Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
NCT04185363 · View on ClinicalTrials.gov ↗
Study Summary
The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
Conditions Studied
Interventions
- DRUG Maralixibat
Study Locations (20)
Other
- Hospital Italiano de Buenos Aires — Buenos Aires
- Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde — Vienna
- Cliniques Universitaires Saint-Luc — Brussels
- Sociedade Beneficente de Senhoras Hospital Sírio-Libanês — São Paulo
- Fundacion Cardioinfantil — Bogotá
- Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon — Lyon
- CHU de Toulouse - Hôpital des Enfants — Toulouse
- Medizinische Hochschule — Hanover
- Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria — Bergamo
Texas
- University of Texas Southwestern Medical Center — Dallas
- University of Texas, Health Science Center San Antonio — San Antonio
California
- Children's Hospital Los Angeles — Los Angeles
District of Columbia
- Medstar Georgetown University Hospital — Washington D.C.
Florida
- Advent Health — Orlando
New York
- Children's Hospital at Montefiore — The Bronx
Ohio
- Cincinnati Children's Hospital — Cincinnati
Pennsylvania
- Children Hospital of Pittsburgh — Pittsburgh
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 90 participants |
| Start Date | 2020-01-08 |
| Est. Completion | 2025-04-23 |
| Phase | Phase 3 |
Interested in This Trial?
Always speak with your doctor before enrolling in a clinical trial.
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04185363
The ClinicalTrials.gov registry entry for NCT04185363 describes a study currently listed as completed. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 90 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Mirum Pharmaceuticals, which has 14 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Progressive Familial Intrahepatic Cholestasis (PFIC) appearing as the primary indexed condition, and to 1 intervention — of which Maralixibat is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04185363 reports 20 study locations spanning 11 distinct geographic areas — top geographies include Other, Texas, California. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04185363 about?
NCT04185363 is a clinical study titled "An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)". The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
What is the current status of trial NCT04185363?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 90 participants. The study started on 2020-01-08. Estimated completion is 2025-04-23.
What conditions does trial NCT04185363 study?
This clinical trial studies the following conditions: Progressive Familial Intrahepatic Cholestasis (PFIC). These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04185363?
The interventions under investigation include: Maralixibat (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04185363?
This trial is sponsored by Mirum Pharmaceuticals, which has 14 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04185363 being conducted?
This trial has 20 study locations across California, District of Columbia, Florida, New York, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How Clinical Trials Work
Understand phases 1-4, trial design, randomization, and the informed consent process.
Patient Rights in Clinical Trials
Your rights as a participant: consent, withdrawal, privacy, and who to contact.
Finding the Right Clinical Trial
A practical guide to searching trials, understanding eligibility, and evaluating options.
All Guides
Browse our complete library of clinical trial educational resources.
Read our methodology — how this data is sourced, computed, and verified.