Odevixibat for PFIC
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests the long-term safety and effectiveness of a new treatment called odevixibat for children with Progressive Familial Intrahepatic Cholestasis (PFIC), a rare liver condition. The medicine aims to manage symptoms such as severe itching and high bile acid levels in the blood. Participants will take capsules daily for 72 weeks. Children diagnosed with PFIC who experience significant itching or flare-ups might be suitable candidates for this study. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.
Do I have to stop taking my current medications for the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, it does exclude patients taking bile acid or lipid binding resins and medications that slow down gut movement.
Is there any evidence suggesting that A4250 (odevixibat) is likely to be safe for humans?
Research has shown that odevixibat, also known as A4250, is generally well tolerated by people with progressive familial intrahepatic cholestasis (PFIC), a rare liver disease. One study found that patients using odevixibat experienced ongoing improvements in their symptoms, with most not encountering serious side effects.
In this study, patients took odevixibat for an extended period, providing insight into its effectiveness in everyday life. Notably, odevixibat is already approved for another condition, offering some reassurance about its safety. Overall, current research supports the safety of odevixibat for people with PFIC, although individual experiences may vary.12345Why do researchers think this study treatment might be promising for PFIC?
Unlike the standard treatments for Progressive Familial Intrahepatic Cholestasis (PFIC), which often involve surgical interventions or liver transplants, odevixibat offers a non-invasive option. Odevixibat is unique because it works by inhibiting the ileal bile acid transporter (IBAT), which reduces bile acid buildup in the liver. This new approach has the potential to alleviate symptoms and improve liver function without the need for surgery, making it an exciting development for both patients and researchers.
What evidence suggests that A4250 might be an effective treatment for PFIC?
Research has shown that odevixibat can help children with progressive familial intrahepatic cholestasis (PFIC), a rare liver disorder. Studies have found it reduces bile buildup in the liver and alleviates itching. Real-world experiences support these findings, demonstrating improvements in children with various types of PFIC. This suggests that odevixibat can ease symptoms and enhance the quality of life for those affected.23567
Who Is on the Research Team?
Ipsen Medical Director
Principal Investigator
Ipsen
Are You a Good Fit for This Trial?
This trial is for children with PFIC, a liver disorder causing severe itching and jaundice. Participants need genetic confirmation of PFIC, have had significant pruritus, weigh at least 5 kg, and can use an eDiary. They must have completed or partially completed a prior A4250 study. Those with episodic PFIC should be experiencing a flare-up.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive A4250 capsules for oral administration once daily
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants continue to receive A4250 to evaluate long-term safety and efficacy
What Are the Treatments Tested in This Trial?
Interventions
- A4250 (odevixibat)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Albireo, an Ipsen Company
Lead Sponsor
Albireo
Lead Sponsor