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Eine Studie zu Elafibranor bei erwachsenen japanischen Teilnehmern mit primärer biliärer Cholangitis (PBC) (ELONSEN)

20. Oktober 2026 aktualisiert von: Ipsen

Eine offene, einarmige Phase-III-Studie zur Untersuchung der Wirksamkeit und Sicherheit von 80 mg Elafibranor bei erwachsenen japanischen Teilnehmern mit primärer biliärer Cholangitis (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

Studienübersicht

Status

Aktiv, nicht rekrutierend

Bedingungen

Intervention / Behandlung

Studientyp

Interventionell

Einschreibung (Tatsächlich)

18

Phase

  • Phase 3

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienorte

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

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

18 Jahre bis 75 Jahre (Erwachsene, Älterer Erwachsener)

Akzeptiert gesunde Freiwillige

Nein

Studienberechtigte Geschlechter

Alle

Beschreibung

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.

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

  • Hauptzweck: Behandlung
  • Zuteilung: N / A
  • Interventionsmodell: Einzelgruppenzuweisung
  • Maskierung: Keine (Offenes Etikett)

Waffen und Interventionen

Teilnehmergruppe / Arm
Intervention / Behandlung
Experimental: Elafibranor

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

Tablet of 80 mg

Was misst die Studie?

Primäre Ergebnismessungen

Ergebnis Maßnahme
Zeitfenster
Prozentsatz der Teilnehmer mit alkalischer Phosphatase (ALP) <1,67x ULN, ALP-Abnahme ≥15 % und Gesamtbilirubin (TB) ≤ ULN
Zeitfenster: In Woche 52
In Woche 52

Sekundäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Prozentsatz der Teilnehmer, die die ALP-Werte normalisiert haben
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Änderung der ALP-Werte gegenüber dem Ausgangswert
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Prozentsatz der Teilnehmer mit ALP <1,5×ULN, ALP-Abnahme ≥40 % und TB ≤ULN.
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Prozentsatz der Teilnehmer mit ALP <3×ULN, Aspartataminotransferase (AST) <2×ULN und Gesamtbilirubin (TB) <1 mg/dl (Paris-I-Kriterien).
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Prozentsatz der Teilnehmer mit ALP ≤1,5×ULN, AST ≤1,5×ULN und TB ≤ULN (Paris-II-Kriterien).
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Prozentsatz der Teilnehmer mit einer TB-Ansprechrate von 15 % Veränderung.
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Prozentsatz der Teilnehmer mit Normalisierung abnormaler TB und/oder Albumin (Rotterdam-Kriterien).
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Prozentsatz der Teilnehmer mit TB ≤0,6×ULN
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Prozentsatz der Teilnehmer mit ALP ≤ 1,67 × ULN und TB ≤ 1 mg/dl.
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Prozentsatz der Teilnehmer ohne Verschlechterung der TB-Werte
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Prozentsatz der Teilnehmer mit ALP ≤ 1,67 × ULN, ALP-Abnahme ≥ 15 % und TB ≤ ULN.
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Prozentsatz der Teilnehmer mit ALP <1,5×ULN, ALP-Abnahme ≥15 % und TB ≤ULN.
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Prozentsatz der Teilnehmer mit ALP ≤ULN und TB ≤ULN.
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Reaktion basierend auf der Normalisierung von Bilirubin.
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Die Reaktion basiert auf der Normalisierung des Albuminspiegels.
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Anteil der Teilnehmer ohne Verschlechterung des Juckreizes gegenüber dem Ausgangswert
Zeitfenster: In Woche 26 und in Woche 52 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 26 und in Woche 52 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Zeit bis zum ersten Auftreten jedes einzelnen beurteilten klinischen Ergebnisereignisses
Zeitfenster: In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 4, Woche 13, Woche 26, Woche 39, Woche 52, Woche 78 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Änderung des Nüchtern-Plasmaglukosespiegels (FPG) gegenüber dem Ausgangswert
Zeitfenster: In Woche 52 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
In Woche 52 und alle 26 Wochen bis zum Ende des Studiums (bis zu 6 Jahre)
Fläche unter der Plasmakonzentrations-Zeit-Kurve vom Zeitpunkt 0 bis 24 Stunden: AUC0-24
Zeitfenster: In Woche 4
In Woche 4
Maximale (Spitzen-)Plasma-Wirkstoffkonzentration: Cmax
Zeitfenster: In Woche 4
In Woche 4
Zeit bis zum Erreichen der maximalen (Spitzen-)Plasmakonzentration nach Arzneimittelverabreichung): Tmax
Zeitfenster: In Woche 4
In Woche 4
Percentage of Participants With ALP Level response
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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.
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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).
Zeitfenster: 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.
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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).
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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).
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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)

Mitarbeiter und Ermittler

Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.

Sponsor

Ipsen

Ermittler

  • Studienleiter: Ipsen Medical, Director, Ipsen

Studienaufzeichnungsdaten

Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.

Haupttermine studieren

Studienbeginn (Tatsächlich)

16. Januar 2025

Primärer Abschluss (Geschätzt)

17. Dezember 2026

Studienabschluss (Geschätzt)

8. April 2032

Studienanmeldedaten

Zuerst eingereicht

3. Dezember 2024

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

11. Dezember 2024

Zuerst gepostet (Tatsächlich)

12. Dezember 2024

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Tatsächlich)

23. Oktober 2026

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

20. Oktober 2026

Zuletzt verifiziert

1. Oktober 2026

Mehr Informationen

Begriffe im Zusammenhang mit dieser Studie

Zusätzliche relevante MeSH-Bedingungen

Andere Studien-ID-Nummern

  • CLIN-60190-462

Plan für individuelle Teilnehmerdaten (IPD)

Planen Sie, individuelle Teilnehmerdaten (IPD) zu teilen?

JA

Beschreibung des IPD-Plans

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.

IPD-Sharing-Zeitrahmen

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.

IPD-Sharing-Zugriffskriterien

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

Arzneimittel- und Geräteinformationen, Studienunterlagen

Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt

Nein

Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt

Nein

Diese Informationen wurden ohne Änderungen direkt von der Website clinicaltrials.gov abgerufen. Wenn Sie Ihre Studiendaten ändern, entfernen oder aktualisieren möchten, wenden Sie sich bitte an register@clinicaltrials.gov. Sobald eine Änderung auf clinicaltrials.gov implementiert wird, wird diese automatisch auch auf unserer Website aktualisiert .

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