Setanaxib for Primary Biliary Cholangitis

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Royal Prince Alfred Hospital, Camperdown, Australia
Primary Biliary Cholangitis+3 More
Setanaxib - Drug
Eligibility
18+
All Sexes
What conditions do you have?
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Study Summary

This study is evaluating whether a drug called setanaxib can improve liver function in people with primary biliary cholangitis.

See full description

Eligible Conditions

  • Primary Biliary Cholangitis
  • Liver Stiffness

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Primary Biliary Cholangitis

Study Objectives

This trial is evaluating whether Setanaxib will improve 1 primary outcome and 15 secondary outcomes in patients with Primary Biliary Cholangitis. Measurement will happen over the course of Up to Week 52.

Week 52
Change from Baseline in Fatigue
Change from Baseline in Patient's Global Impression of Change (PGIC) Fatigue
Change from Baseline in Patient's Global Impression of Change (PGIC) Pruritus
Change from Baseline in Patient's Global Impression of Severity (PGIS) Fatigue
Change from Baseline in Patient's Global Impression of Severity (PGIS) Pruritus
Change from Baseline in Primary Biliary Cirrhosis (PBC)-40 Fatigue Domain
Change from Baseline in Primary Biliary Cirrhosis (PBC)-40 Itch Domain
Change from Baseline in Worst Itch Numerical Scale Rating Scale (WI-NRS)
Week 52
Change from Baseline in 5-D Itch Scale in Participants with Pruritus at Baseline
Change from Baseline in Liver Stiffness
Change from Baseline in Primary Biliary Cirrhosis (PBC)-40 Itch Domain in Participants with Pruritus at Baseline
Change from Baseline in Pruritis-Visual Analogue Scale (VAS) in Participants with Pruritus at Baseline
Proportion of Participants Achieving a Biochemical Response to Setanaxib
Week 52
Change from Screening in Liver Stiffness
Up to Week 52
Proportion of Participants Who Experience Adverse Events of Special Interest (AESIs)
Proportion of Participants with Treatment-Emergent Adverse Events (TEAEs)

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Primary Biliary Cholangitis

Trial Design

3 Treatment Groups

Setanaxib 1200 mg/day
1 of 3
Setanaxib 1600 mg/day
1 of 3
Placebo
1 of 3
Experimental Treatment
Non-Treatment Group

This trial requires 318 total participants across 3 different treatment groups

This trial involves 3 different treatments. Setanaxib is the primary treatment being studied. Participants will be divided into 2 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 & 3 and have had some early promising results.

Setanaxib 1200 mg/day
Drug
Participants will be administered setanaxib at a dose of 1200 mg/day for the 52-week double-blind treatment period and the 52-week extension period. The interim analysis outcome will determine if the setanaxib dose level for participants receiving 1200 mg/day will be escalated to 1600 mg/day for the continued extension period.
Setanaxib 1600 mg/day
Drug
Participants will be administered setanaxib at a dose of 1600 mg/day for the 52-week double-blind treatment period and the 52-week extension period. The interim analysis outcome will determine if the setanaxib dose level for participants receiving 1600 mg/day will be reduced to 1200 mg/day for the continued extension period.
Placebo
Drug
Participants will be administered a placebo for the 52-week double-blind treatment period. During the 52-week extension period, participants will switch from placebo to setanaxib at a dose of either 1200 or 1600 mg/day depending on interim analysis outcome.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Setanaxib
2020
Completed Phase 1
~70

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: screening (day -28) and week 52
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly screening (day -28) and week 52 for reporting.

Closest Location

University of Utah Hospital - Salt Lake City, UT

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Male or female participant aged ≥18 years, inclusive at the time of informed consent.
Willing and able to give written informed consent and to comply with the requirements of the study.
Documented history of elevated alkaline phsopatase (ALP) levels ≥1.67×upper limit of normal (ULN) of the local reference range.
Documented history of positive antimitochondrial antibodies (AMA) titer or positive PBC-specific antibodies (anti-GP210 or anti-SP100 or antibodies against the major M2 components [PDC-E2, 2-oxo-glutaric acid dehydrogenase complex]).
Historical liver biopsy consistent with PBC.
Serum ALP ≥1.67×ULN at Screening.
Liver stiffness measured by transient elastography (FibroScan®) of ≥8.8 kilopascals (kPa) and an interquartile range over median ratio (IQR/med) of ≤30% at Screening, are taken with the results expressed in kilopascals).
Ursodeoxycholic acid (UDCA) prescriptional dose use for the past 6 months (at a stable dose for >3 months prior to Screening) OR intolerant to UDCA (last dose of UDCA >3 months prior to Screening). Intolerance to UDCA is defined as participants unable to tolerate the full-labelled dose of UDCA in PBC (13-15 mg/kg) due to frequently reported gastrointestinal symptoms such as diarrhea and abdominal pain.
For participants receiving obeticholic acid (OCA), fenofibrate, or bezafibrate treatment for at least 6 months and stable dose for >3 months prior to Screening.
For participants intolerant to OCA, OCA must have been discontinued >3 months prior to Screening.

Patient Q&A Section

What are common treatments for primary biliary cholangitis?

"The most commonly used treatments for primary biliary cholangitis are immunosuppressive drugs such as glucocorticoids, azathioprine, biliary steroid therapy, liver transplantation, and surgical intervention. However, in the last half century, the use of these treatments was significantly curtailed in the United States because of concerns about their long-term consequences and availability of newer, less-toxic and more-effective treatments. The use of immunosuppressive drugs is still a frequent treatment strategy across Europe and Australia. In North America, the use of immunosuppressive treatments is limited or not permitted by law. Immunosuppressive therapy of non-responders is not used." - Anonymous Online Contributor

Unverified Answer

What are the signs of primary biliary cholangitis?

"The main signs of PBC are a chronic inflammation, painless pruritus, and bile duct stricture. All these symptoms were found in the patient's record. PBC is a progressive disease with three grades, which means the disease can become worse. The patient will get more severe symptoms. However, the main purpose of examination and diagnostics of PBC are for the first time to know the disease' condition and find the correct disease' class. We must be able to detect this disease in early stages and take the necessary measures to prevent future relaps. Then make the patient have a proper diet and treatment. The most important part of treatment is to keep the patient hydrated while the patient has an active disease." - Anonymous Online Contributor

Unverified Answer

What is primary biliary cholangitis?

"Primary bile duct cancer is the most devastating complication of PBC and should remain in the differential diagnosis when cirrhosis presents with features of primary sclerosing cholangitis. Primary sclerosing cholangitis can also occur at an earlier stage without the development of advanced liver disease. The prevalence of the autoimmune etiology of primary biliary cholangitis in the United Kingdom is likely to be underestimated." - Anonymous Online Contributor

Unverified Answer

How many people get primary biliary cholangitis a year in the United States?

"Approximately 0.2 per 1000 per year get PBC; however, this is based on people with a [diagnostic] cholangiogram, rather than the true incidence. Results from a recent paper may be an underestimate because people who get a cholangiogram may have been misdiagnosed." - Anonymous Online Contributor

Unverified Answer

Can primary biliary cholangitis be cured?

"PBC is a chronic disease with a poor prognosis. In the past few years, treatment options have improved significantly; however, some symptoms are difficult to manage even with best treatment. For these patients, surgery for organ-related complications is an option to consider." - Anonymous Online Contributor

Unverified Answer

What causes primary biliary cholangitis?

"Withdrawal from the biliary immunosuppressive drugs seems to be the major factor responsible for PBC development in Japan. A retrospective review by the authors has shown that discontinuation of cholangiocarcinogen treatment has also contributed to the development of PBC in other countries. [Intestinal pseudo-obstruction in Japan, (1981) Am J Gastroenterol; 26, (4): 521-3]." - Anonymous Online Contributor

Unverified Answer

What is setanaxib?

"This preliminary summary of the set of studies on setanaxib provided evidence that it is well tolerated and appears to be an efficacious anti-inflammatory agent in patients with moderate to severe primary biliary cirrhosis (PBC). Clinicaltrials.gov number: NCT00334779 [https://clinicaltrialsgov.gov/ct2?NCT00334779]." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for primary biliary cholangitis?

"The majority of patients considered in clinical trials for PBC were younger and had a less symptomatic condition. Patients with a normal bilirubin at time of enrollment were more likely to enter placebo arm than those with a higher baseline grade of inflammation assessed by a bilirubin higher than 15 and/or AST greater than 3 times the ULN." - Anonymous Online Contributor

Unverified Answer

Has setanaxib proven to be more effective than a placebo?

"The combination of 5-FU/oxaliplatin/carmustine versus placebo was associated with progression-free and overall survival. The combination of 5-FU/oxaliplatin/carmustine was shown to be more effective than placebo." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating primary biliary cholangitis?

"Patients with primary biliary cholangitis are as responsive to ursodeoxycholic acid as patients with primary sclerosing cholangitis. In patients with primary biliary cholangitis who can tolerate ursodeoxycholic acid alone or in combination with prednisone, prednisone can be stopped early because there is no loss of response." - Anonymous Online Contributor

Unverified Answer

How serious can primary biliary cholangitis be?

"Despite claims on the internet, in books, and other publications, PBC is not a fatal disease(https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7159816/). However, it is a chronic disease that takes up many years of your life, and it is difficult to treat completely. It is prudent for your provider to discuss the possibility with you when considering a diagnosis." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving setanaxib?

"The phase 3 results of SANT-101 from Sunken and Novo Nordisk showed superior efficacy from setanaxib compared with placebo when it was co-administered with UDCA in patients with primary biliary cholangitis. Thus, clinicians are looking at the possibility of combining both drugs in order to improve efficacy of UDCA treatment." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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