period, followed by a 36 week open-label extension period. Following a 3 to 4-week run-in period on stable-dose somatostatin analog (SSA) therapy (octreotide or lanreotide) participants were randomized to receive one 250 mg telotristat etiprate tablet plus one placebo-matching telotristat etiprate tablet administered three times daily for 12 Weeks in the double-blind treatment period, followed by a 36 week open-label extension period. Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive, one telotristat etiprate 250 mg plus one placebo-matching telotristat etiprate tablet administered 3 times daily for 1 week, followed by two telotristat etiprate (250 mg) tablets administered three times daily for 11 weeks in the double-blind treatment period, followed by a 36 week open-label extension period. Total of all reporting groups
Overall Number of Baseline Participants 45 45 45 135
Hide Baseline Analysis Population Description
Safety population
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 45 participants 45 participants 45 participants 135 participants
63.3  (8.67) 62.4  (9.12) 64.9  (9.06) 63.2  (9.28)
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
< 65 years Number Analyzed 45 participants 45 participants 45 participants 135 participants
25
  55.6%
26
  57.8%
22
  48.9%
73
  54.1%
≥ 65 years Number Analyzed 45 participants 45 participants 45 participants 135 participants
20
  44.4%
19
  42.2%
23
  51.1%
62
  45.9%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 45 participants 45 participants 135 participants
Female
21
  46.7%
24
  53.3%
20
  44.4%
65
  48.1%
Male
24
  53.3%
21
  46.7%
25
  55.6%
70
  51.9%
Ethnicity (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 45 participants 45 participants 135 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
1
   2.2%
1
   0.7%
Not Hispanic or Latino
45
 100.0%
44
  97.8%
44
  97.8%
133
  98.5%
Unknown or Not Reported
0
   0.0%
1
   2.2%
0
   0.0%
1
   0.7%
[1]
Measure Description: Ethnicity data was not provided for 1 participant in France.
Race (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 45 participants 45 participants 135 participants
American Indian or Alaska Native
1
   2.2%
0
   0.0%
0
   0.0%
1
   0.7%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   2.2%
0
   0.0%
0
   0.0%
1
   0.7%
White
40
  88.9%
41
  91.1%
40
  88.9%
121
  89.6%
More than one race
0
   0.0%
0
   0.0%
1
   2.2%
1
   0.7%
Unknown or Not Reported
3
   6.7%
4
   8.9%
4
   8.9%
11
   8.1%
[1]
Measure Analysis Population Description: Race information was not provided for 11 participants from France.
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Canada Number Analyzed 45 participants 45 participants 45 participants 135 participants
3 2 2 7
Sweden Number Analyzed 45 participants 45 participants 45 participants 135 participants
3 3 3 9
Netherlands Number Analyzed 45 participants 45 participants 45 participants 135 participants
2 3 3 8
Belgium Number Analyzed 45 participants 45 participants 45 participants 135 participants
1 0 0 1
United States Number Analyzed 45 participants 45 participants 45 participants 135 participants
12 13 12 37
United Kingdom Number Analyzed 45 participants 45 participants 45 participants 135 participants
8 3 8 19
Italy Number Analyzed 45 participants 45 participants 45 participants 135 participants
1 4 3 8
Israel Number Analyzed 45 participants 45 participants 45 participants 135 participants
2 0 0 2
Australia Number Analyzed 45 participants 45 participants 45 participants 135 participants
1 2 3 6
France Number Analyzed 45 participants 45 participants 45 participants 135 participants
3 4 4 11
Germany Number Analyzed 45 participants 45 participants 45 participants 135 participants
5 8 6 19
Spain Number Analyzed 45 participants 45 participants 45 participants 135 participants
4 3 1 8
Somatostatin Analog (SSA) Therapy Schedule at Study Entry   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 45 participants 45 participants 135 participants
3-Week
11
  24.4%
11
  24.4%
17
  37.8%
39
  28.9%
4-Week
34
  75.6%
34
  75.6%
28
  62.2%
96
  71.1%
[1]
Measure Description: Patients who were on a 2-week SSA therapy or receiving SSA therapy via a subcutaneous continuous infusion pump are included in the "4-week" category.
SSA Therapy Name at Study Entry  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 45 participants 45 participants 135 participants
Octreotide
30
  66.7%
40
  88.9%
33
  73.3%
103
  76.3%
Lanreotide
15
  33.3%
5
  11.1%
12
  26.7%
32
  23.7%
Childbearing Potential  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 45 participants 45 participants 135 participants
Yes
3
   6.7%
0
   0.0%
0
   0.0%
3
   2.2%
No
18
  40.0%
24
  53.3%
20
  44.4%
62
  45.9%
Not Applicable
24
  53.3%
21
  46.7%
25
  55.6%
70
  51.9%
Urinary 5-hydroxyindoleacetic acid (u5-HIAA) at Randomization   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 45 participants 45 participants 135 participants
≤ULN
12
  26.7%
12
  26.7%
12
  26.7%
36
  26.7%
>ULN
26
  57.8%
26
  57.8%
26
  57.8%
78
  57.8%
Unknown
7
  15.6%
7
  15.6%
7
  15.6%
21
  15.6%
[1]
Measure Description: ULN=upper limit of normal
Region   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 45 participants 45 participants 135 participants
North America
15
  33.3%
15
  33.3%
14
  31.1%
44
  32.6%
Europe
27
  60.0%
28
  62.2%
28
  62.2%
83
  61.5%
Rest of the World
3
   6.7%
2
   4.4%
3
   6.7%
8
   5.9%
[1]
Measure Description: North America includes USA and Canada; Europe includes Belgium, France, Germany, Italy, Netherlands, Spain, Sweden, and United Kingdom; Rest of the World includes Australia and Israel.
Weight   [1] 
Mean (Standard Deviation)
Unit of measure:  Kilogram (kg)
Number Analyzed 43 participants 44 participants 44 participants 131 participants
70.87  (13.940) 70.05  (14.832) 73.44  (19.971) 71.46  (16.419)
[1]
Measure Analysis Population Description: Weight data was not available for all participants.
Height   [1] 
Mean (Standard Deviation)
Unit of measure:  Centimeter (cm)
Number Analyzed 39 participants 41 participants 40 participants 120 participants
168.8  (10.707) 169.32  (9.607) 169.93  (10.436) 169.35  (10.175)
[1]
Measure Analysis Population Description: Height data was not available for all participants.
Body Mass Index (BMI)   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Kg/m^2
Number Analyzed 38 participants 41 participants 39 participants 118 participants
25.13  (4.790) 24.26  (4.702) 25.24  (5.352) 24.87  (4.931)
[1]
Measure Description: BMI was calculated by weight (kg) / (height (cm) *0.01)^2.
[2]
Measure Analysis Population Description: Not all participants had weight and height data available to calculate BMI.
1.Primary Outcome
Title Change From Baseline in the Number of Bowel Movements (BMs) Per Day Averaged Over 12 Weeks
Hide Description Participants recorded the number of bowel movements per day in a daily diary. The total number of BMs per day were averaged over the 12-week period. A negative change from Baseline indicates improvement.
Time Frame Baseline and 12 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Intent-to-treat population, all randomized participants, with data available were included in the analyses.
Arm/Group Title Placebo 250 mg Telotristat Etiprate 500 mg Telotristat Etiprate
Hide Arm/Group Description:
Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive two placebo-matching telotristat etiprate tablets administered three times daily for 12 weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Following a 3 to 4-week run-in period on stable-dose somatostatin analog (SSA) therapy (octreotide or lanreotide) participants were randomized to receive one 250 mg telotristat etiprate tablet plus one placebo-matching telotristat etiprate tablet administered three times daily for 12 Weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive, one telotristat etiprate 250 mg plus one placebo-matching telotristat etiprate tablet administered 3 times daily for 1 week, followed by two telotristat etiprate (250 mg) tablets administered three times daily for 11 weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Overall Number of Participants Analyzed 45 45 45
Mean (Standard Deviation)
Unit of Measure: counts/day
-0.623  (0.8275) -1.433  (1.3652) -1.455  (1.3098)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, 250 mg Telotristat Etiprate
Comments Primary analysis used a blocked 2-sample Wilcoxon rank sum statistic stratified by the urinary 5-HIAA stratification at randomization.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value < 0.001
Comments [Not Specified]
Method Wilcoxon rank sum
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.81
Confidence Interval (2-Sided) 95%
-1.283 to -0.337
Estimation Comments Mean difference is calculated as LX1606-Placebo
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, 500 mg Telotristat Etiprate
Comments Primary analysis used a blocked 2-sample Wilcoxon rank sum statistic stratified by the urinary 5-HIAA stratification at randomization.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value < 0.001
Comments [Not Specified]
Method Wilcoxon rank sum
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.833
Confidence Interval (2-Sided) 95%
-1.292 to -0.374
Estimation Comments Mean difference is calculated as LX1606-Placebo
2.Primary Outcome
Title Number of Participants With Treatment-Emergent Adverse Events (TEAEs) in the Double-Blind Treatment Period
Hide Description An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. A TEAE was an AE reported after the first dose of randomized treatment on Day 1.
Time Frame First dose of study drug to within 30 days of last dose of study drug in the Double-Blind Treatment Period (Up to 17.6 Weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population, defined as all subjects who received at least one dose of study drug was used for analysis.
Arm/Group Title Placebo 250 mg Telotristat Etiprate 500 mg Telotristat Etiprate
Hide Arm/Group Description:
Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive two placebo-matching telotristat etiprate tablets administered three times daily for 12 weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Following a 3 to 4-week run-in period on stable-dose somatostatin analog (SSA) therapy (octreotide or lanreotide) participants were randomized to receive one 250 mg telotristat etiprate tablet plus one placebo-matching telotristat etiprate tablet administered three times daily for 12 Weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive, one telotristat etiprate 250 mg plus one placebo-matching telotristat etiprate tablet administered 3 times daily for 1 week, followed by two telotristat etiprate (250 mg) tablets administered three times daily for 11 weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Overall Number of Participants Analyzed 45 45 45
Measure Type: Count of Participants
Unit of Measure: Participants
39
  86.7%
37
  82.2%
42
  93.3%
3.Primary Outcome
Title Number of Participants With TEAEs in the Open-Label Extension Period
Hide Description An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. A TEAE was an AE reported after the first dose of randomized treatment on Day 1.
Time Frame First dose of study drug to within 30 days of last dose of study drug in the Open-Label Extension Period (Up to 54.3 Weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population, defined as all subjects who received at least one dose of study drug was used for analysis.
Arm/Group Title Telotristat Etiprate Open-Label Extension
Hide Arm/Group Description:
Patients previously assigned to 250 mg or 500 mg three times daily of telotristat etiprate were administered two 250 mg telotristat etiprate tablets three times daily in a 36 week open-label extension (OLE) period. Patients previously assigned to placebo were administered one 250 mg telotristat etiprate tablet plus one placebo-matching tablet three times daily for one week, followed by two 250 mg telotristat etiprate tablets three times daily for 35 weeks.
Overall Number of Participants Analyzed 115
Measure Type: Count of Participants
Unit of Measure: Participants
110
  95.7%
4.Secondary Outcome
Title Change From Baseline in Urinary 5-hydroxyindoleacetic Acid (u5-HIAA) Levels
Hide Description u5-HIAA is a standard test used in clinical practice to assess neuroendocrine tumor (NET) activity and is collected as a 24-hour urine specimen. A negative change from Baseline indicates improvement.
Time Frame Baseline and Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Intent-to-treat population, all randomized participants, with u5-HIAA data available at Baseline and Week 12 were included in the analyses.
Arm/Group Title Placebo 250 mg Telotristat Etiprate 500 mg Telotristat Etiprate
Hide Arm/Group Description:
Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive two placebo-matching telotristat etiprate tablets administered three times daily for 12 weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Following a 3 to 4-week run-in period on stable-dose somatostatin analog (SSA) therapy (octreotide or lanreotide) participants were randomized to receive one 250 mg telotristat etiprate tablet plus one placebo-matching telotristat etiprate tablet administered three times daily for 12 Weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive, one telotristat etiprate 250 mg plus one placebo-matching telotristat etiprate tablet administered 3 times daily for 1 week, followed by two telotristat etiprate (250 mg) tablets administered three times daily for 11 weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Overall Number of Participants Analyzed 30 32 31
Mean (Standard Deviation)
Unit of Measure: mg/24 hours
11.350  (35.0346) -40.134  (84.7663) -57.519  (82.3273)
5.Secondary Outcome
Title Change From Baseline in the Number of Daily Cutaneous Flushing Episodes Averaged Across All Time-Points
Hide Description Participants recorded the number daily flushing episodes per day in a daily diary. The total number of flushing episodes per day were averaged over the 12-week period. A negative change from Baseline indicates improvement.
Time Frame Baseline and 12 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Intent-to-treat population, all randomized participants, with data available were included in the analyses.
Arm/Group Title Placebo 250 mg Telotristat Etiprate 500 mg Telotristat Etiprate
Hide Arm/Group Description:
Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive two placebo-matching telotristat etiprate tablets administered three times daily for 12 weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Following a 3 to 4-week run-in period on stable-dose somatostatin analog (SSA) therapy (octreotide or lanreotide) participants were randomized to receive one 250 mg telotristat etiprate tablet plus one placebo-matching telotristat etiprate tablet administered three times daily for 12 Weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive, one telotristat etiprate 250 mg plus one placebo-matching telotristat etiprate tablet administered 3 times daily for 1 week, followed by two telotristat etiprate (250 mg) tablets administered three times daily for 11 weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Overall Number of Participants Analyzed 45 45 45
Mean (Standard Deviation)
Unit of Measure: counts/day
-0.164  (1.1572) -0.296  (1.3097) -0.525  (1.3413)
6.Secondary Outcome
Title Change From Baseline in Abdominal Pain Averaged Across All Time-Points
Hide Description Participants recorded abdominal pain in a daily diary. Participants evaluated the level of any abdominal pain using an 11-point numeric rating scale, where: 0=no pain to 10=worst pain ever experienced. The average daily abdominal pain was averaged over the 12-week period. A negative change from Baseline indicates improvement.
Time Frame Baseline and 12 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Intent-to-treat population, all randomized participants, with available data were included in the analyses.
Arm/Group Title Placebo 250 mg Telotristat Etiprate 500 mg Telotristat Etiprate
Hide Arm/Group Description:
Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive two placebo-matching telotristat etiprate tablets administered three times daily for 12 weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Following a 3 to 4-week run-in period on stable-dose somatostatin analog (SSA) therapy (octreotide or lanreotide) participants were randomized to receive one 250 mg telotristat etiprate tablet plus one placebo-matching telotristat etiprate tablet administered three times daily for 12 Weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive, one telotristat etiprate 250 mg plus one placebo-matching telotristat etiprate tablet administered 3 times daily for 1 week, followed by two telotristat etiprate (250 mg) tablets administered three times daily for 11 weeks in the double-blind treatment period, followed by a 36 week open-label extension period.
Overall Number of Participants Analyzed 45 45 45
Mean (Standard Deviation)
Unit of Measure: units on a scale
-0.226  (1.1601) -0.489  (1.4423) -0.333  (1.1784)
Time Frame First dose of study drug to within 30 days of last dose of study drug (Up to 72.2 Weeks)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo 250 mg Telotristat Etiprate 500 mg Telotristat Etiprate Telotristat Etiprate Open-Label Extension
Hide Arm/Group Description Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive two placebo-matching telotristat etiprate tablets administered three times daily for 12 weeks in the double-blind treatment period. Following a 3 to 4-week run-in period on stable-dose somatostatin analog (SSA) therapy (octreotide or lanreotide) participants were randomized to receive one 250 mg telotristat etiprate tablet plus one placebo-matching telotristat etiprate tablet administered three times daily for 12 Weeks in the double-blind treatment period. Following a 3 to 4-week run-in period on stable-dose SSA therapy (octreotide or lanreotide) participants were randomized to receive, one 250 mg telotristat etiprate tablet plus one placebo-matching telotristat etiprate tablet administered 3 times daily for 1 week, followed by two 250 mg telotristat etiprate tablets administered three times daily for 11 weeks in the double-blind treatment period. Patients previously assigned to 250 mg or 500 mg three times daily of telotristat etiprate were administered two 250 mg telotristat etiprate tablets three times daily in a 36 week open-label extension (OLE) period. Patients previously assigned to placebo were administered one 250 mg telotristat etiprate tablet plus one placebo-matching tablet three times daily for one week, followed by two 250 mg telotristat etiprate tablets three times daily for 35 weeks.
All-Cause Mortality
Placebo 250 mg Telotristat Etiprate 500 mg Telotristat Etiprate Telotristat Etiprate Open-Label Extension
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   3/45 (6.67%)   1/45 (2.22%)   1/45 (2.22%)   9/115 (7.83%) 
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Placebo 250 mg Telotristat Etiprate 500 mg Telotristat Etiprate Telotristat Etiprate Open-Label Extension
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   7/45 (15.56%)   7/45 (15.56%)   8/45 (17.78%)   37/115 (32.17%) 
Blood and lymphatic system disorders         
Desseminated intravascular coagulation  1  0/45 (0.00%)  1/45 (2.22%)  0/45 (0.00%)  0/115 (0.00%) 
Anaemia of malignant disease  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Cardiac disorders         
Carcinoid heart disease  1  1/45 (2.22%)  0/45 (0.00%)  0/45 (0.00%)  0/115 (0.00%) 
Cardiac arrest  1  0/45 (0.00%)  0/45 (0.00%)  1/45 (2.22%)  0/115 (0.00%) 
Cardiovascular disorder  1  1/45 (2.22%)  0/45 (0.00%)  0/45 (0.00%)  0/115 (0.00%) 
Acute myocardial infarction  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Cardiac failure  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Supraventricular tachycardia  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Endocrine disorders         
Carcinoid syndrome  1  0/45 (0.00%)  0/45 (0.00%)  1/45 (2.22%)  1/115 (0.87%) 
Gastrointestinal disorders         
Abdominal Pain  1  0/45 (0.00%)  1/45 (2.22%)  0/45 (0.00%)  5/115 (4.35%) 
Constipation  1  0/45 (0.00%)  0/45 (0.00%)  1/45 (2.22%)  1/115 (0.87%) 
Ileus  1  1/45 (2.22%)  0/45 (0.00%)  0/45 (0.00%)  0/115 (0.00%) 
Rectal hemorrhage  1  0/45 (0.00%)  0/45 (0.00%)  1/45 (2.22%)  0/115 (0.00%) 
Vomiting  1  1/45 (2.22%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Nausea  1  1/45 (2.22%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Faecaloma  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  2/115 (1.74%) 
Diarrhoea  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Gastrointestinal haemorrhage  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Haematemesis  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Ileal perforation  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Large intestine perforation  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Peritoneal adhesions  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Small intestinal obstruction  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Subileus  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Colonic stenosis  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
General disorders         
Disease progression  1  1/45 (2.22%)  1/45 (2.22%)  0/45 (0.00%)  1/115 (0.87%) 
Pyrexia  1  0/45 (0.00%)  1/45 (2.22%)  0/45 (0.00%)  0/115 (0.00%) 
General physical health deterioration  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  3/115 (2.61%) 
Fatigue  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Multi-organ failure  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Pain  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Hepatobiliary disorders         
Bile duct stenosis  1  0/45 (0.00%)  1/45 (2.22%)  0/45 (0.00%)  0/115 (0.00%) 
Cholestasis  1  0/45 (0.00%)  1/45 (2.22%)  0/45 (0.00%)  0/115 (0.00%) 
Cholecystitis acute  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Infections and infestations         
Sepsis  1  1/45 (2.22%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Peritonitis  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  2/115 (1.74%) 
Catheter site infection  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Meningitis bacterial  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Pyonephrosis  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Septic shock  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Urinary tract infection  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Injury, poisoning and procedural complications         
Muscle injury  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Post embolisation syndrome  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Investigations         
Investigation  1  0/45 (0.00%)  2/45 (4.44%)  1/45 (2.22%)  4/115 (3.48%) 
Blood potassium decreased  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Cholangiogram  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Transaminases increased  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Metabolism and nutrition disorders         
Hypokalemia  1  1/45 (2.22%)  0/45 (0.00%)  1/45 (2.22%)  0/115 (0.00%) 
Cachexia  1  1/45 (2.22%)  0/45 (0.00%)  0/45 (0.00%)  0/115 (0.00%) 
Dehydration  1  0/45 (0.00%)  1/45 (2.22%)  0/45 (0.00%)  1/115 (0.87%) 
Hyponatraemia  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Musculoskeletal and connective tissue disorders         
Flank pain  1  1/45 (2.22%)  0/45 (0.00%)  0/45 (0.00%)  0/115 (0.00%) 
Back pain  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Carcinoid tumour  1  1/45 (2.22%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Pancreatic neuroendocrine tumor metastatic  1  0/45 (0.00%)  1/45 (2.22%)  0/45 (0.00%)  0/115 (0.00%) 
Tumour pain  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  2/115 (1.74%) 
Breast cancer  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Neuroendocrine tumour  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Nervous system disorders         
Sensory disturbance  1  0/45 (0.00%)  0/45 (0.00%)  1/45 (2.22%)  0/115 (0.00%) 
Syncope  1  0/45 (0.00%)  0/45 (0.00%)  1/45 (2.22%)  1/115 (0.87%) 
Cognitive disorder  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Epilepsy  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Presyncope  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Psychiatric disorders         
Confusional state  1  0/45 (0.00%)  0/45 (0.00%)  1/45 (2.22%)  0/115 (0.00%) 
Renal and urinary disorders         
Haematuria  1  0/45 (0.00%)  1/45 (2.22%)  0/45 (0.00%)  0/115 (0.00%) 
Renal failure acute  1  0/45 (0.00%)  1/45 (2.22%)  0/45 (0.00%)  1/115 (0.87%) 
Haemorrhage urinary tract  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Nephrolithiasis  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Renal failure  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Respiratory, thoracic and mediastinal disorders         
Dyspnoea  1  0/45 (0.00%)  1/45 (2.22%)  0/45 (0.00%)  0/115 (0.00%) 
Surgical and medical procedures         
Skin neoplasm excision  1  0/45 (0.00%)  1/45 (2.22%)  0/45 (0.00%)  0/115 (0.00%) 
Radiotherapy  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  3/115 (2.61%) 
Chemotherapy  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Gastrostomy  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Nephrostomy tube placement  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Surgery  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Therapeutic embolisation  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Vascular disorders         
Hypertensive crisis  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
Superior vena cava syndrome  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  1/115 (0.87%) 
1
Term from vocabulary, MedDRA (15.1)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo 250 mg Telotristat Etiprate 500 mg Telotristat Etiprate Telotristat Etiprate Open-Label Extension
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   39/45 (86.67%)   37/45 (82.22%)   42/45 (93.33%)   110/115 (95.65%) 
Gastrointestinal disorders         
Nausea  1  5/45 (11.11%)  6/45 (13.33%)  14/45 (31.11%)  27/115 (23.48%) 
Abdominal pain  1  8/45 (17.78%)  5/45 (11.11%)  10/45 (22.22%)  35/115 (30.43%) 
Vomiting  1  4/45 (8.89%)  2/45 (4.44%)  5/45 (11.11%)  16/115 (13.91%) 
Abdominal pain upper  1  0/45 (0.00%)  3/45 (6.67%)  5/45 (11.11%)  13/115 (11.30%) 
Abdominal distension  1  3/45 (6.67%)  2/45 (4.44%)  1/45 (2.22%)  13/115 (11.30%) 
Diarrhoea  1  3/45 (6.67%)  3/45 (6.67%)  0/45 (0.00%)  11/115 (9.57%) 
Flatulence  1  1/45 (2.22%)  3/45 (6.67%)  2/45 (4.44%)  11/115 (9.57%) 
Dyspepsia  1  3/45 (6.67%)  1/45 (2.22%)  1/45 (2.22%)  0/115 (0.00%) 
Constipation  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  8/115 (6.96%) 
General disorders         
Fatigue  1  4/45 (8.89%)  4/45 (8.89%)  7/45 (15.56%)  13/115 (11.30%) 
Asthenia  1  3/45 (6.67%)  2/45 (4.44%)  1/45 (2.22%)  11/115 (9.57%) 
Oedema peripheral  1  2/45 (4.44%)  3/45 (6.67%)  2/45 (4.44%)  10/115 (8.70%) 
General physical health deterioration  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  8/115 (6.96%) 
Pyrexia  1  2/45 (4.44%)  3/45 (6.67%)  0/45 (0.00%)  8/115 (6.96%) 
Infections and infestations         
Nasopharyngitis  1  1/45 (2.22%)  2/45 (4.44%)  3/45 (6.67%)  6/115 (5.22%) 
Pneumonia  1  0/45 (0.00%)  0/45 (0.00%)  3/45 (6.67%)  0/115 (0.00%) 
Urinary tract infection  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  10/115 (8.70%) 
Investigations         
Gamma-glutamyl transferase increased  1  0/45 (0.00%)  4/45 (8.89%)  4/45 (8.89%)  9/115 (7.83%) 
Alanine amino transferase increased  1  0/45 (0.00%)  1/45 (2.22%)  3/45 (6.67%)  0/115 (0.00%) 
Blood alkaline phosphatase increased  1  0/45 (0.00%)  0/45 (0.00%)  3/45 (6.67%)  0/115 (0.00%) 
Metabolism and nutrition disorders         
Decreased appetite  1  2/45 (4.44%)  3/45 (6.67%)  7/45 (15.56%)  13/115 (11.30%) 
Hypokalaemia  1  3/45 (6.67%)  3/45 (6.67%)  5/45 (11.11%)  8/115 (6.96%) 
Musculoskeletal and connective tissue disorders         
Back pain  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  9/115 (7.83%) 
Arthralgia  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  8/115 (6.96%) 
Muscle spasms  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  6/115 (5.22%) 
Nervous system disorders         
Headache  1  2/45 (4.44%)  5/45 (11.11%)  5/45 (11.11%)  12/115 (10.43%) 
Dizziness  1  2/45 (4.44%)  0/45 (0.00%)  4/45 (8.89%)  0/115 (0.00%) 
Memory impairment  1  3/45 (6.67%)  0/45 (0.00%)  1/45 (2.22%)  0/115 (0.00%) 
Psychiatric disorders         
Depression  1  3/45 (6.67%)  2/45 (4.44%)  7/45 (15.56%)  10/115 (8.70%) 
Depressed mood  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  9/115 (7.83%) 
Decreased interest  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  7/115 (6.09%) 
Insomnia  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  6/115 (5.22%) 
Respiratory, thoracic and mediastinal disorders         
Dyspnoea  1  0/45 (0.00%)  2/45 (4.44%)  5/45 (11.11%)  7/115 (6.09%) 
Cough  1  1/45 (2.22%)  1/45 (2.22%)  3/45 (6.67%)  0/115 (0.00%) 
Epistaxis  1  0/45 (0.00%)  0/45 (0.00%)  3/45 (6.67%)  0/115 (0.00%) 
Skin and subcutaneous tissue disorders         
Rash  1  3/45 (6.67%)  0/45 (0.00%)  1/45 (2.22%)  0/115 (0.00%) 
Vascular disorders         
Flushing  1  2/45 (4.44%)  3/45 (6.67%)  3/45 (6.67%)  7/115 (6.09%) 
Hypertension  1  0/45 (0.00%)  0/45 (0.00%)  0/45 (0.00%)  6/115 (5.22%) 
1
Term from vocabulary, MedDRA (15.1)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Institution must provide any proposed publication or presentation to Sponsor for Sponsor's review, comment and approval at least thirty (30) days prior to the proposed submission for publication date or the proposed presentation date. Sponsor shall have the right to have deleted from the final version of the publication any confidential information, proprietary information, or patentable subject matter.
Results Point of Contact
Name/Title: Pablo Lapuerta, MD
Organization: Lexicon Pharmaceuticals, Inc.
Responsible Party: Lexicon Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01677910     History of Changes
Other Study ID Numbers: LX1606.1-301-CS
LX1606.301 ( Other Identifier: Lexicon Pharmaceuticals, Inc. )
2012-003460-47 ( EudraCT Number )
First Submitted: August 30, 2012
First Posted: September 3, 2012
Results First Submitted: March 29, 2017
Results First Posted: September 18, 2017
Last Update Posted: February 27, 2018