Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 32 of 277 for:    Non-alcoholic Steatohepatitis | United States

Safety and Tolerability Study of SHP626 in Overweight and Obese Adults

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02287779
Recruitment Status : Completed
First Posted : November 11, 2014
Results First Posted : December 7, 2016
Last Update Posted : March 26, 2019
Sponsor:
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Non-Alcoholic Steatohepatitis
Interventions Drug: SHP626
Drug: Placebo
Enrollment 84
Recruitment Details Study was conducted at one study centre in North America, from 19 January 2015 to 19 June 2015.
Pre-assignment Details Overall, 84 participants were randomised and completed the study.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description Participants received placebo matched to volixibat tablet orally for 12 days. Participants received volixibat (SHP626) 5 milligram (mg) capsule orally twice a day (BID) for 12 days. Participants received volixibat (SHP626) 10 mg capsule orally once a day (QD) for 12 days. Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days. Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12. Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days. Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days. Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Period Title: Overall Study
Started 21 9 9 9 9 9 9 9
Completed 21 9 9 9 9 9 9 9
Not Completed 0 0 0 0 0 0 0 0
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD Total
Hide Arm/Group Description Participants received placebo matched to volixibat tablet orally for 12 days Participants received volixibat (SHP626) 5 milligram (mg) capsule orally twice a day (BID) for 12 days. Participants received volixibat (SHP626) 10 mg capsule orally once a day (QD) for 12 days. Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days. Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12. Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days. Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days. Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12. Total of all reporting groups
Overall Number of Baseline Participants 21 9 9 9 9 9 9 9 84
Hide Baseline Analysis Population Description
Safety analysis set included all participants for whom a randomization number was assigned and who had taken greater than equal (>=) 1 dose of investigational medicinal product (IMP).
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Age Number Analyzed 21 participants 9 participants 9 participants 9 participants 9 participants 9 participants 9 participants 9 participants 84 participants
41.5  (9.47) 37.1  (15.40) 46.2  (7.61) 33.3  (9.82) 46.2  (7.82) 44.6  (10.70) 36.6  (8.92) 33.2  (11.34) 40.1  (10.93)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants 9 participants 9 participants 9 participants 9 participants 9 participants 9 participants 9 participants 84 participants
Female
3
  14.3%
0
   0.0%
2
  22.2%
0
   0.0%
0
   0.0%
1
  11.1%
0
   0.0%
0
   0.0%
6
   7.1%
Male
18
  85.7%
9
 100.0%
7
  77.8%
9
 100.0%
9
 100.0%
8
  88.9%
9
 100.0%
9
 100.0%
78
  92.9%
1.Primary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Standard Hematology
Hide Description TEAEs were defined as events that either had a start date on or after the first dose of investigational medicinal product (IMP) or has a start date before the date of the first dose of IMP, but increased in severity on or after the date of the first dose of IMP. An adverse event (AE) that occurred more than 9 days after the date of the last dose of double-blind IMP was not counted as a TEAE. Hematology parameters included evaluation of hemoglobin, hematocrit, red blood cells, platelets, white blood cell count; total and differential, neutrophils (absolute), eosinophils (absolute), monocytes (absolute), basophils (absolute) and lymphocytes (absolute).
Time Frame From the start of the study drug administration up to 9 days after the last dose of study drug administration
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set included all participants for whom a randomization number was assigned and who had taken >= 1 dose of IMP.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 milligram (mg) capsule orally twice a day (BID) for 12 days.
Participants received volixibat (SHP626) 10 mg capsule orally once a day (QD) for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 21 9 9 9 9 9 9 9
Measure Type: Number
Unit of Measure: participants
0 0 0 0 0 0 0 0
2.Primary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Fat Soluble Vitamins (Vitamin A, D, & E)
Hide Description TEAEs were defined as events that either had a start date on or after the first dose of IMP or has a start date before the date of the first dose of IMP, but increased in severity on or after the date of the first dose of IMP. An AE that occurred more than 9 days after the date of the last dose of double-blind IMP was not counted as a TEAE. Fat soluble vitamin included vitamin A (serum retinol), vitamin D (serum 25-hydroxycholecalciferol) and vitamin E (serum alfa-tocopherol).
Time Frame From the start of the study drug administration up to 9 days after the last dose of study drug administration
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule QD for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 21 9 9 9 9 9 9 9
Measure Type: Number
Unit of Measure: participants
0 0 0 0 0 0 0 0
3.Primary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Lipid Panel
Hide Description TEAEs were defined as events that either had a start date on or after the first dose of IMP or has a start date before the date of the first dose of IMP, but increased in severity on or after the date of the first dose of IMP. An AE that occurred more than 9 days after the date of the last dose of double-blind IMP was not counted as a TEAE. Lipid panel parameters included evaluation of total cholesterol, triglycerides, high-density lipoprotein (HDL) cholesterol, very low-density lipoprotein (VLDL) cholesterol and low-density lipoprotein (LDL) cholesterol.
Time Frame From the start of the study drug administration up to 9 days after the last dose of study drug administration
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 21 9 9 9 9 9 9 9
Measure Type: Number
Unit of Measure: participants
0 0 0 0 0 0 0 0
4.Primary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Thyroid Hormone Panel
Hide Description TEAEs were defined as events that either had a start date on or after the first dose of IMP or has a start date before the date of the first dose of IMP, but increased in severity on or after the date of the first dose of IMP. An AE that occurred more than 9 days after the date of the last dose of double-blind IMP was not counted as a TEAE. Thyroid hormone panel parameters included evaluation of thyroid hormones (TSH [thyroid stimulating hormone]; T3 [triiodothyronine] and T4 [thyroxine]).
Time Frame From the start of the study drug administration up to 9 days after the last dose of study drug administration
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule QD for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 21 9 9 9 9 9 9 9
Measure Type: Number
Unit of Measure: participants
0 0 0 0 0 0 0 0
5.Primary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Coagulation
Hide Description TEAEs were defined as events that either had a start date on or after the first dose of IMP or has a start date before the date of the first dose of IMP, but increased in severity on or after the date of the first dose of IMP. An AE that occurred more than 9 days after the date of the last dose of double-blind IMP was not counted as a TEAE. Coagulation included international normalized ratio, activated partial thromboplastin time and prothrombin time.
Time Frame From the start of the study drug administration up to 9 days after the last dose of study drug administration
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 21 9 9 9 9 9 9 9
Measure Type: Number
Unit of Measure: participants
0 0 0 0 0 0 0 0
6.Primary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Standard Chemistry
Hide Description TEAEs were defined as events that either had a start date on or after the first dose of IMP or has a start date before the date of the first dose of IMP, but increased in severity on or after the date of the first dose of IMP. An AE that occurred more than 9 days after the date of the last dose of double-blind IMP was not counted as a TEAE. Standard chemistry parameters included evaluation of sodium, potassium, glucose, blood urea nitrogen, creatinine, calcium, chloride, thyrotropin, thyroxine, tri-iodothyronine, phosphorus, protein, bicarbonate or carbon dioxide, albumin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyltransferase, alkaline phosphatase, total bilirubin, urate, beta-human chorionic gonadotropin and follicle-stimulating hormone levels. Participant with TEAE related to standard chemistry were reported with hepatic enzyme increase.
Time Frame From the start of the study drug administration up to 9 days after the last dose of study drug administration
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule QD for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 21 9 9 9 9 9 9 9
Measure Type: Number
Unit of Measure: participants
0 0 0 1 0 0 0 0
7.Primary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Urinalysis Parameters
Hide Description TEAEs were defined as events that either had a start date on or after the first dose of IMP or has a start date before the date of the first dose of IMP, but increased in severity on or after the date of the first dose of IMP. An AE that occurred more than 9 days after the date of the last dose of double-blind IMP was not counted as a TEAE. Urinalysis parameters included evaluation of pH, glucose, protein, nitrites, leukocyte esterase, occult blood, ketones, bilirubin and specific gravity levels.
Time Frame From the start of the study drug administration up to 9 days after the last dose of study drug administration
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule orally once a day (QD) for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 21 9 9 9 9 9 9 9
Measure Type: Number
Unit of Measure: participants
0 0 0 0 0 0 0 0
8.Primary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Vital Signs
Hide Description TEAEs were defined as events that either had a start date on or after the first dose of IMP or has a start date before the date of the first dose of IMP, but increased in severity on or after the date of the first dose of IMP. An AE that occurred more than 9 days after the date of the last dose of double-blind IMP was not counted as a TEAE. Vital signs parameter included evaluation of orthostatic blood pressure, respiratory rate and body temperature.
Time Frame From the start of the study drug administration up to 9 days after the last dose of study drug administration
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 21 9 9 9 9 9 9 9
Measure Type: Number
Unit of Measure: participants
Tachycardia 0 1 0 0 0 0 0 0
Pyrexia 0 2 0 0 0 0 0 0
9.Primary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Electrocardiogram (12-lead)
Hide Description TEAEs were defined as events that either had a start date on or after the first dose of IMP or has a start date before the date of the first dose of IMP, but increased in severity on or after the date of the first dose of IMP. An AE that occurred more than 9 days after the date of the last dose of double-blind IMP was not counted as a TEAE. Twelve lead electrocardiogram parameters [(heart rate (HR), PR, RR, QRS and QT intervals and information on T-wave morphology (normal/abnormal) and U-wave morphology (absent/normal or abnormal)] were assessed.
Time Frame From the start of the study drug administration up to 9 days after the last dose of study drug administration
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 21 9 9 9 9 9 9 9
Measure Type: Number
Unit of Measure: participants
0 0 0 0 0 0 0 0
10.Primary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Treatment-emergent Adverse Events (STEAEs)
Hide Description TEAEs were defined as events that either had a start date on or after the first dose of IMP or has a start date before the date of the first dose of IMP, but increased in severity on or after the date of the first dose of IMP. An AE that occurred more than 9 days after the date of the last dose of double-blind IMP was not counted as a TEAE.
Time Frame From the start of the study drug administration up to 9 days after the last dose of study drug administration
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 21 9 9 9 9 9 9 9
Measure Type: Number
Unit of Measure: participants
Participants with TEAEs 14 9 9 9 9 9 9 9
Participants with STEAEs 0 0 0 0 0 0 0 0
11.Primary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs) Who Discontinued From the Study
Hide Description TEAEs were defined as events that either had a start date on or after the first dose of IMP or has a start date before the date of the first dose of IMP, but increased in severity on or after the date of the first dose of IMP. An AE that occurred more than 9 days after the date of the last dose of double-blind IMP was not counted as a TEAE.
Time Frame From the start of the study drug administration up to 9 days after the last dose of study drug administration
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 21 9 9 9 9 9 9 9
Measure Type: Number
Unit of Measure: participants
0 0 0 0 0 0 0 0
12.Secondary Outcome
Title Average Total Fecal Bile Acid (FBA) Concentration
Hide Description Stool samples for the determination of total FBA were collected in 48-hour windows from 48 hours before dosing on Day 1 through Day 14. The average of daily total FBA excretion is calculated before (Day -1 and Day -2) as the first pre dose of IMP and after (Day 1-12) as the first post-dose of IMP. The FBA is calculated as Total FBA (micromoles) = FBA (micromol per liter) * weight (grams) divided by 10^3. Participants with fecal bile acid concentration and their average pre-first dose and average post-first dose were reported.
Time Frame Day -2 up to Day 14
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic set consisted of all participant in the safety analysis set for whom the primary pharmacodynamic data were considered sufficient and interpretable.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 20 8 9 9 9 9 8 9
Mean (Standard Deviation)
Unit of Measure: nanomoles*gram per liter
Average Pre First Dose 183.84  (176.229) 166.95  (84.057) 172.49  (180.992) 238.22  (211.406) 364.78  (304.905) 408.49  (475.553) 335.89  (277.883) 116.73  (91.962)
Average Post First Dose 224.75  (195.403) 941.32  (338.783) 668.64  (365.183) 1051.86  (554.781) 1055.60  (394.246) 987.01  (321.184) 1172.08  (591.774) 665.82  (539.150)
13.Secondary Outcome
Title Mean Serum 7- Alpha-hydroxy-4-cholesten-3-one (C4) Concentration
Hide Description Serum 7- alpha-hydroxy-4-cholesten-3-one (C4) concentrations were reported.
Time Frame Day -1 to Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic set.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 20 8 9 9 9 9 8 9
Mean (Standard Deviation)
Unit of Measure: nanogram per milliliter
Day -1, Pre-Dose 15.702  (19.1363) 10.956  (3.7149) 13.806  (10.0637) 11.129  (6.7566) 19.549  (12.2775) 24.726  (37.6474) 47.138  (42.7980) 22.632  (18.9895)
Day -1, 5 Hours Post-Dose 31.952  (29.6023) 17.418  (9.3636) 26.272  (24.6615) 35.649  (22.7807) 34.262  (19.5308) 42.490  (37.9101) 58.411  (61.0322) 27.311  (14.7722)
Day -1, 13 Hours Post-Dose 11.830  (10.3028) 6.224  (3.9307) 10.463  (7.9914) 15.812  (7.6084) 17.013  (9.7056) 16.459  (15.9875) 26.676  (26.9306) 13.977  (8.4524)
Baseline 17.464  (29.6114) 10.644  (6.2617) 10.441  (7.8955) 15.446  (13.4289) 26.871  (17.6706) 25.269  (34.4194) 45.714  (49.4616) 19.453  (16.9595)
Day 1, Pre-Dose 17.464  (29.6114) 10.644  (6.2617) 10.441  (7.8955) 15.446  (13.4289) 26.871  (17.6706) 25.269  (34.4194) 45.714  (49.4616) 19.453  (16.9595)
Day 1, 5 Hours Post-Dose 27.300  (21.4321) 29.041  (18.4231) 54.602  (47.7655) 60.478  (22.8216) 65.289  (22.8088) 63.769  (54.1516) 88.713  (53.8752) 35.520  (18.2429)
Day 1, 13 Hours Post-Dose 22.930  (29.6148) 32.991  (23.3494) 57.611  (28.4635) 60.922  (20.1868) 65.478  (27.8476) 88.300  (64.8546) 104.150  (65.6060) 66.311  (21.3091)
Day 6, Pre-Dose 34.048  (57.5519) 100.788  (48.0025) 103.789  (66.6396) 114.289  (81.4964) 157.718  (69.9298) 134.378  (90.2305) 190.025  (114.5808) 111.346  (91.6773)
Day 6, 5 Hours Post-Dose 33.972  (20.7411) 129.625  (71.4311) 145.111  (84.6147) 143.756  (80.3355) 185.278  (88.4269) 190.378  (96.9316) 202.788  (122.4445) 100.089  (45.3246)
Day 6, 13 Hours Post-Dose 15.847  (13.7638) 71.200  (30.2892) 84.611  (71.3102) 88.722  (68.5693) 98.122  (43.5820) 110.167  (46.8967) 129.100  (74.2770) 76.811  (40.6780)
Day 12, Pre-Dose 23.299  (26.1217) 97.538  (68.0330) 101.522  (95.1932) 118.900  (78.4954) 198.311  (87.5824) 169.778  (101.2574) 192.225  (128.8916) 107.200  (77.8666)
Day 12, 5 Hours Post-Dose 32.567  (24.7723) 120.738  (61.5278) 144.767  (130.3808) 156.689  (67.9233) 232.189  (106.7850) 196.933  (118.2849) 210.088  (117.8523) 104.167  (68.2195)
Day 12, 13 Hours Post-Dose 16.497  (12.9150) 77.636  (40.5962) 80.189  (77.9001) 90.400  (55.9921) 152.989  (81.6364) 114.500  (40.2073) 107.875  (52.3505) 66.444  (36.9048)
Day 13, Pre-Dose 21.822  (32.8831) 130.750  (85.2496) 84.544  (55.4006) 85.822  (42.7988) 184.222  (66.6179) 126.322  (65.9867) 128.738  (83.4282) 72.867  (49.7977)
Day 13, 5 Hours Post-Dose 29.110  (22.6451) 107.325  (59.7595) 94.022  (73.9921) 78.100  (34.5601) 161.556  (95.9363) 110.756  (49.6502) 89.363  (39.2784) 57.567  (28.0922)
Day 13, 13 Hours Post-Dose 22.728  (24.6379) 67.660  (46.6124) 53.333  (40.0821) 65.833  (34.3484) 78.911  (50.2001) 66.633  (23.7029) 66.550  (30.6498) 50.333  (23.8917)
Day 15, Pre-Dose 21.848  (24.0679) 14.454  (11.0078) 25.908  (18.3102) 19.906  (11.2784) 38.434  (21.0173) 38.870  (38.5249) 37.300  (26.1975) 17.563  (12.4113)
Day 15, 5 Hours Post-Dose 30.099  (19.2391) 34.663  (14.0156) 51.009  (39.8047) 30.868  (19.5278) 63.067  (34.0796) 55.257  (47.2884) 36.960  (18.2242) 26.322  (12.8678)
Day 15, 13 Hours Post-Dose 19.140  (18.9237) 21.068  (10.4419) 31.759  (28.8913) 23.667  (10.0772) 49.867  (21.1864) 29.282  (23.4813) 32.750  (22.6428) 19.173  (8.3821)
14.Secondary Outcome
Title Number of Participants With Stool Hardness Using Bristol Stool Chart
Hide Description Stool hardness was assessed after each evacuation using the bristol stool chart, a medical aid designed to classify the form of human feces into 7 categories where type 1 is the hardest and type 7 is the softest.
Time Frame Day -2 to Day 14
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic set.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 20 8 9 9 9 9 8 9
Measure Type: Number
Unit of Measure: participants
Day -2: Type 1 4 0 1 0 0 0 0 1
Day -2: Type 2 2 1 1 1 1 3 0 1
Day -2: Type 3 5 3 1 1 3 1 1 1
Day -2: Type 4 5 1 5 1 3 0 2 4
Day -2: Type 5 3 0 1 3 0 1 1 1
Day -2: Type 6 0 0 0 0 1 3 4 0
Day -2: Type 7 0 0 0 1 0 0 0 0
Day -1: Type 1 2 1 0 1 1 0 1 1
Day -1: Type 2 0 1 1 2 1 1 1 2
Day -1: Type 3 8 2 1 2 1 1 1 1
Day -1: Type 4 3 1 4 1 3 3 3 1
Day -1: Type 5 1 0 1 0 0 1 0 0
Day -1: Type 6 3 1 1 1 1 2 2 0
Day -1: Type 7 1 0 0 1 0 0 0 1
Day 1: Type 1 1 0 0 0 1 0 0 1
Day 1: Type 2 4 1 0 0 1 0 0 1
Day 1: Type 3 3 0 0 0 0 2 0 0
Day 1: Type 4 1 0 0 0 0 0 1 1
Day 1: Type 5 1 4 1 2 1 0 0 1
Day 1: Type 6 2 2 5 3 5 5 4 4
Day 1: Type 7 1 1 2 4 0 2 3 1
Day 2: Type 1 1 0 0 0 0 0 0 0
Day 2: Type 2 5 0 0 0 0 0 0 1
Day 2: Type 3 4 0 0 1 0 1 0 1
Day 2: Type 4 5 1 1 0 0 1 1 0
Day 2: Type 5 1 1 2 1 1 0 0 1
Day 2: Type 6 2 4 6 4 6 7 5 4
Day 2: Type 7 0 1 0 2 0 0 1 2
Day 3: Type 1 1 0 0 0 1 0 0 0
Day 3: Type 2 6 0 1 0 0 0 0 1
Day 3: Type 3 0 0 0 0 0 0 1 0
Day 3: Type 4 4 1 1 0 0 2 0 0
Day 3: Type 5 3 0 0 0 2 0 1 0
Day 3: Type 6 1 4 5 8 4 7 4 7
Day 3: Type 7 1 2 1 0 2 0 1 1
Day 4: Type 1 6 0 0 0 1 0 0 0
Day 4: Type 2 3 0 0 0 0 0 0 0
Day 4: Type 3 3 0 1 0 0 0 0 0
Day 4: Type 4 2 0 2 0 0 0 1 0
Day 4: Type 5 3 1 3 1 0 1 0 0
Day 4: Type 6 2 3 2 5 4 8 5 7
Day 4: Type 7 0 1 0 2 3 0 2 1
Day 5: Type 1 0 0 0 0 0 0 0 0
Day 5: Type 2 5 0 0 0 0 0 0 0
Day 5: Type 3 3 1 1 0 0 0 0 0
Day 5: Type 4 1 0 0 0 0 0 0 0
Day 5: Type 5 2 0 0 0 1 1 0 0
Day 5: Type 6 4 5 6 5 6 5 5 8
Day 5: Type 7 0 1 2 2 1 1 3 0
Day 6: Type 1 3 0 0 0 0 0 0 0
Day 6: Type 2 6 1 0 0 0 0 0 0
Day 6: Type 3 3 1 0 0 0 0 0 0
Day 6: Type 4 2 0 2 0 0 1 0 0
Day 6: Type 5 0 0 0 0 1 0 0 1
Day 6: Type 6 4 4 4 7 7 6 3 7
Day 6: Type 7 0 2 2 1 1 1 5 0
Day 7: Type 1 4 0 0 0 0 0 0 0
Day 7: Type 2 4 0 0 0 0 0 0 0
Day 7: Type 3 1 0 0 0 0 0 0 0
Day 7: Type 4 4 0 0 0 0 1 0 0
Day 7: Type 5 2 0 2 1 1 1 0 1
Day 7: Type 6 4 6 5 5 6 7 5 6
Day 7: Type 7 0 0 0 1 1 0 3 0
Day 8: Type 1 3 0 0 0 0 0 0 0
Day 8: Type 2 6 0 0 0 0 0 0 0
Day 8: Type 3 1 0 0 1 0 0 0 0
Day 8: Type 4 3 0 0 0 1 0 0 0
Day 8: Type 5 2 2 3 0 2 1 1 0
Day 8: Type 6 0 4 5 4 3 7 5 6
Day 8: Type 7 1 0 0 3 2 1 2 1
Day 9: Type 1 1 0 0 0 0 0 0 0
Day 9: Type 2 5 0 0 0 0 0 0 0
Day 9: Type 3 1 0 0 0 0 0 0 0
Day 9: Type 4 4 0 0 0 0 1 0 0
Day 9: Type 5 1 1 0 0 1 0 0 0
Day 9: Type 6 4 3 8 6 6 7 7 5
Day 9: Type 7 0 2 0 3 1 1 1 2
Day 10: Type 1 3 0 0 0 0 0 0 0
Day 10: Type 2 6 0 0 0 0 0 0 0
Day 10: Type 3 2 1 0 0 0 0 0 0
Day 10: Type 4 4 0 1 0 0 0 0 0
Day 10: Type 5 2 0 1 0 0 0 0 1
Day 10: Type 6 1 5 4 4 7 8 3 5
Day 10: Type 7 0 1 2 4 1 1 4 0
Day 11: Type 1 5 0 0 0 0 0 0 0
Day 11: Type 2 2 0 0 0 0 0 0 0
Day 11: Type 3 2 0 0 0 0 0 0 0
Day 11: Type 4 6 0 0 0 0 0 0 0
Day 11: Type 5 2 1 0 0 0 0 1 1
Day 11: Type 6 2 3 9 9 7 8 2 6
Day 11: Type 7 0 4 0 0 1 1 5 1
Day 12: Type 1 2 0 0 0 0 1 0 0
Day 12: Type 2 6 0 0 0 0 0 0 0
Day 12: Type 3 2 0 0 0 0 0 0 0
Day 12: Type 4 2 0 0 0 0 0 0 0
Day 12: Type 5 0 2 0 1 1 0 0 0
Day 12: Type 6 2 4 7 3 8 6 5 8
Day 12: Type 7 0 1 0 4 0 1 3 0
Day 13: Type 1 3 0 0 0 0 0 0 0
Day 13: Type 2 5 0 0 0 0 0 0 1
Day 13: Type 3 0 1 0 0 1 1 0 0
Day 13: Type 4 6 0 1 2 2 0 2 0
Day 13: Type 5 0 1 1 1 0 0 0 0
Day 13: Type 6 4 2 3 1 2 0 3 1
Day 13: Type 7 1 0 0 0 0 0 0 0
Day 14: Type 1 1 0 0 1 1 0 0 0
Day 14: Type 2 5 1 0 0 2 3 1 4
Day 14: Type 3 1 1 2 0 2 2 1 2
Day 14: Type 4 3 3 2 4 3 3 2 1
Day 14: Type 5 3 1 1 1 0 1 1 0
Day 14: Type 6 1 0 1 1 1 0 1 1
Day 14: Type 7 0 0 0 1 0 0 0 0
15.Secondary Outcome
Title Maximum Observed Plasma Concentration (Cmax) of Volixibat
Hide Description [Not Specified]
Time Frame Day 1 to Day 14
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic set consisted of all participants in the safety analysis set for whom the primary pharmacokinetic data were considered sufficient and interpretable. Due to minimal absorption of volixibat no participant had sufficient and interpretable primary pharmacokinetic data.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days.
Participants received volixibat (SHP626) 10 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 0 0 0 0 0 0 0 0
No data displayed because Outcome Measure has zero total analyzed.
16.Secondary Outcome
Title Area Under the Plasma Concentration-Time Curve (AUC) of Volixibat (SHP626)
Hide Description [Not Specified]
Time Frame Day 1 to Day 14
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic set. Due to minimal absorption of volixibat no participant had sufficient and interpretable primary pharmacokinetic data.
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description:
Participants received placebo matched to volixibat tablet orally for 12 days.
Participants received volixibat (SHP626) 5 milligram (mg) capsule orally twice a day (BID) for 12 days.
Participants received volixibat (SHP626) 10 mg capsule orally once a day (QD) for 12 days.
Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12.
Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days.
Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
Overall Number of Participants Analyzed 0 0 0 0 0 0 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame From the start of the study drug administration up to 9 days after the last dose of study drug administration
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Hide Arm/Group Description Participants received placebo matched to volixibat tablet orally for 12 days. Participants received volixibat (SHP626) 5 mg capsule orally BID for 12 days. Participants received volixibat (SHP626) 10 mg capsule QD for 12 days. Participants received volixibat (SHP626) 20 mg capsule orally QD for 12 days. Participants received volixibat (SHP626) 2 mg capsule orally QD on Days 1-3, 5 mg capsule orally QD on Days 4-6, 10 mg capsule QD orally on Days 7-9, and 20 mg capsule QD orally on Days 10-12. Participants received volixibat (SHP626) 30 mg capsule orally QD for 12 days. Participants received volixibat (SHP626) 40 mg capsule orally QD for 12 days. Participants received volixibat (SHP626) 80 mg capsule orally QD on Day 1, 40 mg capsule orally QD on Day 2, and 20 mg capsule orally QD on Days 3-12.
All-Cause Mortality
Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--      --/--      --/--      --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/21 (0.00%)      0/9 (0.00%)      0/9 (0.00%)      0/9 (0.00%)      0/9 (0.00%)      0/9 (0.00%)      0/9 (0.00%)      0/9 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo Volixibat 5 mg BID Volixibat 10 mg QD Volixibat 20 mg QD Volixibat 2-5-10-20 mg QD Volixibat 30 mg QD Volixibat 40 mg QD Volixibat 80-40-20 mg QD
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   13/21 (61.90%)      9/9 (100.00%)      9/9 (100.00%)      9/9 (100.00%)      9/9 (100.00%)      9/9 (100.00%)      9/9 (100.00%)      9/9 (100.00%)    
Cardiac disorders                 
Tachycardia * 1  0/21 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Gastrointestinal disorders                 
Abdominal discomfort * 1  0/21 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Abdominal pain * 1  1/21 (4.76%)  1 0/9 (0.00%)  0 1/9 (11.11%)  1 2/9 (22.22%)  2 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Abdominal pain upper * 1  0/21 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 3/9 (33.33%)  3 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Anorectal discomfort * 1  0/21 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 2/9 (22.22%)  2 1/9 (11.11%)  1 0/9 (0.00%)  0
Defaecation urgency * 1  0/21 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 2/9 (22.22%)  2 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Diarrhoea * 1  11/21 (52.38%)  27 9/9 (100.00%)  33 9/9 (100.00%)  27 9/9 (100.00%)  32 9/9 (100.00%)  26 9/9 (100.00%)  16 9/9 (100.00%)  26 9/9 (100.00%)  22
Dyspepsia * 1  0/21 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0
Gastrointestinal sounds abnormal * 1  0/21 (0.00%)  0 0/9 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0
Haematochezia * 1  0/21 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0
Nausea * 1  0/21 (0.00%)  0 1/9 (11.11%)  1 1/9 (11.11%)  1 1/9 (11.11%)  2 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Proctalgia * 1  0/21 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Vomiting * 1  0/21 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 1/9 (11.11%)  2 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
General disorders                 
Application site irritation * 1  1/21 (4.76%)  1 0/9 (0.00%)  0 1/9 (11.11%)  1 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Pain * 1  0/21 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Pyrexia * 1  0/21 (0.00%)  0 2/9 (22.22%)  2 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Injury, poisoning and procedural complications                 
Thermal burn * 1  0/21 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0
Investigations                 
Hepatic enzyme increased * 1  0/21 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Musculoskeletal and connective tissue disorders                 
Muscle contracture * 1  0/21 (0.00%)  0 0/9 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Nervous system disorders                 
Dizziness postural * 1  0/21 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0
Headache * 1  2/21 (9.52%)  2 1/9 (11.11%)  1 0/9 (0.00%)  0 1/9 (11.11%)  1 1/9 (11.11%)  1 0/9 (0.00%)  0 2/9 (22.22%)  3 0/9 (0.00%)  0
Reproductive system and breast disorders                 
Erection increased * 1  0/21 (0.00%)  0 0/9 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Respiratory, thoracic and mediastinal disorders                 
Dry throat * 1  0/21 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Oropharyngeal pain * 1  0/21 (0.00%)  0 0/9 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Skin and subcutaneous tissue disorders                 
Dermatitis allergic * 1  0/21 (0.00%)  0 0/9 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0
Rash * 1  0/21 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 0/9 (0.00%)  0 1/9 (11.11%)  1 0/9 (0.00%)  0 0/9 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (17.1)
None of the participants in the safety analysis set were included in the pharmacokinetic set because no participant had sufficient and interpretable primary pharmacokinetic data.
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.
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
Results Point of Contact
Name/Title: Study Physician
Organization: Mirum
Phone: 1 650-667-4085
Responsible Party: Mirum Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02287779     History of Changes
Other Study ID Numbers: SHP626-101
First Submitted: November 6, 2014
First Posted: November 11, 2014
Results First Submitted: June 17, 2016
Results First Posted: December 7, 2016
Last Update Posted: March 26, 2019