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Badanie elafibranora u dorosłych Japończyków chorych na pierwotne żółciowe zapalenie dróg żółciowych (PBC) (ELONSEN)

20 października 2026 zaktualizowane przez: Ipsen

Otwarte badanie fazy III prowadzone w jednej grupie, mające na celu ocenę skuteczności i bezpieczeństwa stosowania elafibranoru w dawce 80 mg u dorosłych Japończyków chorych na pierwotne żółciowe zapalenie dróg żółciowych (PBC)

The purpose of this study is to find out about the safety and how well the study intervention (elafibranor) works in participants with PBC. The participants in this study will have confirmed PBC with inadequate response or intolerance to UDCA, which is a medication used in the management and treatment of cholestatic liver disease.

PBC is a slowly progressive disease characterised by damage of the bile ducts in the liver, leading to a build-up of bile acids which causes further damage. The liver damage in PBC may lead to scarring (cirrhosis).

PBC may also be associated with multiple symptoms. Many patients with PBC may require a liver transplant or may die if the disease progresses and a liver transplant is not done. In this study all participants will receive a daily dose of elafibranor (the study intervention).

The main aim of this study is to determine if elafibranor reduces alkaline phosphatase (ALP) and total bilirubin levels. High ALP and bilirubin levels in the blood can indicate liver disease.

There will be 4 periods in this study: A screening period (up to 10 weeks) to assess whether the participant can take part. A treatment period (52 weeks) where all eligible participants will receive elafibranor. A variable treatment extension period (2-5 years) from End Of Treatment (EOT) period up to the commercial availability of elafibranor in Japan. A follow-up period (4 weeks) where participants' health will be monitored.

Participants will undergo blood sampling, urine collections, physical examinations, clinical evaluations, electrocardiograms (ECG: recording of the electrical activity of heart), ultrasound examinations (a non-invasive test that passes a probe over skin to look at the bladder, urinary tract, and liver), and Fibroscan® examinations (to measure stiffness of the liver). They will also be asked to fill in questionnaires. Each participant will be in this study for up to approximately 6 years

Przegląd badań

Status

Aktywny, nie rekrutujący

Warunki

Interwencja / Leczenie

Typ studiów

Interwencyjne

Zapisy (Rzeczywisty)

18

Faza

  • Faza 3

Kontakty i lokalizacje

Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.

Lokalizacje studiów

  • Japonia
      • Chiba, Japonia
        • Nippon Medical School - Chiba Hokusoh Hospital
      • Fukushima, Japonia
        • Fukushima Medical University Hospital
      • Hiroshima, Japonia
        • Chugoku Rosai Hospital
      • Hokkaido, Japonia
        • Teine Keijinkai Hospital
      • Kagawa, Japonia
        • Kagawa University Hospital
      • Kagoshima, Japonia
        • Kagoshima University Hospital
      • Nagano, Japonia
        • Shinshu University Hospital
      • Nagasaki, Japonia
        • National Hospital Organization Nagasaki Medical Center
      • Nara, Japonia
        • Nara Medical University Hospital
      • Niigata, Japonia
        • Niigata University Medical & Dental Hospital
      • Niigata, Japonia
        • Nagaoka Red Cross Hospital
      • Osaka, Japonia
        • National Hospital Organization Osaka National Hospital
      • Shizuoka, Japonia
        • Hamamatsu University Hospital
      • Tokyo, Japonia
        • Juntendo University Hospital
      • Tokyo, Japonia
        • Teikyo University Hospital
      • Tokyo, Japonia
        • Tokyo Metropolitan Komagome Hospital

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

18 lat do 75 lat (Dorosły, Starszy dorosły)

Akceptuje zdrowych ochotników

Nie

Płeć kwalifikująca się do nauki

Wszystko

Opis

Inclusion Criteria:

  • Must have provided written informed consent and agree to comply with the study protocol.
  • Japanese male or female participants aged 18 to 75 years inclusive at Screening Visit 1 (SV1).
  • PBC diagnosis as described in the study protocol
  • ALP ≥1.67×ULN (mean value based on samples collected at SV1 and SV2).
  • TB ≤2×ULN at SV1 and SV2.
  • Must have at least 4 available values for PBC Worst Itch Numeric Rating Scale (NRS) during each of the 7-day intervals in the 14 days prior to visit (V)1, for a total of at least 8 values for PBC Worst Itch NRS in the last 14 days prior to V1.
  • Participants taking UDCA for at least 12 months (stable dose ≥3 months) prior to screening, or unable to tolerate UDCA treatment (no UDCA for ≥3 months) prior to screening (per country standard-of-care dosing).
  • If on colchicine, must be on a stable dose for ≥3 months prior to screening.
  • Medications for management of pruritus (for example, cholestyramine, rifampicin, naltrexone, sertraline or nalfurafine hydrochloride) must be on a stable dose for ≥3 months prior to screening.
  • Participants taking statins or ezetimibe must be on a stable dose for ≥2 months prior to screening.
  • Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies

Exclusion Criteria

  • History or presence of other concomitant liver disease

  • Participants with known cirrhosis who have a Child-Pugh B or C classification.

  • Participants with cirrhosis with Child-Pugh A classification are allowed.

  • History or presence of clinically significant hepatic decompensation,

  • Medical conditions that may cause non-hepatic increases in ALP (for example, Paget's disease) or which may diminish life expectancy to <2 years, including known cancers.

  • Known malignancy or history of malignancy within the last 5 years, with the exception of local, successfully treated basal cell carcinoma or in-situ carcinoma of the uterine cervix.

  • Participant has a positive test for human immunodeficiency virus (HIV) Type 1 or 2 at screening, or participant is known to have tested positive for HIV.

  • Evidence of any other unstable or untreated clinically significant immunological, endocrine, haematologic, gastrointestinal, neurological, or psychiatric disease as evaluated by the investigator; other clinically significant medical conditions that are not well controlled.

  • History of alcohol abuse, defined as consumption of more than 30 g pure alcohol per day for men, and more than 20 g pure alcohol per day for women, or other substance abuse within 1 year prior to SV1.

  • For female participants: known pregnancy, or has a positive serum pregnancy test, or breastfeeding.

  • Administration of the following medications are prohibited as specified below:

    1. 1 month prior to screening: fibrates.
    2. 2 months prior to screening: glitazones.
    3. For participants with previous exposure to obeticholic acid (OCA), OCA should be discontinued 3 months prior to screening.
    4. 3 months prior to screening: azathioprine, cyclosporine (systemic), methotrexate, mycophenolate, pentoxifylline, budesonide and other systemic corticosteroids (parenteral and oral chronic administration only); potentially hepatotoxic drugs (including α-methyldopa, sodium valproate/valproic acid isoniazid, or nitrofurantoin).
    5. 12 months prior to screening: antibodies or immunotherapy directed against interleukins (ILs) or other cytokines or chemokines.

  • Participants who are currently participating in, plan to participate in, or have participated in an investigational drug study or medical device study containing active substance within 30 days or 5 half-lives, whichever is longer, prior to screening; for participants with previous exposure to seladelpar, seladelpar should be discontinued 3 months prior to screening.

  • Participants with previous exposure to elafibranor.

  • SV1 or SV2 value ALT and/or AST >5×ULN.

  • For participants with aminotransferases or TB >ULN at SV1, variability (between SV1 and SV2) of aminotransferases or TB >40%.

  • SV1 value albumin <3.0 g/dL.

  • Severely advanced participants according to Rotterdam criteria (TB >ULN and albumin <LLN).

  • SV1 international normalised ratio (INR) >1.3 due to altered hepatic function.

  • SV1 creatine phosphokinase (CPK) >2×ULN.

  • SV1 serum creatinine >1.5 mg/dL.

  • Significant renal disease, including nephritic syndrome, chronic kidney disease (defined as participants with markers of kidney failure damage or estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m2) calculated by modification of diet in renal disease study (MDRD).

  • SV1 platelet count <150×103/μL.

  • Alpha-fetoprotein (AFP) >20 ng/mL with 4-phase liver computerised tomography (CT) or magnetic resonance imaging (MRI) imaging suggesting presence of liver cancer.

  • Known hypersensitivity to the investigational product or to any of the formulation excipients of the elafibranor tablet.

  • Mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.

Plan studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

  • Główny cel: Leczenie
  • Przydział: Nie dotyczy
  • Model interwencyjny: Zadanie dla jednej grupy
  • Maskowanie: Brak (otwarta etykieta)

Broń i interwencje

Grupa uczestników / Arm
Interwencja / Leczenie
Eksperymentalny: Elafibranor

Participants will take the study intervention by mouth (swallow whole) once daily
Lek: Elafibranor

Tablet of 80 mg

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Ramy czasowe
Odsetek uczestników z fosfatazą alkaliczną (ALP) <1,67x GGN, spadkiem ALP ≥15% i bilirubiną całkowitą (TB) ≤ GGN
Ramy czasowe: W tygodniu 52
W tygodniu 52

Miary wyników drugorzędnych

Miara wyniku
Opis środka
Ramy czasowe
Odsetek uczestników, którzy znormalizowali poziomy ALP
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Zmiana poziomów ALP w stosunku do wartości wyjściowych
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odsetek uczestników z ALP <1,5×GGN, spadkiem ALP ≥40% i gruźlicą ≤ULN.
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odsetek uczestników z ALP <3×GGN, aminotransferazą asparaginianową (AST) <2×GGN i bilirubiną całkowitą (TB) <1 mg/dl (kryteria paryskie I).
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odsetek uczestników z ALP ≤1,5×ULN, AST ≤1,5×ULN i gruźlicą ≤ULN (kryteria paryskie II).
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odsetek uczestników, u których wskaźnik odpowiedzi na gruźlicę wyniósł 15%, zmienił się.
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odsetek uczestników z normalizacją nieprawidłowej gruźlicy i/lub albumin (kryteria rotterdamskie).
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odsetek uczestników z gruźlicą ≤0,6×GGN
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odsetek uczestników z ALP ≤1,67×GGN i gruźlicą ≤1 mg/dl.
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odsetek uczestników, u których nie doszło do pogorszenia poziomu gruźlicy
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odsetek uczestników z ALP ≤1,67×GGN, spadkiem ALP ≥15% i gruźlicą ≤ULN.
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odsetek uczestników z ALP <1,5×GGN, spadkiem ALP ≥15% i gruźlicą ≤ULN.
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odsetek uczestników z ALP ≤ULN i gruźlicą ≤ULN.
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odpowiedź na podstawie normalizacji bilirubiny.
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odpowiedź oparta na normalizacji poziomów albumin.
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Odsetek uczestników, u których świąd nie uległ pogorszeniu w stosunku do wartości wyjściowych
Ramy czasowe: W 26. i 52. tygodniu oraz co 26 tygodni aż do zakończenia badania (do 6 lat)
W 26. i 52. tygodniu oraz co 26 tygodni aż do zakończenia badania (do 6 lat)
Czas do pierwszego wystąpienia każdego z indywidualnie rozstrzygniętych zdarzeń klinicznych
Ramy czasowe: W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
W tygodniu 4, tygodniu 13, tygodniu 26, tygodniu 39, tygodniu 52, tygodniu 78 i co 26 tygodni aż do końca badania (do 6 lat)
Zmiana w stosunku do wartości wyjściowych poziomów glukozy w osoczu na czczo (FPG).
Ramy czasowe: W 52. tygodniu i co 26 tygodni aż do zakończenia badania (do 6 lat)
W 52. tygodniu i co 26 tygodni aż do zakończenia badania (do 6 lat)
Pole pod krzywą zależności stężenia w osoczu od czasu od czasu 0 do 24 godzin: AUC0-24
Ramy czasowe: W tygodniu 4
W tygodniu 4
Maksymalne (szczytowe) stężenie leku w osoczu: Cmax
Ramy czasowe: W tygodniu 4
W tygodniu 4
Czas do osiągnięcia maksymalnego (szczytowego) stężenia w osoczu po podaniu leku): Tmax
Ramy czasowe: W tygodniu 4
W tygodniu 4
Percentage of Participants With ALP Level response
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

Defined as 10%, 20% and 40% ALP reduction from baseline
At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants with complete biochemical response
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

Defined as normal ALP; TB; AST; alanine aminotransferase (ALT); albumin; and INR.
At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in PBC risk scores based in Global PBC Study Group (GLOBE) score
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

The GLOBE score is a validated risk assessment tool providing an estimate of transplant-free survival for patients with PBC. It was developed by the Global PBC Study Group using Cox regression model on over 4,000 patients with PBC. Lower GLOBE score predicts lower risk.
At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in PBC risk scores based on United Kingdom (UK)-PBC score.
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

PBC risk score developed by United Kingdom (UK)-PBC Consortium is a scoring system and the calculation is based on laboratory test measurements and upper limits of normal (ULN) for the total bilirubin (BIL12); alanine transaminase or aspartate transaminase (TA12), and alkaline phosphatase (ALP12) after at least 12 months of UDCA, and the laboratory test measurements and lower limits of normal (LLN) for the serum albumin and platelet count in the same timeframe. A high number is indicative of a worse score.
At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in hepatobiliary injury and liver function
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

Hepatobiliary injury and liver function will be measured by AST, ALT, gamma-glutamyl transferase (GGT), 5'nucleotidase (5' NT), total and conjugated bilirubin, albumin, INR, and ALP fractionated (hepatic).
At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in lipid parameters
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

Lipid parameters as measured by total cholesterol (TC), low-density lipoprotein cholesterol ( LDL-C), high-density lipoprotein cholesterol (HDL-C), calculated very low-density lipoprotein cholesterol (VLDL-C) and triglycerides (TG).
At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in liver stiffness measurement (LSM)
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

Assessed by vibration-controlled transient elastography (VCTE) using Fibroscan®
At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in pruritus
Ramy czasowe: At Week 26, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

Based on PBC Worst Itch Numeric Rating Scale (NRS) score in participants with baseline PBC Worst Itch NRS score ≥4.
At Week 26, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Proportion of responders in PBC Worst Itch NRS according to clinically meaningful change from baseline
Ramy czasowe: At Week 26 and at Week 52 and every 26 weeks until the end of study (up to 6 years)

At least 30% reduction; and one point, two points or three points decrease in score in participants with a baseline NRS score ≥4.
At Week 26 and at Week 52 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in 5D-Itch scale
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

Questionnaire that assesses symptoms in terms of 5 domains: degree, duration, direction, disability and distribution. Participants rate their symptoms over the preceding 2-week period on a 1 to 5 scale, with 5 being the most affected.
At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form 7a scores
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

PROMIS Fatigue Short Form 7a scores consists of 7 items that measure both the experience of fatigue and the interference of fatigue on daily activities over the past week. Response options are on a 5-point Likert scale, ranging from 1 to 5. Scores can range from 7 to 35, with higher scores indicating greater fatigue.
At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in Epworth Sleepiness Scale (ESS).
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

Self-administered questionnaire that consists of 8 questions asking to rate how likely it is to fall asleep in different situations commonly encountered in daily life (each question can be scored from 0 to 3 points; '0' indicates no sleepiness, '3' indicates significant sleepiness). It provides a total score which has been shown to relate to the participant's level of daytime sleepiness (total score range 0-24 points).
At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in PBC-40.
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

40-item questionnaire that assesses symptoms across 6 domains: fatigue, emotional and social, cognitive function, general symptoms and itch. Participants respond on a verbal response scale, depending on the section options range from 'never' / 'not at all' / 'strongly disagree' to 'always' / 'very much' / 'strongly agree'. Six items (3/3 in the itch domain, 2/10 in the social domain, and 1/7 in the general symptoms domain) also include a 'does not apply' option. A score for each domain is provided (but a total score is not calculated), with each verbal response scale correlating to a score of 1 to 5 per item (0 to 5 on items with a 'does not apply' option) with 5 being the most affected (greatest burden).
At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in EuroQol 5-dimensional 5-level questionnaire (EQ-5D-5L)
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

Self-administered standardised questionnaire that assesses the 5-dimensions of mobility, self-care, usual activities, pain/discomfort, anxiety/depression descriptively (each dimension has 5 levels) and the overall health state via an EQ Visual Analogue Scale (VAS).
At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in Patient Global Impression of Severity (PGI-S)
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

A 1-item, 5-point scale designed to assess the participant's impression of change in disease severity since the baseline visit
At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Patient Global Impression of Change (PGI-C)
Ramy czasowe: At Week 4, Week 13, Week 26, Week 39, and Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)]

A 1-item, 5-point scale designed to assess the participant's impression of change in disease severity since the baseline visit
At Week 4, Week 13, Week 26, Week 39, and Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)]
Percentage of participants who experience Treatment Emergent Adverse Events (TEAEs), treatment related TEAEs, Serious Adverse Events (SAEs), and Adverse Events of Special Interest (AESIs).
Ramy czasowe: From baseline to end of study (up to 6 years)

An Adverse Event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. AESIs are AEs that may not be serious but are of special importance to a particular drug or class of drugs.
From baseline to end of study (up to 6 years)
Percentage of participants who develop clinically significant changes from baseline in physical examination findings
Ramy czasowe: From baseline to end of study (up to 6 years)

Clinically significant change in physical examination findings will be reported. The clinical significance will be graded by the investigator.
From baseline to end of study (up to 6 years)
Percentage of participants who develop clinically significant changes from baseline in vital signs
Ramy czasowe: From baseline to end of study (up to 6 years)

Clinically significant change in vital signs will be reported. The clinical significance will be graded by the investigator.
From baseline to end of study (up to 6 years)
Percentage of participants with clinically significant changes in laboratory parameters (blood chemistry, hematology, liver tests, renal tests (including urinalysis).
Ramy czasowe: From baseline to end of study (up to 6 years)

Clinically significant change in laboratory parameters will be reported. The clinical significance will be graded by the investigator.
From baseline to end of study (up to 6 years)
Percentage of participants who develop clinically significant changes from baseline in ECG readings
Ramy czasowe: From baseline to end of study (up to 6 years)

Clinically significant change in ECG readings. The clinical significance will be graded by the investigator.
From baseline to end of study (up to 6 years)
Change from baseline in serum markers of bone turnover and in bone mineral density (hip and lumbar)
Ramy czasowe: At Week 52 and every 26 weeks until the end of study (up to 6 years)

Assessed by dual-energy X-ray absorptiometry (DEXA) scanning.
At Week 52 and every 26 weeks until the end of study (up to 6 years)

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

Ipsen

Śledczy

  • Dyrektor Studium: Ipsen Medical, Director, Ipsen

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów (Rzeczywisty)

16 stycznia 2025

Zakończenie podstawowe (Szacowany)

17 grudnia 2026

Ukończenie studiów (Szacowany)

8 kwietnia 2032

Daty rejestracji na studia

Pierwszy przesłany

3 grudnia 2024

Pierwszy przesłany, który spełnia kryteria kontroli jakości

11 grudnia 2024

Pierwszy wysłany (Rzeczywisty)

12 grudnia 2024

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Rzeczywisty)

23 października 2026

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

20 października 2026

Ostatnia weryfikacja

1 października 2026

Więcej informacji

Terminy związane z tym badaniem

Dodatkowe istotne warunki MeSH

Inne numery identyfikacyjne badania

  • CLIN-60190-462

Plan dla danych uczestnika indywidualnego (IPD)

Planujesz udostępniać dane poszczególnych uczestników (IPD)?

TAK

Opis planu IPD

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.

Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.

Ramy czasowe udostępniania IPD

Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.

Kryteria dostępu do udostępniania IPD

Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).

Informacje o lekach i urządzeniach, dokumenty badawcze

Bada produkt leczniczy regulowany przez amerykańską FDA

Nie

Bada produkt urządzenia regulowany przez amerykańską FDA

Nie

Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .

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