Active — Not RecruitingPhase 2

A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis.

United States68 participantsStarted 2022-12-29

Plain-language summary

This study will evaluate the effects of elafibranor (the study drug) in participants with Primary Sclerosing Cholangitis (PSC). PSC is a rare disease of the liver that leads to injury and destruction of bile ducts. Damage to bile ducts leads to buildup of bile in the liver, which then causes further damage, and leads to disease progression. This study will compare elafibranor to a placebo, a dummy treatment. The main objective of the trial will be to study the safety and side effects of the study drug. The trial will also study the study drug's effects on blood tests and other tests related to PSC disease activity.

Who can participate

Age range18 Years – 75 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria : * Participants with a diagnosis of Primary sclerosing cholangitis (PSC) as demonstrated by the presence of the following, and in the absence of apparent causes of secondary sclerosing cholangitis: i) Historical evidence of an elevated Alkaline phosphatase (ALP) \> Upper Limit Normal (ULN) since at least 6 months prior to SV1. ii) Cholangiogram (e.g. magnetic resonance cholangiopancreatography (MRCP), endoscopic retrograde cholangiopancreatography (ERCP), percutaneous transhepatic cholangiography (PTC) with features compatible with large duct PSC. * ALP ≥1.5x ULN during screening (with variability ≤30% based on two values). * Total bilirubin ≤2.0x ULN at Screening Visit 1(SV1) * Participants taking ursodeoxycholic acid (UDCA) at a total daily dose ≤23 mg/kg/day, with a minimum of 6 months of stable treatment prior to screening period and expected to remain on stable dose through the 12-week DBP. Minimum of 3 months off treatment prior to screening period if UDCA was recently discontinued. * For participants with Inflammatory bowel disease (IBD): i) Participants with Crohn's disease must be in remission based on the investigator's clinical assessment and should be on stable treatment prior to randomisation and during screening. ii) Participants with ulcerative colitis must be in remission or have low activity disease as per the judgement of the investigator and should be on stable treatment prior to randomisation and during screening. iii) Current treatmen…

What they're measuring

Percentage of Participants With Treatment Emergent Adverse Event (TEAEs), Treatment Related TEAEs, Serious Adverse (SAEs) and Adverse Events of Special Interest (AESIs)

Timeframe: Double Blind Period: Baseline up to week 12, Open Label Extension (OLE) Period: Baseline up to week 100

Percentage of Participants With Clinically Significant Changes in Physical Examination Findings

Timeframe: Double Blind Period: Baseline up to week 12, OLE Period: Baseline up to week 100

Percentage of Participants With Clinically Significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)

Timeframe: Double Blind Period: Baseline up to week 12, OLE Period: Baseline up to week 100

Percentage of Participants With Clinically Significant Changes in Vital Signs

Timeframe: Double Blind Period: Baseline up to week 12, OLE Period: Baseline up to week 100

Percentage of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Readings

Timeframe: Double Blind Period: Baseline up to week 12, OLE Period: Baseline up to week 100

Trial details

NCT IDNCT05627362

SponsorIpsen

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-08-28

View on ClinicalTrials.gov More Primary Sclerosing Cholangitis trials