Plain-English translation of NCT03905330 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Liver disease research guide →Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.
This trial tested whether , an oral medication taken twice daily, could safely and effectively treat progressive familial intrahepatic cholestasis (PFIC)—a rare inherited liver disease that causes severe itching and liver damage in children and young adults. Participants were randomly assigned to receive either the medication or a placebo (an inactive look-alike) for 26 weeks. Researchers tracked how the medication affected itching, liver function, and overall safety.
PFIC is a serious inherited condition with limited treatment options, and children with this disease suffer from intense itching that can severely affect their quality of life. This trial was designed to determine whether this medication could offer a safe and effective way to reduce itching and slow liver disease progression.
Participants received either the medication or placebo orally twice daily for 26 weeks. They tracked their itching symptoms daily using a questionnaire and attended regular visits (some conducted remotely) to measure how well the liver was functioning, check for side effects, and assess itching severity. Participants and their caregivers needed to keep detailed records and be available for remote check-ins throughout the study period.
AI-generated summary from trial data · Sep 3, 2026 · Not medical advice
United States