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

Conditions

• Primary Sclerosing Cholangitis

Eligibility Criteria

Sex

ALL

Age

18 Years - 75 Years

Healthy Volunteers

Not accepted

Interventions

• Elafibranor 80 mg — DRUG
  Oral Tablet
• Elafibranor 120 mg — DRUG
  Oral Tablet
• Placebo Matched to Elafibranor 80 mg — DRUG
  Oral Tablet
• Placebo Matched to Elafibranor 120 mg — DRUG
  Oral Tablet

Study Details

This study will evaluate the effects of elafibranor (the study drug) in participants with Primary Sclerosing Cholangitis (PSC) during the double-blind period, an initial 96 week open-label extension (OLE) period, and an optional open-label extension long-term (OLE-LT) period beyond OLE Week 96. 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, including long-term safety during the open-label extension periods. The trial will also study the study drug's effects on blood tests and other tests related to PSC disease activity.

Key Dates

Start date

Dec 29, 2022

Status verified

Jun 2026

Primary completion

Sep 23, 2031

Completion

Sep 23, 2031

Study Design

Enrollment

68 participants (actual)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Double-Blind Period: Elafibranor 80 mg
  Participant will receive two tablets per day (one tablet of elafibranor 80 mg + 1 tablet of placebo matching the 120 mg sized tablet) over the 12 weeks in Double-blind period.
• Experimental: Double-Blind Period: Elafibranor 120 mg
  Participant will receive 2 tablets per day (one tablet of elafibranor 120 mg + 1 tablet of placebo matching the 80 mg sized tablet) over the 12 weeks in Double-blind period.
• Placebo Comparator: Double-Blind Period: Placebo
  Participant will receive 2 placebo tablets per day (one matching the 80 mg sized tablet + one matching the 120 mg sized tablet) over the 12 weeks in Double-blind period.
• Experimental: Initial Open-Label Extension (OLE) Period up to OLE Week 96: Elafibranor 120 mg
  Participant will receive one tablet per day (elafibranor 120 mg) during the initial 96-weeks Open-Label extension period.
• Experimental: Open-Label Extension Long-Term (OLE-LT) Period Beyond OLE Week 96: Elafibranor 120 mg
  Participant will receive one tablet per day (elafibranor 120 mg) during the optional Open-Label Extension Long-Term period beyond OLE Week 96

Primary Outcome Measure

Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Related TEAEs, Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs) [ Time Frame: Double Blind Period: Baseline up to week 12, Initial Open Label Extension (OLE) Period: Baseline up to week 100, Open-Label Extension Long-Term (OLE-LT) Period Beyond OLE Week 96: OLE-LT baseline up to OLE-LT Week 260 ]

Locations (30)

Find similar trials in Lancaster, CA

Related Studies

• Treating Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin
  PHASE1 · Recruiting · Sacramento Pediatric Gastroenterology · Sacramento, California
• MRI Based Biomarkers in Pediatric Autoimmune Liver Disease
  Recruiting · Children's Hospital Medical Center, Cincinnati · Cincinnati, Ohio
• MRI Biomarkers in as Predictor of Clinical Endpoints in Pediatric Autoimmune Liver Disease
  Recruiting · Children's Hospital Medical Center, Cincinnati · Cincinnati, Ohio
• Primary Sclerosing Cholangitis in Children
  Recruiting · Arbor Research Collaborative for Health · Los Angeles, California