Trials / Completed
CompletedNCT03566238
This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2
A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1)
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 62 (actual)
- Sponsor
- Albireo · Industry
- Sex
- All
- Age
- 6 Months – 18 Years
- Healthy volunteers
- Not accepted
Summary
Double blind, randomized, placebo controlled, Phase 3 study to investigate the efficacy and safety of low doses and high doses of A4250 compared to placebo in children with progressive familial intrahepatic cholestasis (PFIC) types 1 and 2.
Detailed description
Up to 50 sites in the following countries will take part in this study: Australia, Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Spain, Sweden, Turkey, United Kingdom, United States, and Saudi Arabia
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | A4250 (odevixibat) | A4250 is a small molecule and selective inhibitor of ileal bile acid transporter (IBAT). |
| DRUG | Placebo | Placebo identical in appearance to active drug (A4250). |
Timeline
- Start date
- 2018-05-16
- Primary completion
- 2020-07-27
- Completion
- 2020-07-28
- First posted
- 2018-06-25
- Last updated
- 2025-11-28
- Results posted
- 2021-09-05
Locations
45 sites across 15 countries: United States, Australia, Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Saudi Arabia, Spain, Sweden, Turkey (Türkiye), United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03566238. Inclusion in this directory is not an endorsement.