Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC
Public ClinicalTrials.gov record NCT03659916. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Open-label extension study to evaluate long-term safety and persistence of effect of A4250 in children with progressive familial intrahepatic cholestasis (PFIC).
Study identification
- NCT ID
- NCT03659916
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 116 participants
Conditions and interventions
Eligibility (public fields only)
- Age range
- 0 Months to 100 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 28, 2018 (Actual)
- Primary completion
- Feb 15, 2024 (Actual)
- Completion
- Dec 2, 2025 (Actual)
- Last update posted
- Oct 21, 2026
2018 – 2025
United States locations
- U.S. sites
- 11
- U.S. states
- 9
- U.S. cities
- 10
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | — |
| Children's Hospital Colorado | Denver | Colorado | 80045 | — |
| Emory University School of Medicine | Atlanta | Georgia | 30329 | — |
| Riley Hospital for Children - Riley Children's Specialists | Indianapolis | Indiana | 46202 | — |
| Johns Hopkins School of Medicine | Baltimore | Maryland | 21287 | — |
| Washington University School of Medicine | St Louis | Missouri | 63110 | — |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | — |
| Columbia University Medical Center - Presbyterian Hospital Building | New York | New York | 10032 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
| Baylor College of Medicine - Texas Children's Liver Center | Houston | Texas | 77030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 30 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03659916, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 21, 2026 · Synced Oct 21, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03659916 live on ClinicalTrials.gov.