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Primary Biliary CholangitisPhase 2TRANSFORMNCT05014672

A Trial of Setanaxib in Patients With Primary Biliary Cholangitis (PBC) and Liver Stiffness

A Phase 2 study testing Setanaxib in people with Primary Biliary Cholangitis and Liver Stiffness, run by Calliditas Therapeutics Suisse SA at 128 sites in 18 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The primary objective of this study is to evaluate the effect of setanaxib on alkaline phosphatase (ALP) at Week 24 in participants with PBC and with elevated liver stiffness and intolerance or inadequate response to ursodeoxycholic acid (UDCA). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Primary Biliary Cholangitis, Liver Stiffness
What is being tested?
Setanaxib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
76 participants
Where
128 sitesin 18 countries
Study dates
14 Feb 2022to 2 Jul 2024
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.
  • Definite or probable PBC diagnosis as demonstrated by the presence of ≥2 of the following 3 diagnostic factors:
  • Documented history of elevated ALP levels ≥1.67×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.
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  • 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.
  • For participants previously treated with bezafibrate or fenofibrate, and these agents were discontinued prior to screening, they must have been discontinued >3 months prior to Screening.
  • Female participants 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 up to 90 days after the last dose of investigational medicinal product (IMP).
  • For the purposes of this trial, women of childbearing potential are defined as "Fertile, following menarche and until becoming postmenopausal unless permanently sterile. Permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy."
  • Postmenopausal state is defined as no menses for 12 months without an alternative medical cause. In female participants who are not using hormonal contraception or hormonal replacement therapy but with suspected menopause and less than 12 months of amenorrhea, a high follicle stimulating hormone (FSH) level in the postmenopausal range will be required at Screening to confirm a postmenopausal state. Confirmation with more than one FSH measurement is required.
  • Highly effective contraception is defined as methods that can achieve a failure rate of less than 1% per year when used consistently and correctly. These methods are:
  • Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal)
  • Progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable)
  • Intrauterine device
  • Intrauterine hormone-releasing system
  • Bilateral tubal occlusion
  • Vasectomized partner
  • Sexual abstinence (refraining from heterosexual intercourse during the entire period of risk associate with the study treatments). The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the participant. Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method are not acceptable methods of contraception. Female condom and male condom should not be used together.
  • Female participants of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline/Randomization before dosing.
  • Male participants with female partners of childbearing potential must be willing to use a condom and require their partner to use a highly effective contraceptive method. This requirement begins at the time of informed consent and ends 90 days after receiving the last dose of IMP.
  • Male participants must be willing not to donate sperm, and female study participants must be willing not to donate eggs, from Baseline until 90 days after the last dose of IMP.

Who cannot join

  • A positive pregnancy test or breastfeeding for female participants.
  • Any historical or current hepatic decompensation event defined as variceal/portal hypertension bleed and/or hepatic encephalopathy, spontaneous bacterial peritonitis, ascites requiring treatment, or liver transplantation list inclusion.
  • History of liver transplantation, current placement on a liver transplant list or current model for end stage liver disease (MELD) score of ≥12 unless the participant is on anticoagulant therapy, or a Child-Pugh Score of ≥6.
  • Cirrhosis with complications, including history or presence of hepatocellular carcinoma.
  • Total bilirubin >2×ULN. In case of total bilirubin elevation >ULN the Screening serum albumin must be within the reference range.
  • Plasma alanine aminotransferase (ALT) >3×ULN and/or aspartate aminotransferase (AST) >3×ULN.
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  • International normalized ratio (INR) >1.2 unless participant is on anticoagulant therapy. One repeat blood sampling (with analysis by the central laboratory) can be performed at the discretion of the Investigator at Screening Visit 2 for participants who meet this exclusion criterion at Screening Visit 1. If this exclusion criterion is not met at Screening Visit 2, the participant may be eligible for the study.
  • Estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m^2, as calculated by the central laboratory using the chronic kidney disease-epidemiology collaboration (CKD- EPI) equation.
  • Thyroid-stimulating hormone >ULN at Screening. One repeat blood sampling (with analysis by the central laboratory) can be performed at the discretion of the Investigator at Screening Visit 2 for participants who meet this exclusion criterion at Screening Visit 1. If this exclusion criterion is not met at Screening Visit 2, the participant may be eligible for the study.
  • Competing etiology for liver disease (eg, hepatitis C [unless effectively cured of hepatitis C, with a sustained virologic response for at least 6 months prior to Screening], active hepatitis B [HBsAg positive], nonalcoholic steatohepatitis [NASH], alcoholic liver disease, autoimmune hepatitis, autoimmune hepatitis-PBC overlap syndrome, primary sclerosing cholangitis, Gilbert's Syndrome).
  • Medical conditions that could cause nonhepatic increases in ALP (eg, Paget's disease).
  • Known history of human immunodeficiency virus (HIV) infection.
  • Surgery (eg, stomach bypass) or medical condition that might significantly affect absorption of medicines (as judged by the Investigator).
  • Positive urine drug screen (if not due to prescriptional use of a concomitant medication, as confirmed by the Investigator) at Screening. Participants on stable methadone or buprenorphine maintenance treatment for at least 6 months prior to Screening Visit 1 may be included in the study. Medicinal cannabis and cannabidiol products are not exclusionary and may be allowed if the prescription and diagnosis are reviewed and approved by the Investigator.
  • Participants receiving prohibited medications within 3 months of Screening Visit 1.
  • Treatment with any investigational agent within 12 weeks of Screening Visit 1 or 5 half-lives of the IMP (if known) (whichever is longer) or current enrollment in an interventional clinical trial.
  • Evidence of any of the following cardiac conduction abnormalities: A QTc Fridericia interval >450 milliseconds for males or >470 milliseconds for females, as calculated by the central reader. Participants with a second- or third-degree atrioventricular block are to be excluded.
  • History of a malignancy within 5 years of Screening with the following exceptions:
  • Adequately treated carcinoma in situ of the cervix.
  • Adequately treated basal or squamous cell cancer or other localized nonmelanoma skin cancer.
  • The occurrence of any acute infection requiring systemic antibiotic therapy within the 2 weeks prior to Screening Visit 1.
  • A history of bone marrow disorder including aplastic anemia, or any current marked anemia defined as hemoglobin <10.0 g/dL.
  • Prior treatment with setanaxib or participation in a previous setanaxib clinical trial.
  • Unstable cardiovascular disease.
  • Presence of any laboratory abnormality or condition that, in the opinion of the Investigator, could interfere with or compromise a participant's treatment, assessment, or compliance with the protocol and/or study procedures.
  • Any other condition which, in the opinion of the Investigator, constitutes a risk or contraindication for the participation of the participant in the study, or that could interfere with the study objectives, conduct, or evaluation.
  • Hypersensitivity or intolerance to setanaxib or to any of its excipients or placebo compounds.

Trial locations and contacts

128 sites in 18 countries.

128 sites in 18 countries

United States29 sites

  • A. Alfred Taubman Health Care Center

    Ann Arbor, Michigan, 48109

  • University of Cincinnati

    Cincinnati, Ohio, 45267-0595

  • Henry Ford Hospital

    Detroit, Michigan, 48202

  • Northwestern University

    Evanston, Illinois, 60611

  • Liver Specialists of Texas

    Houston, Texas, 77030

  • Pioneer Research Solutions

    Houston, Texas, 77099

  • Gastroenterology Associates - Crystal River

    Inverness, Florida, 34452

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242

  • Summit - Southern Therapy and Advanced Research

    Jackson, Mississippi, 39216

  • Cedars-Sinai Medical Center

    Los Angeles, California, 90048

  • Northwell Health

    Manhasset, New York, 11030

  • University of Miami Leonard M. Miller School of Medicine

    Miami, Florida, 33136

  • Froedtert and Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Vanderbilt Digestive Disease Center

    Nashville, Tennessee, 37232

  • Tulane Medical Center

    New Orleans, Louisiana, 70112

  • New York University Hepatology Associates

    New York, New York, 10016

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

  • AdventHealth Transplant Institute

    Orlando, Florida, 32804

  • Advanced Research Institute, Inc.

    Orlando, Florida, 32825

  • California Liver Research Institute

    Pasadena, California, 91105

  • Einstein Medical Center

    Philadelphia, Pennsylvania, 19141

  • Rapid City Medical Center

    Rapid City, South Dakota, 57701

  • University of California Davis Medical Center

    Sacramento, California, 95817

  • University of Utah Hospital

    Salt Lake City, Utah, 84132

  • Springfield Clinic

    Springfield, Illinois, 62794-9248

  • Tampa General Hospital

    Tampa, Florida, 33606

  • Kansas Medical Clinic - Gastroenterology

    Topeka, Kansas, 66606

  • UA Thomas D. Boyer Liver Institute

    Tucson, Arizona, 85724

  • Wake Forest University Baptist Medical Center

    Winston-Salem, North Carolina, 27157

Other trials in United States

Spain16 sites

  • Hospital General Universitari d'Alicante

    Alicante, 03010

  • Complejo Hospitalario Torrecárdenas

    Almería, 04009

  • Hospital Germans Trias i Pujol

    Badalona, Barcelona, 08916

  • Hospital del Mar - Parc de Salut Mar

    Barcelona, 08003

  • Hospital Clínic de Barcelona

    Barcelona, 08036

  • Hospital Universitario Reina Sofía

    Córdoba, 14004

  • Hospital Universitario de La Princesa

    Madrid, 28006

  • Hospital General Universitario Gregorio Marañón

    Madrid, 28007

  • Hospital Universitario La Paz

    Madrid, 28046

  • Hospital Universitario Virgen de la Victoria

    Málaga, 29010

  • Hospital de Sabadell

    Sabadell, Barcelona, 08208

  • Hospital Universitario de Canarias

    San Cristóbal de La Laguna, Santa Cruz De Tenerife, 38320

  • Hospital Clínico Universitario de Santiago

    Santiago, 15706

  • Hospital Universitario Virgen del Rocío

    Seville, 41011

  • Consorci Hospital General Universitari de València

    Valencia, 46014

  • Hospital Universitario Miguel Servet

    Zaragoza, 50009

Other trials in Spain

France14 sites

  • Centre Hospitalier Universitaire Amiens-Picardie - Site Sud

    Amiens, Picardie, 80054

  • Centre Hosptitalier Universitaire d'Angers

    Angers, Pays de la Loire Region, 49 933

  • Hôpitaux Universitaires Henri Mondor

    Créteil, Île-de-France Region, 94000

  • Centre Hospitalier Universitaire Grenoble Alpes

    Grenoble, Isère, 38043

  • Hôpital Claude Huriez

    Lille, Hauts-de-France, 59037

  • Hôpital Claude Huriez

    Lille, 59037

  • Hopital Dupuytren

    Limoges, Limousin, 87042

  • Hôpital de la Croix Rousse

    Lyon, Auvergne-Rhône-Alpes, 69317

  • Hôpital Saint Joseph Marseille

    Marseille, 13008

  • Hôpital l'Archet

    Nice, 06202

  • Hôpital Saint-Antoine

    Paris, 75012

  • Hôpital Rangueil

    Toulouse, Occitanie, 31059

  • Clinique Pasteur - Toulouse

    Toulouse, Occitanie, 31076

  • Hôpitaux de Brabois

    Vandœuvre-lès-Nancy, Lorraine, 54511

Other trials in France

Australia10 sites

  • Flinders Medical Centre

    Bedford Park, South Australia, 5042

  • Eastern Health - Australia

    Box Hill, Victoria, 3128

  • Royal Prince Alfred Hospital

    Camperdown, New South Wales, 2050

  • Monash Medical Centre

    Clayton, Victoria, 3168

  • Nepean Hospital

    Kingswood, 2747

  • Liverpool Hospital

    Liverpool, 2170

  • The Alfred Hospital

    Melbourne, 3004

  • Fiona Stanley Hospital

    Murdoch, Western Australia, 6150

  • John Hunter Hospital

    New Lambton Heights, New South Wales, 2305

  • Mater Misericordiae - Hospital Brisbane

    South Brisbane, Queensland, 4101

Other trials in Australia

Canada8 sites

  • William Osler Health System - Brampton Civic Hospital

    Brampton, L6R 3J7

  • University of Calgary

    Calgary, Alberta, T2N 4Z6

  • Queen Elizabeth II Health Sciences Centre - Victoria General

    Halifax, Nova Scotia, B3H 3A7

  • St. Joseph's Healthcare Hamilton - Charlton Campus

    Hamilton, Ontario, L8N 4A6

  • Centricity Research (LMC Manna Research) - London

    London, Ontario, N6A 2C2

  • Centre Hospitalier de l'Université de Montréal (CHUM)

    Montreal, Quebec, H2X 3J4

  • Office Of Stephane M. Gauthier

    North Bay, Ontario, P1B 2H3

  • Toronto Liver Center

    Toronto, Ontario, M6H 3M1

Other trials in Canada

Italy8 sites

  • Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona

    Ancona, 60020

  • Azienda Ospedaliera Universitaria Policlinico Gaetano Martino

    Messina, 98124

  • Ospedale San Giuseppe

    Milan, Milano, 20123

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, 20122

  • Azienda Socio Sanitaria Territoriale Monza - Ospedale San Gerardo

    Monza, Monza and Brianza, 20900

  • Università degli Studi di Napoli Federico II

    Naples, 80131

  • Azienda Ospedaliero-Universitaria Maggiore della Carità di Novara

    Novara, 28100

  • Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Istituto Clinico Humanitas

    Rozzano, Milan, 20089

Other trials in Italy

Israel7 sites

  • Soroka Medical Center

    Beersheba, Southern District, 84101

  • Carmel Medical Center

    Haifa, Haifa District, 3436212

  • Rambam Health Care Campus

    Haifa, 3109601

  • Hadassah University Hospital Ein Kerem

    Jerusalem, 9112001

  • Western Galilee Hospital-Nahariya

    Nahariya, Northern District, 22100

  • Rabin Medical Center - Beilinson Hospital

    Petah Tikva, Tel Aviv, 4941492

  • Tel Aviv Sourasky Medical Center

    Tel Aviv, 64239

Other trials in Israel

United Kingdom7 sites

  • NHS Greater Glasgow and Clyde

    Glasgow, Scotland, G4 0SF

  • Gloucestershire Hospitals NHS Foundation Trust

    Gloucester, England, GL1 3NN

  • King's College Hospital NHS Foundation Trust

    London, England, SE5 9RS

  • The Newcastle Upon Tyne Hospitals NHS Foundation Trust

    Newcastle upon Tyne, England, NE7 7DN

  • Nottingham University Hospitals NHS Trust

    Nottingham, England, NG7 2UH

  • Sheffield Teaching Hospitals NHS Foundation Trust

    Sheffield, England, S10 2JF

  • University Hospital Southampton NHS Foundation Trust

    Southampton, England, SO16 6YD

Other trials in United Kingdom

Germany6 sites

  • Universitätsklinikum Frankfurt

    Frankfurt am Main, Hesse, 60590

  • Medizinische Hochschule Hannover

    Hanover, Lower Saxony, 30625

  • Universitätsklinikum des Saarlandes

    Homburg, 66421

  • Eugastro

    Leipzig, Saxony, 04103

  • Klinikum rechts der Isar der Technischen Universität München

    Munich, Bavaria, 81675

  • St. Josefs-Hospital Wiesbaden

    Wiesbaden, Hesse, 65189

Other trials in Germany

Austria4 sites

  • Universitaetsklinikum Graz - Universitätsklinik für Innere Medizin

    Graz, Styria, 8036

  • Medizinische Universität Innsbruck

    Innsbruck, Tyrol, 6020

  • Ordensklinikum Linz GmbH Barmherzige Schwestern

    Linz, Upper Austria, 4010

  • Klinikum Wels-Grieskirchen

    Wels, Upper Austria, 4600

Other trials in Austria

Belgium4 sites

  • Hôpital Erasme

    Brussels, Brussels Capital, 1070

  • Universitair Ziekenhuis Gent

    Ghent, Oost-Vlaanderen, 9000

  • Centre Hospitalier Universitaire Brugmann - Site Victor Horta

    Laken, Brussels Capital, 1020

  • Universitair Ziekenhuis Leuven - Campus Gasthuisberg

    Leuven, Flemish Brabant, 3000

Other trials in Belgium

New Zealand4 sites

  • Wellington Regional Hospital

    Crofton Downs, Wellington Region, 6021

  • Dunedin Hospital

    Dunedin, 9016

  • Auckland City Hospital

    Grafton, Auckland, 1023

  • Waikato Hospital

    Hamilton, 3240

Other trials in New Zealand

Poland3 sites

  • Szpital Specjalistyczny Nr 1 w Bytomiu

    Bytom, 41-902

  • ID Clinic

    Mysłowice, 41-400

  • Centrum Badań Klinicznych Piotr Napora Lekarze Sp. p.

    Wroclaw, 51-162

Other trials in Poland

Czechia2 sites

  • Hepato-Gastroenterologie HK

    Hradec Králové, 500 12

  • Ústřední Vojenská Nemocnice Praha

    Prague, Prague, 169 02

Other trials in Czechia

Greece2 sites

  • University General Hospital of Heraklion (PAGNI)

    Heraklion, 71110

  • University General Hospital of Larissa

    Larissa, 41110

Other trials in Greece

Switzerland2 sites

  • Fondazione Epatocentro Ticino

    Lugano, Canton Ticino, 6900

  • Kantonsspital Sankt Gallen

    Sankt Gallen, 9007

Other trials in Switzerland

Hungary1 site

  • Semmelweis Egyetem - I. Sz. Sebészeti és Intervenciós Gasztroenterológiai Klinika

    Budapest, 1082

Other trials in Hungary

Sweden1 site

  • Akademiska Sjukhuset - Uppsala

    Uppsala, 751 85

Other trials in Sweden

About this study

Official title: TRANSFORM: A 24-week, Randomized, Placebo-controlled, Double-blind, Phase 2b Trial of Setanaxib in Patients With Primary Biliary Cholangitis (PBC) and Elevated Liver Stiffness

The primary objective of this study is to evaluate the effect of setanaxib on alkaline phosphatase (ALP) at Week 24 in participants with PBC and with elevated liver stiffness and intolerance or inadequate response to ursodeoxycholic acid (UDCA).

Being tested
Setanaxib, Placebo
Keywords
Setanaxib, Primary Biliary Cholangitis, Elevated Liver Stiffness
Medical subject headings
Liver Cirrhosis, Biliary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • SetanaxibDrug

    Oral tablets, 400mg per tablet

  • PlaceboDrug

    Oral tablets

Study groups in NCT05014672
Study groupRoleWhat they receive
Setanaxib 1200 mg/dayExperimentalParticipants will be administered setanaxib at a dose of 1200 mg/day for the 24-week double-blind treatment period.
Receives: Drug: Setanaxib
Setanaxib 1600 mg/dayExperimentalParticipants will be administered setanaxib at a dose of 1600 mg/day for the 24-week double-blind treatment period.
Receives: Drug: Setanaxib
PlaceboPlacebo comparatorParticipants will be administered a placebo for the 24-week double-blind treatment period.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in ALP at Week 24 Compared to Baseline

    Change in ALP at Week 24 Compared to Baseline, ratio of week 24 value to baseline value.

    Measured: Baseline (Day 1) and Week 24

Secondary outcomes (14)

  1. Change in Fatigue at Week 24 Compared to Baseline, as Assessed by the PROMIS Short Form-Fatigue 7b Daily

    The Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form 7b measures the severity of fatigue since waking. There are 7 questions, each scored between 1 and 5, with a total score between 7 to 35. A higher score indicates worse fatigue. This raw score is converted to a T score (a standardized score with a mean of 50 and a SD of 10).

    Measured: Baseline (Day 1) and Week 24

  2. Change in Fatigue at Week 24 Compared to Baseline, as Assessed by the Patient's Global Impression of Severity (PGIS) Fatigue

    The Patient's Global Impression of Severity (PGIS)-Fatigue is a global index where subjects are asked to rate the severity of fatigue on a 5-point scale within the past 7 days, with choices of 1=None, 2=Mild, 3=Moderate, 4=Severe and 5=Very Severe. Higher scores indicate a worse assessment for fatigue.

    Measured: Baseline (Day 1) and Week 24

  3. Change in Fatigue at Week 24 Compared to Baseline, as Assessed by the Patient's Global Impression of Change (PGIC) Fatigue

    The Patient's Global Impression of Change (PGIC)-Fatigue is a 7-point scale reflecting a subject's rating of overall improvement where subjects are asked to rate their overall improvement with fatigue with 1=Very much improved, 2=Much improved, 3=Minimally improved, 4=No change, 5=Worse, 6=Much worse, 7=Very much worse. Higher scores indicate a worse outcome for change in fatigue.

    Measured: Week 24

  4. Change in Fatigue at Week 24 Compared to Baseline, as Assessed by the PBC-40 Questionnaire (PBC 40) Fatigue Domain

    PBC-40 fatigue domain includes 11 item questions, items scores range from 1 to 5. The individual item scores are summed to obtain a total domain score (Range for fatigue domain: 11 to 55), high scores represent high impact and low scores indicate low impact of PBC on the quality of life.

    Measured: Baseline (Day 1) and Week 24

  5. Change in Liver Stiffness at Week 24 Compared to Screening

    Change in liver stiffness at Week 24 compared to Screening, as assessed by transient elastography (FibroScan®)

    Measured: Screening (Day -28) and Week 24

  6. Change in Pruritus at Week 24 Compared to Baseline, as Assessed by the Worst Itch Numerical Rating Scale (WI-NRS)

    WI-NRS is a daily patient-reported measure of itch intensity using an 11-point scale where 0=no itch and 10=worst itching imaginable. The WI-NRS score is calculated by averaging the daily WI-NRS scores before the visit date (inclusive). Higher scores indicate worse functioning for pruritus on the WI-NRS.

    Measured: Baseline (Day 1) and Week 24

  7. Change in Pruritus at Week 24 Compared to Baseline, as Assessed by the PBC-40 Itch Domain

    Pruritus was assessed by answering 3 questions on the PBC-40 Itch domain from 1 to 5, which was also summed to obtain a total domain score (range 3 to 15). A high score represents a high impact, and a low score indicates low impact of pruritus on the quality of life.

    Measured: Baseline (Day 1) and Week 24

  8. Change in Pruritus at Week 24 Compared to Baseline, as Assessed by the PGIS Pruritus

    PGIS-Pruritus is a global index where subjects are asked to rate the severity of pruritus on a 5-point scale within the past 7 days, with choices of 1=None, 2=Mild, 3=Moderate, 4=Severe and 5=Very Severe. Higher scores indicate a worse assessment for pruritus.

    Measured: Baseline (Day 1) and Week 24

  9. Change in Pruritus at Week 24 Compared to Baseline, as Assessed by the PGIC Pruritus

    The PGIC-Pruritus is a 7-point scale reflecting a subject's rating of overall improvement where subjects are asked to rate their overall improvement with fatigue with 1=Very much improved, 2=Much improved, 3=Minimally improved, 4=No change, 5=Worse, 6=Much worse, 7=Very much worse. Higher scores indicate a worse outcome for change in pruritus.

    Measured: Week 24

  10. Changes in Markers of Cholestasis at Week 24

    Changes in markers of cholestasis as assessed by proportion of patients at Week 24 with:

    • ALP reduction to <1.67×ULN and total bilirubin ≤1×ULN and a ≥15% or ≥30% or ≥40% or ≥70% ALP reduction from Baseline, respectively
    • ALP reduction to <1.5×ULN and total bilirubin ≤1×ULN and a ≥40% ALP reduction from Baseline
    • ALP <1×ULN and total bilirubin ≤1×ULN
    • Total bilirubin <0.6×ULN

    Measured: Baseline (Day 1) and Week 24

  11. Change in ALP at Week 24 Compared to Baseline, Where Setanaxib Doses Are Combined

    Change in ALP at Week 24 Compared to Baseline, ratio of week 24 value to baseline value. Setanaxib doses are combined and the change from baseline is estimated using a mixed model-repeated measures approach with fixed factors for treatment, visit, log(baseline), treatment*visit, and log(baseline)*visit, subject is included as a random effect. The variance-covariance matrix is therefore not the same as in the three arm analysis, which causes treatment effects estimated in the placebo to group differ from those estimated in the analysis with three treatment arms.

    Measured: Baseline (Day 1) and Week 24

  12. Change in Fatigue at Week 24 Compared to Baseline, as Assessed by the PROMIS Short Form-Fatigue 7b Daily, Where Setanaxib Doses Are Combined

    The Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form 7b measures the severity of fatigue since waking. There are 7 questions, each scored between 1 and 5, with a total score between 7 to 35. A higher score indicates worse fatigue. This raw score is converted to a T score (a standardized score with a mean of 50 and a SD of 10).

    Setanaxib doses are combined and the change from baseline is estimated using a mixed model-repeated measures approach with fixed factors for treatment, visit, log(baseline), treatment*visit, and log(baseline)*visit, subject is included as a random effect. The variance-covariance matrix is therefore not the same as in the three arm analysis, which causes treatment effects estimated in the placebo to group differ from those estimated in the analysis with three treatment arms.

    Measured: Baseline (Day 1) and Week 24

  13. Change in Fatigue at Week 24 Compared to Baseline, as Assessed by the PBC-40 Questionnaire (PBC 40) Fatigue Domain, Where Setanaxib Doses Are Combined

    PBC-40 fatigue domain includes 11 item questions, items scores range from 1 to 5. The individual item scores are summed to obtain a total domain score (Range for fatigue domain: 11 to 55), high scores represent high impact and low scores indicate low impact of PBC on the quality of life.

    Setanaxib doses are combined and the change from baseline is estimated using a mixed model-repeated measures approach with fixed factors for treatment, visit, log(baseline), treatment*visit, and log(baseline)*visit, subject is included as a random effect. The variance-covariance matrix is therefore not the same as in the three arm analysis, which causes treatment effects estimated in the placebo to group differ from those estimated in the analysis with three treatment arms.

    Measured: Baseline (Day 1) and Week 24

  14. Change in Liver Stiffness at Week 24 Compared to Screening, Where Setanaxib Doses Are Combined

    Change in liver stiffness at Week 24 compared to Screening, as assessed by transient elastography (FibroScan®). Setanaxib doses are combined and the change from baseline is estimated using a mixed model-repeated measures approach with fixed factors for treatment, visit, log(baseline), treatment*visit, and log(baseline)*visit, subject is included as a random effect. The variance-covariance matrix is therefore not the same as in the three arm analysis, which causes treatment effects estimated in the placebo to group differ from those estimated in the analysis with three treatment arms.

    Measured: Screening (Day -28) and Week 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Aug 2021
  2. Study start14 Feb 2022
  3. Primary completion31 May 2024
  4. Study completion2 Jul 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Placebo-controlled 1:1:1 in double blind treatment period.
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
76 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05014672
Sponsor's protocol ID
GSN000350
Other IDs
2021-001810-13
Acronym
TRANSFORM
Status last verified
Apr 2025
First submitted
10 Aug 2021
Last updated
24 Apr 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Setanaxib in people with Primary Biliary Cholangitis and Liver Stiffness, run by Calliditas Therapeutics Suisse SA at 128 sites in 18 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 26 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 24 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 128 sites in United States, Spain, France, Australia, Canada and 13 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 24 Apr 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in ALP at Week 24 Compared to Baseline (time frame: Baseline (Day 1) and Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05014672.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2025.

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