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Study to Assess Safety & Efficacy of GKT137831 in Patients With Primary Biliary Cholangitis Receiving Ursodiol.

Primary Purpose

Primary Biliary Cirrhosis

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
GKT137831
Placebo oral capsule
Sponsored by
Calliditas Therapeutics AB
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Biliary Cirrhosis

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Male or female aged 18 to 80 years, inclusive.
  2. Willing and able to give written informed consent and to comply with the requirements of the study.
  3. PBC diagnosis as demonstrated by the presence of ≥ 2 of the following 3 diagnostic factors:

    • History of elevated ALP levels (> ULN) for at least 6 months
    • Positive anti-mitochondrial antibody (AMA) titer or if AMA negative or in low titer (< 1:80) PBC-specific antibodies (anti-GP210 and/or anti-SP100 and/or antibodies against the major M2 components [PDC-E2, 2-oxo-glutaric acid dehydrogenase complex])
    • Liver biopsy consistent with PBC (based on historic liver biopsy), including non-suppurative, destructive cholangitis affecting mainly the interlobular and septal bile ducts.
  4. Serum ALP ≥ 1.5 x ULN.
  5. Serum GGT ≥ 1.5 x ULN.
  6. UDCA treatment for at least 6 months and stable dose for at least 3 months prior to Visit 1.
  7. Subjects being treated for pruritus with colestyramine must be on a stable dose of colestyramine for at least 8 weeks prior to baseline/Day 1 (Visit 2). Subjects must be willing and able to take colestyramine at least 2 hours before or after study medication.
  8. Female subjects of childbearing potential must use a highly effective method of contraception to prevent pregnancy for 4 weeks before randomization and must agree to continue strict contraception for 90 days after last administration of investigational medicinal product (IMP). Male participants with female partners of childbearing potential must be willing to use a condom and require their partner to use an additional form of adequate contraception as approved by the Investigator. This requirement begins at the time of informed consent and ends 90 days after the last administration of IMP. Male study participants must also not donate sperm from baseline until 90 days after the last administration of IMP.

Exclusion Criteria:

  1. A positive pregnancy test or breast-feeding for female subjects.
  2. Any hepatic decompensation, defined as a past or current history of hepatic encephalopathy, gastrointestinal tract bleeding due to esophageal varices, or ascites.
  3. International normalized ratio (INR) > 1.2 unless subject is on anticoagulant therapy.
  4. ALT > 3 x ULN.
  5. Total bilirubin > 1 x ULN.
  6. Planned or current plasmapheresis or other extra-corporeal treatments (e.g., molecular adsorbent recirculation system (MARS)) for treatment-refractory pruritus.
  7. History of liver transplantation, current placement on a liver transplant list or current Model for End Stage Liver Disease (MELD) score ≥ 15.
  8. Cirrhosis with complications, including history or presence of: spontaneous bacterial peritonitis, hepatocellular carcinoma.
  9. Hepatorenal syndrome (type I or II) or Screening serum creatinine > ULN.
  10. Competing etiology for liver disease (e.g., hepatitis C, active hepatitis B, non-alcoholic steatohepatitis (NASH), alcoholic liver disease (ALD), autoimmune hepatitis, primary sclerosing cholangitis, Gilbert's Syndrome).
  11. Subjects receiving prohibited medications within 3 months of Screening (Visit 1) according to the list (a, b and c) provided in Section 6.6.2.
  12. Treatment with any investigational agent within 4 weeks of Visit 1 or 5 half-lives of the investigational medicinal product (whichever is longer).
  13. A history of long QT syndrome.
  14. Evidence of any of the following cardiac conduction abnormalities during the screening period:

    • A QTc Fredericia interval >450 milliseconds for males and >470 milliseconds for females.
    • A second or third degree atrioventricular block not successfully treated with a pacemaker.
  15. History of cancer in the preceding 5 years, except adequately treated non-melanoma skin cancer, carcinoma in situ of the cervix, in situ prostate cancer, in situ breast ductal carcinoma, or superficial bladder cancer stage 0).
  16. The occurrence of any acute infection requiring systemic antibiotic therapy within the 2 weeks prior the Screening Visit (Visit 1), or human immunodeficiency virus (HIV) infection.
  17. A history of bone marrow disorder including aplastic anemia, or marked anemia defined as hemoglobin < 10.0 g/dL (or 6.2 mmol/L).
  18. Any condition which, in the opinion of the Investigator, constitutes a risk or contraindication for the participation of the subject in the study, or which could interfere with the study objectives, conduct, or evaluation.

Sites / Locations

  • Mayo Clinic
  • University California Davis
  • Ventura Clinical Trials
  • Yale School of Medicine
  • MedStar Georgetown University Hospital
  • University of Miami
  • Northwestern University
  • Tulane University Medical Center
  • Jackson Liver and GI Specialist c/o (STAR) LLC
  • North Shore University Hospital
  • NYU Hepatology Associates
  • Mount Sinai Health System
  • University of Rochester Medical Centre
  • Dayton Gastroenterology Inc.
  • University Hospitals Cleveland Medical Center
  • UPMC Center for Liver Diseases
  • Methodist University Hospital
  • St Lukes Episcopal Hospital
  • Pinnacle Clinical Research, PLLC
  • Bon Secours Liver Institute of Hampton Roads
  • Bon Secours Liver Institute of Richmond
  • CUB Hôpital Erasme
  • UZ Gent
  • UZ Leuven
  • University of Calgary Liver Unit
  • University of Manitoba
  • Centre hospitalier de l'Universite de Montreal (CHUM) Centre de Recherche Service d'Hepatologie
  • McGill University Health Centre (MUHC)
  • Friedrich-Alexander University Erlangen-Nürnberg
  • Johann Wolfgang Goethe-University
  • Universitatsklinikum Bonn
  • Universitätsklinikum Heidelberg
  • Universitätsmedizin Mainz
  • General Hospital of Athens "Hippocratio"
  • Laiko General Hospital
  • University Hospital of Larissa
  • Rambam Health Centre
  • Shaare Zedek Medical Center
  • Hadassah Medical Organization
  • Rabin Medical Centre
  • Sheba Medical Centre
  • Sourasky Tel-Aviv Medical Center
  • University of Milan-Bicocca
  • Università Politecnica delle Marche - Facoltà di Medicina e Chirurgia
  • Policlinico of Bologna
  • San Giovanni Rotondo Hospital (Puglia)
  • University Hospital Padova
  • Hospital Clinic de Barcelona
  • Hospital Puerta de Hierro-Majadahonda
  • University of Alcalá
  • Virgen De La Victoria University Hospital
  • Hospital Universitario Virgen del Rocio
  • Cambridge University Hospitals NHS Foundation Trust
  • Plymouth Hospital NHS Trust
  • Hull and East Yorkshire Hospitals NHS Trust
  • Gloucestershire Royal Hospital
  • Oxford University Hospitals NHS Foundation Trust
  • Tayside Medical Science Centre (TASC)
  • University Hospitals Birmingham NHS Foundation Trust
  • King's College Hospital NHS Foundation Trust
  • Singleton Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

GKT137831 400mg twice daily

GKT137831 400mg once daily

Placebo Arm

Arm Description

GKT137831 400mg twice daily Patients will self-administer 4 capsules in the morning and 4 capsules in the evening.

GKT137831 400mg once daily Patients will self-administer 4 capsules in the morning and 4 capsules in the evening. The capsules in the evening will be placebos.

Patients will self-administer 4 capsules in the morning and 4 capsules in the evening. All capsules will be placebos.

Outcomes

Primary Outcome Measures

The Percent Change in Serum GGT.
Percent change in serum GGT from baseline to Week 24 (serum GGT was measured in U/L)

Secondary Outcome Measures

Absolute Change in Serum GGT
Absolute change in serum GGT from baseline to each assessment.
Percent Change in Serum GGT
Percent change in serum GGT from baseline to each assessment (serum GGT was measured in U/L)
Percent Change in Serum ALP
Percent change in serum ALP from baseline to each assessment (serum ALP was measured in U/L).
Absolute Change in Serum ALP
Absolute change in serum ALP from baseline to each assessment.
Absolute Change in Serum Conjugated Bilirubin.
Absolute change in serum conjugated bilirubin from baseline to each assessment.
Percent Change in Serum Conjugated Bilirubin.
Percent change in serum Conjugated bilirubin, from baseline to each assessment (serum conjugated bilirubin is measured in μmol/L).
Absolute Change in Serum Total Bilirubin.
Absolute change in serum total bilirubin, from baseline to each assessment.
Percent Change in Serum Total Bilirubin.
Percent change in Serum Total Bilirubin from baseline to each assessment (serum total bilirubin is measured in μmol/L).
Absolute Change in Liver Stiffness as Assessed by Transient Elastography (FibroScan® or Similar Technology).
Absolute change in liver stiffness as assessed by transient elastography (FibroScan® or similar technology), from baseline to Week 24, in subjects with values at baseline and Week 24.
Percent Change in Liver Stiffness as Assessed by Transient Elastography (FibroScan® or Similar Technology).
Percent change in liver stiffness as assessed by transient elastography (FibroScan® or similar technology), from baseline to Week 24, in subjects with values at baseline and Week 24 (liver stiffness is measured in kPa).
Percent Change in Serum Levels of Collagen Fragments Indicative of Collagen Formation and Degradation.
Percent change in serum levels of collagen fragments indicative of collagen formation and degradation, from baseline to Weeks 12 and 24 (serum levels of collagen fragments are measured in ng/mL).
Absolute Change in Liver Stiffness as Assessed by Transient Elastography by Subgroup (FibroScan® or Similar Technology).
Absolute change in liver stiffness by subgroup (>=9.6 kPa) as assessed by transient elastography (FibroScan® or similar technology), from baseline to Week 24, in subjects with values at baseline and Week 24.
Percent Change in Liver Stiffness as Assessed by Transient Elastography by Subgroup (FibroScan® or Similar Technology).
Percent change in liver stiffness by subgroup (>=9.6 kPa) as assessed by transient elastography (FibroScan® or similar technology), from baseline to Week 24, in subjects with values at baseline and Week 24 (liver stiffness is measured in kPa).
Absolute Change in Pruritis Visual Analogue Scale (VAS) Scores
Absolute Change in Pruritis Visual Analogue Scale (VAS) scores from baseline to weeks 12 and 24- Score from 0 to 10, 0= no itch and 10= severe itch, continuous, day and night intolerable itch.
Percent Change in Pruritis Visual Analogue Scale (VAS) Scores
Percent Change in Pruritis Visual Analogue Scale (VAS) scores from baseline to weeks 12 and 24- Score from 0 to 10, 0= no itch and 10= severe itch, continuous, day and night intolerable itch.
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Absolute Change in PBC-40 Domain scores from baseline to weeks 12 and 24 Symptoms domain: items 1 to 7 of the PBC-40 questionnaire (score from 6 to 35). Itch domain: items 8 to 10 of the PBC-40 questionnaire (score from 0 to 15). Fatigue domain: items 11 to 21 of the PBC-40 questionnaire (score from 11 to 55). Cognitive domain: items 22 to 27 of the PBC-40 questionnaire (score from 6 to 30). Emotional domain: items 28, 30 and 33 of the PBC-40 questionnaire (score from 3 to 15). Social domain: items 29, 31, 32, 34 to 40 of the PBC-40 questionnaire (score from 8 to 50). Higher scores mean worse outcome.
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Percent Change in PBC-40 Domain scores from baseline to weeks 12 and 24 Symptoms domain: items 1 to 7 of the PBC-40 questionnaire (score from 6 to 35). Itch domain: items 8 to 10 of the PBC-40 questionnaire (score from 0 to 15). Fatigue domain: items 11 to 21 of the PBC-40 questionnaire (score from 11 to 55). Cognitive domain: items 22 to 27 of the PBC-40 questionnaire (score from 6 to 30). Emotional domain: items 28, 30 and 33 of the PBC-40 questionnaire (score from 3 to 15). Social domain: items 29, 31, 32, 34 to 40 of the PBC-40 questionnaire (score from 8 to 50). Higher scores mean worse outcome.

Full Information

First Posted
June 30, 2017
Last Updated
June 27, 2022
Sponsor
Calliditas Therapeutics AB
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1. Study Identification

Unique Protocol Identification Number
NCT03226067
Brief Title
Study to Assess Safety & Efficacy of GKT137831 in Patients With Primary Biliary Cholangitis Receiving Ursodiol.
Official Title
A Double-Blind, Randomized, Placebo-Controlled Clinical Trial to Assess the Efficacy & Safety of Oral GKT137831 in Patients With Primary Biliary Cholangitis Receiving Ursodeoxycholic Acid and With Persistently Elevated Alkaline Phosphatase
Study Type
Interventional

2. Study Status

Record Verification Date
March 2022
Overall Recruitment Status
Completed
Study Start Date
June 26, 2017 (Actual)
Primary Completion Date
April 11, 2019 (Actual)
Study Completion Date
April 11, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Calliditas Therapeutics AB

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to assess the safety and efficacy of GKT13783 in patients with Primary Biliary Cholangitis (PBC) who are taking a stable dose of ursodeoxycholic acid (UDCA) treatment, and have persistently high levels of a liver enzyme called Alkaline Phosphatase (ALP).
Detailed Description
Primary biliary cholangitis (PBC) is a disease of the liver. It is caused a sustained attack by the body's immune system on the bile ducts (canals) inside the liver. This continuous assault leads to their gradual destruction and eventual disappearance. This results in obstruction to the flow of bile which gets worse with disease progression. Once the bile duct injury has been established, the disease progresses due to ongoing obstruction of bile flow, inflammation and scarring of the liver tissue(fibrosis). The liver eventually fails. This research is looking into whether the study drug is better than a dummy drug when given to patients with PBC. This trial will monitor the patients taking part with regular blood tests and ultrasound liver scans before, during, and at the end of the trial. These measures will allow for the ongoing assessment of liver function, and liver stiffness. It is hoped that in patients in whom the study drug is beneficial, the liver function or stiffness may progress at a slower pace, or may even improve during or at the end of the trial. Liver injury, inflammation and fibrosis Participants will be randomly assigned to 1 of 3 treatment groups (active drug once daily, active drug twice daily or placebo). This is a double blinded study so neither the participants nor the staff responsible for their care will know which group they have been assigned to. During the treatment period, participants will take 4 capsules orally at home in the morning and 4 capsules in the evening for 24 weeks. Participants will be in the trial for 32 weeks in total (about 8 months) and will attend approximately 8 clinic visits.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Biliary Cirrhosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
This will be a double-blind, randomized, placebo-controlled, multicenter, parallel group phase 2 trial. A total of 102 subjects will be randomized and allocated to placebo or one of the 2 active treatment arms, according to a 1:1:1 randomization ratio, stratified at study entry by disease severity defined as baseline serum gamma glutamyl transferase (GGT) < 2.5 x ULN or ≥ 2.5 x ULN). Accordingly, approximately 34 subjects will be allocated to each of the 3 treatment arms.
Masking
ParticipantCare ProviderInvestigator
Masking Description
This is a double-blind study: the Sponsor, subjects, investigator staff, persons performing the assessments and data reviewers and statisticians will remain blinded to the identity of the study treatments.
Allocation
Randomized
Enrollment
111 (Actual)

8. Arms, Groups, and Interventions

Arm Title
GKT137831 400mg twice daily
Arm Type
Experimental
Arm Description
GKT137831 400mg twice daily Patients will self-administer 4 capsules in the morning and 4 capsules in the evening.
Arm Title
GKT137831 400mg once daily
Arm Type
Experimental
Arm Description
GKT137831 400mg once daily Patients will self-administer 4 capsules in the morning and 4 capsules in the evening. The capsules in the evening will be placebos.
Arm Title
Placebo Arm
Arm Type
Placebo Comparator
Arm Description
Patients will self-administer 4 capsules in the morning and 4 capsules in the evening. All capsules will be placebos.
Intervention Type
Drug
Intervention Name(s)
GKT137831
Other Intervention Name(s)
setanaxib
Intervention Description
GKT137831 100mg capsules. To be taken as part of two dose arms which are 400mg twice daily or 400mg once daily.
Intervention Type
Drug
Intervention Name(s)
Placebo oral capsule
Other Intervention Name(s)
Placebo
Intervention Description
Matching capsules.
Primary Outcome Measure Information:
Title
The Percent Change in Serum GGT.
Description
Percent change in serum GGT from baseline to Week 24 (serum GGT was measured in U/L)
Time Frame
Baseline to week 24 (visit 7)
Secondary Outcome Measure Information:
Title
Absolute Change in Serum GGT
Description
Absolute change in serum GGT from baseline to each assessment.
Time Frame
From baseline to Weeks 2, 6, 12, 18 and 24
Title
Percent Change in Serum GGT
Description
Percent change in serum GGT from baseline to each assessment (serum GGT was measured in U/L)
Time Frame
From baseline to Weeks 2, 6, 12, 18 and 24
Title
Percent Change in Serum ALP
Description
Percent change in serum ALP from baseline to each assessment (serum ALP was measured in U/L).
Time Frame
From baseline to Weeks 2, 6, 12, 18 and 24
Title
Absolute Change in Serum ALP
Description
Absolute change in serum ALP from baseline to each assessment.
Time Frame
From baseline to Weeks 2, 6, 12, 18 and 24
Title
Absolute Change in Serum Conjugated Bilirubin.
Description
Absolute change in serum conjugated bilirubin from baseline to each assessment.
Time Frame
From baseline to Weeks 2, 6, 12, 18 and 24
Title
Percent Change in Serum Conjugated Bilirubin.
Description
Percent change in serum Conjugated bilirubin, from baseline to each assessment (serum conjugated bilirubin is measured in μmol/L).
Time Frame
From baseline to Weeks 2, 6, 12, 18 and 24
Title
Absolute Change in Serum Total Bilirubin.
Description
Absolute change in serum total bilirubin, from baseline to each assessment.
Time Frame
from baseline to Weeks 2, 6, 12, 18 and 24
Title
Percent Change in Serum Total Bilirubin.
Description
Percent change in Serum Total Bilirubin from baseline to each assessment (serum total bilirubin is measured in μmol/L).
Time Frame
from baseline to Weeks 2, 6, 12, 18 and 24
Title
Absolute Change in Liver Stiffness as Assessed by Transient Elastography (FibroScan® or Similar Technology).
Description
Absolute change in liver stiffness as assessed by transient elastography (FibroScan® or similar technology), from baseline to Week 24, in subjects with values at baseline and Week 24.
Time Frame
From baseline to Week 24, in patients with values at baseline and Week 24.
Title
Percent Change in Liver Stiffness as Assessed by Transient Elastography (FibroScan® or Similar Technology).
Description
Percent change in liver stiffness as assessed by transient elastography (FibroScan® or similar technology), from baseline to Week 24, in subjects with values at baseline and Week 24 (liver stiffness is measured in kPa).
Time Frame
From baseline to Week 24, in patients with values at baseline and Week 24.
Title
Percent Change in Serum Levels of Collagen Fragments Indicative of Collagen Formation and Degradation.
Description
Percent change in serum levels of collagen fragments indicative of collagen formation and degradation, from baseline to Weeks 12 and 24 (serum levels of collagen fragments are measured in ng/mL).
Time Frame
From baseline to Weeks 12 and 24.
Title
Absolute Change in Liver Stiffness as Assessed by Transient Elastography by Subgroup (FibroScan® or Similar Technology).
Description
Absolute change in liver stiffness by subgroup (>=9.6 kPa) as assessed by transient elastography (FibroScan® or similar technology), from baseline to Week 24, in subjects with values at baseline and Week 24.
Time Frame
From baseline to Week 24, in patients with values at baseline and Week 24.
Title
Percent Change in Liver Stiffness as Assessed by Transient Elastography by Subgroup (FibroScan® or Similar Technology).
Description
Percent change in liver stiffness by subgroup (>=9.6 kPa) as assessed by transient elastography (FibroScan® or similar technology), from baseline to Week 24, in subjects with values at baseline and Week 24 (liver stiffness is measured in kPa).
Time Frame
From baseline to Week 24, in patients with values at baseline and Week 24.
Title
Absolute Change in Pruritis Visual Analogue Scale (VAS) Scores
Description
Absolute Change in Pruritis Visual Analogue Scale (VAS) scores from baseline to weeks 12 and 24- Score from 0 to 10, 0= no itch and 10= severe itch, continuous, day and night intolerable itch.
Time Frame
From baseline to Weeks 12 and 24
Title
Percent Change in Pruritis Visual Analogue Scale (VAS) Scores
Description
Percent Change in Pruritis Visual Analogue Scale (VAS) scores from baseline to weeks 12 and 24- Score from 0 to 10, 0= no itch and 10= severe itch, continuous, day and night intolerable itch.
Time Frame
From baseline to Weeks 12 and 24
Title
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Description
Absolute Change in PBC-40 Domain scores from baseline to weeks 12 and 24 Symptoms domain: items 1 to 7 of the PBC-40 questionnaire (score from 6 to 35). Itch domain: items 8 to 10 of the PBC-40 questionnaire (score from 0 to 15). Fatigue domain: items 11 to 21 of the PBC-40 questionnaire (score from 11 to 55). Cognitive domain: items 22 to 27 of the PBC-40 questionnaire (score from 6 to 30). Emotional domain: items 28, 30 and 33 of the PBC-40 questionnaire (score from 3 to 15). Social domain: items 29, 31, 32, 34 to 40 of the PBC-40 questionnaire (score from 8 to 50). Higher scores mean worse outcome.
Time Frame
From baseline to Weeks 12 and 24
Title
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Description
Percent Change in PBC-40 Domain scores from baseline to weeks 12 and 24 Symptoms domain: items 1 to 7 of the PBC-40 questionnaire (score from 6 to 35). Itch domain: items 8 to 10 of the PBC-40 questionnaire (score from 0 to 15). Fatigue domain: items 11 to 21 of the PBC-40 questionnaire (score from 11 to 55). Cognitive domain: items 22 to 27 of the PBC-40 questionnaire (score from 6 to 30). Emotional domain: items 28, 30 and 33 of the PBC-40 questionnaire (score from 3 to 15). Social domain: items 29, 31, 32, 34 to 40 of the PBC-40 questionnaire (score from 8 to 50). Higher scores mean worse outcome.
Time Frame
From baseline to Weeks 12 and 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or female aged 18 to 80 years, inclusive. Willing and able to give written informed consent and to comply with the requirements of the study. PBC diagnosis as demonstrated by the presence of ≥ 2 of the following 3 diagnostic factors: History of elevated ALP levels (> ULN) for at least 6 months Positive anti-mitochondrial antibody (AMA) titer or if AMA negative or in low titer (< 1:80) PBC-specific antibodies (anti-GP210 and/or anti-SP100 and/or antibodies against the major M2 components [PDC-E2, 2-oxo-glutaric acid dehydrogenase complex]) Liver biopsy consistent with PBC (based on historic liver biopsy), including non-suppurative, destructive cholangitis affecting mainly the interlobular and septal bile ducts. Serum ALP ≥ 1.5 x ULN. Serum GGT ≥ 1.5 x ULN. UDCA treatment for at least 6 months and stable dose for at least 3 months prior to Visit 1. Subjects being treated for pruritus with colestyramine must be on a stable dose of colestyramine for at least 8 weeks prior to baseline/Day 1 (Visit 2). Subjects must be willing and able to take colestyramine at least 2 hours before or after study medication. Female subjects of childbearing potential must use a highly effective method of contraception to prevent pregnancy for 4 weeks before randomization and must agree to continue strict contraception for 90 days after last administration of investigational medicinal product (IMP). Male participants with female partners of childbearing potential must be willing to use a condom and require their partner to use an additional form of adequate contraception as approved by the Investigator. This requirement begins at the time of informed consent and ends 90 days after the last administration of IMP. Male study participants must also not donate sperm from baseline until 90 days after the last administration of IMP. Exclusion Criteria: A positive pregnancy test or breast-feeding for female subjects. Any hepatic decompensation, defined as a past or current history of hepatic encephalopathy, gastrointestinal tract bleeding due to esophageal varices, or ascites. International normalized ratio (INR) > 1.2 unless subject is on anticoagulant therapy. ALT > 3 x ULN. Total bilirubin > 1 x ULN. Planned or current plasmapheresis or other extra-corporeal treatments (e.g., molecular adsorbent recirculation system (MARS)) for treatment-refractory pruritus. History of liver transplantation, current placement on a liver transplant list or current Model for End Stage Liver Disease (MELD) score ≥ 15. Cirrhosis with complications, including history or presence of: spontaneous bacterial peritonitis, hepatocellular carcinoma. Hepatorenal syndrome (type I or II) or Screening serum creatinine > ULN. Competing etiology for liver disease (e.g., hepatitis C, active hepatitis B, non-alcoholic steatohepatitis (NASH), alcoholic liver disease (ALD), autoimmune hepatitis, primary sclerosing cholangitis, Gilbert's Syndrome). Subjects receiving prohibited medications within 3 months of Screening (Visit 1) according to the list (a, b and c) provided in Section 6.6.2. Treatment with any investigational agent within 4 weeks of Visit 1 or 5 half-lives of the investigational medicinal product (whichever is longer). A history of long QT syndrome. Evidence of any of the following cardiac conduction abnormalities during the screening period: A QTc Fredericia interval >450 milliseconds for males and >470 milliseconds for females. A second or third degree atrioventricular block not successfully treated with a pacemaker. History of cancer in the preceding 5 years, except adequately treated non-melanoma skin cancer, carcinoma in situ of the cervix, in situ prostate cancer, in situ breast ductal carcinoma, or superficial bladder cancer stage 0). The occurrence of any acute infection requiring systemic antibiotic therapy within the 2 weeks prior the Screening Visit (Visit 1), or human immunodeficiency virus (HIV) infection. A history of bone marrow disorder including aplastic anemia, or marked anemia defined as hemoglobin < 10.0 g/dL (or 6.2 mmol/L). Any condition which, in the opinion of the Investigator, constitutes a risk or contraindication for the participation of the subject in the study, or which could interfere with the study objectives, conduct, or evaluation.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Philippe Wiesel, MD
Organizational Affiliation
Calliditas Therapeutics AB
Official's Role
Study Director
Facility Information:
Facility Name
Mayo Clinic
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85054
Country
United States
Facility Name
University California Davis
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Ventura Clinical Trials
City
Ventura
State/Province
California
ZIP/Postal Code
93003
Country
United States
Facility Name
Yale School of Medicine
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06520-8019
Country
United States
Facility Name
MedStar Georgetown University Hospital
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Northwestern University
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Tulane University Medical Center
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
Facility Name
Jackson Liver and GI Specialist c/o (STAR) LLC
City
Jackson
State/Province
Mississippi
ZIP/Postal Code
39216
Country
United States
Facility Name
North Shore University Hospital
City
Manhasset
State/Province
New York
ZIP/Postal Code
11030
Country
United States
Facility Name
NYU Hepatology Associates
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Mount Sinai Health System
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
University of Rochester Medical Centre
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
Dayton Gastroenterology Inc.
City
Beavercreek
State/Province
Ohio
ZIP/Postal Code
45440
Country
United States
Facility Name
University Hospitals Cleveland Medical Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
UPMC Center for Liver Diseases
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Methodist University Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38104
Country
United States
Facility Name
St Lukes Episcopal Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Pinnacle Clinical Research, PLLC
City
Live Oak
State/Province
Texas
ZIP/Postal Code
78233
Country
United States
Facility Name
Bon Secours Liver Institute of Hampton Roads
City
Newport News
State/Province
Virginia
ZIP/Postal Code
23602
Country
United States
Facility Name
Bon Secours Liver Institute of Richmond
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23226
Country
United States
Facility Name
CUB Hôpital Erasme
City
Brussels
ZIP/Postal Code
1070
Country
Belgium
Facility Name
UZ Gent
City
Gent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
UZ Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
University of Calgary Liver Unit
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2N4z6
Country
Canada
Facility Name
University of Manitoba
City
Winnipeg
State/Province
Manitoba
ZIP/Postal Code
R3E3P4
Country
Canada
Facility Name
Centre hospitalier de l'Universite de Montreal (CHUM) Centre de Recherche Service d'Hepatologie
City
Montreal,
State/Province
Quebec
ZIP/Postal Code
H2X0A9
Country
Canada
Facility Name
McGill University Health Centre (MUHC)
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H4A3J1
Country
Canada
Facility Name
Friedrich-Alexander University Erlangen-Nürnberg
City
Erlangen
State/Province
Bavaria
ZIP/Postal Code
91054
Country
Germany
Facility Name
Johann Wolfgang Goethe-University
City
Frankfurt
State/Province
Hessen
ZIP/Postal Code
60590
Country
Germany
Facility Name
Universitatsklinikum Bonn
City
Bonn
State/Province
North Rhine-Westphalia
ZIP/Postal Code
53105
Country
Germany
Facility Name
Universitätsklinikum Heidelberg
City
Heidelberg
ZIP/Postal Code
D-69210
Country
Germany
Facility Name
Universitätsmedizin Mainz
City
Mainz
ZIP/Postal Code
D-55131
Country
Germany
Facility Name
General Hospital of Athens "Hippocratio"
City
Athens
ZIP/Postal Code
11527
Country
Greece
Facility Name
Laiko General Hospital
City
Athens
ZIP/Postal Code
11527
Country
Greece
Facility Name
University Hospital of Larissa
City
Larissa
ZIP/Postal Code
41100
Country
Greece
Facility Name
Rambam Health Centre
City
Haifa
ZIP/Postal Code
3109601
Country
Israel
Facility Name
Shaare Zedek Medical Center
City
Jerusalem
ZIP/Postal Code
9103102
Country
Israel
Facility Name
Hadassah Medical Organization
City
Jerusalem
ZIP/Postal Code
91120
Country
Israel
Facility Name
Rabin Medical Centre
City
Petach-Tiqva
ZIP/Postal Code
49100
Country
Israel
Facility Name
Sheba Medical Centre
City
Ramat Gan
ZIP/Postal Code
52621
Country
Israel
Facility Name
Sourasky Tel-Aviv Medical Center
City
Tel Aviv
ZIP/Postal Code
3906
Country
Israel
Facility Name
University of Milan-Bicocca
City
Monza
State/Province
Lombardia
ZIP/Postal Code
20900
Country
Italy
Facility Name
Università Politecnica delle Marche - Facoltà di Medicina e Chirurgia
City
Ancona
ZIP/Postal Code
60126
Country
Italy
Facility Name
Policlinico of Bologna
City
Bologna
ZIP/Postal Code
40138
Country
Italy
Facility Name
San Giovanni Rotondo Hospital (Puglia)
City
Foggia
ZIP/Postal Code
71013
Country
Italy
Facility Name
University Hospital Padova
City
Padova
ZIP/Postal Code
35128
Country
Italy
Facility Name
Hospital Clinic de Barcelona
City
Barcelona
ZIP/Postal Code
8036
Country
Spain
Facility Name
Hospital Puerta de Hierro-Majadahonda
City
Madrid
ZIP/Postal Code
28222
Country
Spain
Facility Name
University of Alcalá
City
Madrid
ZIP/Postal Code
28223
Country
Spain
Facility Name
Virgen De La Victoria University Hospital
City
Malaga
ZIP/Postal Code
29015
Country
Spain
Facility Name
Hospital Universitario Virgen del Rocio
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Facility Name
Cambridge University Hospitals NHS Foundation Trust
City
Cambridge
State/Province
Cambridgeshire
ZIP/Postal Code
CB2 0QQ
Country
United Kingdom
Facility Name
Plymouth Hospital NHS Trust
City
Plymouth
State/Province
Devon
ZIP/Postal Code
PL6 8DH
Country
United Kingdom
Facility Name
Hull and East Yorkshire Hospitals NHS Trust
City
Hull
State/Province
East Yorkshire
ZIP/Postal Code
HU3 2JZ
Country
United Kingdom
Facility Name
Gloucestershire Royal Hospital
City
Gloucester
State/Province
Gloucestershire
ZIP/Postal Code
GL13NN
Country
United Kingdom
Facility Name
Oxford University Hospitals NHS Foundation Trust
City
Oxford
State/Province
Oxfordshire
ZIP/Postal Code
OX3 9DU
Country
United Kingdom
Facility Name
Tayside Medical Science Centre (TASC)
City
Dundee
State/Province
Tayside
ZIP/Postal Code
DD19SY
Country
United Kingdom
Facility Name
University Hospitals Birmingham NHS Foundation Trust
City
Birmingham
ZIP/Postal Code
B152GW
Country
United Kingdom
Facility Name
King's College Hospital NHS Foundation Trust
City
London
ZIP/Postal Code
SE5 9RS
Country
United Kingdom
Facility Name
Singleton Hospital
City
Swansea
ZIP/Postal Code
SA28PP
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Study to Assess Safety & Efficacy of GKT137831 in Patients With Primary Biliary Cholangitis Receiving Ursodiol.

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