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Uno studio sull'elafibranor in partecipanti giapponesi adulti affetti da colangite biliare primitiva (PBC) (ELONSEN)

20 ottobre 2026 aggiornato da: Ipsen

Uno studio di Fase III, in aperto, a braccio singolo per indagare l'efficacia e la sicurezza di Elafibranor 80 mg in partecipanti adulti giapponesi affetti da colangite biliare primitiva (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

Panoramica dello studio

Stato

Attivo, non reclutante

Condizioni

Intervento / Trattamento

Tipo di studio

Interventistico

Iscrizione (Effettivo)

18

Fase

  • Fase 3

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Luoghi di studio

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

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

Da 18 anni a 75 anni (Adulto, Adulto più anziano)

Accetta volontari sani

No

Sessi ammissibili allo studio

Tutto

Descrizione

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.

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: N / A
  • Modello interventistico: Assegnazione di gruppo singolo
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: Elafibranor

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

Tablet of 80 mg

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Lasso di tempo
Percentuale di partecipanti con fosfatasi alcalina (ALP) <1,67 volte ULN, diminuzione dell'ALP ≥ 15% e bilirubina totale (TB) ≤ ULN
Lasso di tempo: Alla settimana 52
Alla settimana 52

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Percentuale di partecipanti che hanno normalizzato i livelli ALP
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Variazione rispetto al basale nei livelli ALP
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Percentuale di partecipanti con ALP <1,5×ULN, diminuzione dell'ALP ≥40% e tubercolosi ≤ULN.
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Percentuale di partecipanti con ALP <3×ULN, aspartato aminotransferasi (AST) <2×ULN e bilirubina totale (TB) <1 mg/dL (criteri di Parigi I).
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Percentuale di partecipanti con ALP ≤1,5×ULN, AST ≤1,5×ULN e TB ≤ULN (criteri di Parigi II).
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
La percentuale di partecipanti con un tasso di risposta alla tubercolosi del 15% cambia.
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Percentuale di partecipanti con normalizzazione della tubercolosi anormale e/o dell'albumina (criteri di Rotterdam).
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Percentuale di partecipanti con tubercolosi ≤0,6×ULN
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Percentuale di partecipanti con ALP ≤1,67×ULN e tubercolosi ≤1 mg/dl.
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Percentuale di partecipanti senza peggioramento dei livelli di tubercolosi
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Percentuale di partecipanti con ALP ≤1,67×ULN, diminuzione dell'ALP ≥15% e TB ≤ULN.
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Percentuale di partecipanti con ALP <1,5×ULN, diminuzione dell'ALP ≥15% e tubercolosi ≤ULN.
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Percentuale di partecipanti con ALP ≤ULN e TB ≤ULN.
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Risposta basata sulla normalizzazione della bilirubina.
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Risposta basata sulla normalizzazione dei livelli di albumina.
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Proporzione di partecipanti senza peggioramento del prurito rispetto al basale
Lasso di tempo: Alla settimana 26 e alla settimana 52 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 26 e alla settimana 52 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Tempo fino al primo verificarsi di ciascuno dei singoli eventi di esito clinico aggiudicati
Lasso di tempo: Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 4, settimana 13, settimana 26, settimana 39, settimana 52, settimana 78 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Variazione rispetto al basale dei livelli di glucosio plasmatico a digiuno (FPG).
Lasso di tempo: Alla settimana 52 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Alla settimana 52 e ogni 26 settimane fino alla fine dello studio (fino a 6 anni)
Area sotto la curva concentrazione plasmatica-tempo dal tempo 0 alle 24 ore: AUC0-24
Lasso di tempo: Alla settimana 4
Alla settimana 4
Concentrazione plasmatica massima (picco) del farmaco: Cmax
Lasso di tempo: Alla settimana 4
Alla settimana 4
Tempo per raggiungere la concentrazione plasmatica massima (picco) dopo la somministrazione del farmaco): Tmax
Lasso di tempo: Alla settimana 4
Alla settimana 4
Percentage of Participants With ALP Level response
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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.
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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).
Lasso di tempo: 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.
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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).
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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).
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Ipsen

Investigatori

  • Direttore dello studio: Ipsen Medical, Director, Ipsen

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Effettivo)

16 gennaio 2025

Completamento primario (Stimato)

17 dicembre 2026

Completamento dello studio (Stimato)

8 aprile 2032

Date di iscrizione allo studio

Primo inviato

3 dicembre 2024

Primo inviato che soddisfa i criteri di controllo qualità

11 dicembre 2024

Primo Inserito (Effettivo)

12 dicembre 2024

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

23 ottobre 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

20 ottobre 2026

Ultimo verificato

1 ottobre 2026

Maggiori informazioni

Termini relativi a questo studio

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

  • CLIN-60190-462

Piano per i dati dei singoli partecipanti (IPD)

Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?

SÌ

Descrizione del piano 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.

Periodo di condivisione 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.

Criteri di accesso alla condivisione 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/).

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

No

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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