Odevixibat for Biliary Atresia

Recruiting at 72 trial locations

Overseen By Ipsen Clinical Study Enquiries

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Albireo, an Ipsen Company

Disqualifiers: Intractable ascites, Ileal resection, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 13

Duration: 104 weeks

254 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether participants must stop taking their current medications. Please consult with the study team for guidance on your specific situation.

Is Odevixibat safe for humans?

Odevixibat has been approved in the EU and USA for treating a liver condition called progressive familial intrahepatic cholestasis (PFIC) in young children, which suggests it has been evaluated for safety in humans. However, specific safety data for its use in biliary atresia is not detailed in the available research. ^{1 2 3 4 5}

How does the drug Odevixibat differ from other treatments for biliary atresia?

Odevixibat is unique because it works by reducing bile acid levels in the intestines, which may help improve liver function in biliary atresia. This mechanism is different from other treatments that primarily focus on surgical interventions or managing symptoms. ^{6 7 8 9 10}

What is the purpose of this trial?

This trial is testing odevixibat, a medication that may help children with biliary atresia who have had surgery. The drug aims to lower bile acid levels to protect the liver.

Research Team

Ipsen Medical Director

Principal Investigator

Ipsen

Eligibility Criteria

This trial is for children with Biliary Atresia who've had a Kasai procedure before they were 90 days old. They should be able to start treatment within 3 weeks post-surgery and weigh more than 3.5kg. Children with severe liver issues, relying solely on IV nutrition, or having certain other conditions are not eligible.

Inclusion Criteria

I have been diagnosed with biliary atresia.

I can start the study treatment within 3 weeks after my Kasai procedure.

I had the Kasai procedure done before I was 3 months old.

Exclusion Criteria

I do not have any major health issues that could affect my safety or participation in the study.

ALT ≥10× upper limit of normal (ULN) at screening

I have severe, untreatable fluid buildup in my abdomen.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either odevixibat or placebo capsules for oral administration once daily

104 weeks

Regular visits for monitoring at Weeks 13, 26, 52, and 104

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Odevixibat
• Placebo

Trial Overview The study tests the effectiveness and safety of Odevixibat versus a placebo in kids after Kasai surgery for Biliary Atresia. It's a Phase 3 trial where participants are randomly assigned to either the drug or placebo without knowing which one they receive.

Participant Groups

Group I: Odevixibat (A4250) Experimental Treatment 1 Intervention

Capsules for oral administration once daily for 104 weeks.

Group II: Placebo Placebo Group 1 Intervention

Capsules for oral administration (to match active) once daily for 104 weeks.

Odevixibat is already approved in United States for the following indications:

Approved in United States as Bylvay for:

• Cholestatic pruritus in infants with Alagille syndrome (ALGS) over 12 months of age
• Pruritus in patients with progressive familial intrahepatic cholestasis (PFIC)

Who Is Running the Clinical Trial?

Albireo, an Ipsen Company

Lead Sponsor

Trials

5

Recruited

640+

Albireo

Lead Sponsor

Trials

17

Recruited

1,200+

Findings from Research

Odevixibat (Bylvay™) is an approved treatment for progressive familial intrahepatic cholestasis (PFIC) in patients as young as 6 months in the EU and 3 months in the USA, highlighting its safety and efficacy for this specific cholestatic disease.

The drug is also being investigated for other cholestatic conditions, such as Alagille syndrome and biliary atresia, indicating its potential broader therapeutic applications.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Odevixibat: First Approval. Deeks, ED.[2022]

Combining ursodeoxycholic acid (UDCA) with bezafibrate significantly improves liver biochemistry markers in patients with primary biliary cholangitis (PBC) who do not respond adequately to UDCA alone, based on a meta-analysis of 10 trials involving 369 patients.

The combination therapy effectively reduces levels of alanine aminotransferase, alkaline phosphatase, and other liver-related markers without increasing the risk of adverse effects compared to UDCA monotherapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness of bezafibrate and ursodeoxycholic acid in patients with primary biliary cholangitis: a meta-analysis of randomized controlled trials. Agrawal, R., Majeed, M., Attar, BM., et al.[2022]

The 0.19 mg fluocinolone acetonide implant was successfully used in a 20-year-old woman with juvenile idiopathic arthritis (JIA) and chronic uveitis, showing significant improvements in visual acuity and reduction in central retinal thickness over a 12-month follow-up period.

This case suggests that the fluocinolone implant can be a safe and effective long-term treatment option for JIA-associated uveitis, particularly for patients who do not respond well to traditional corticosteroids or have compliance issues.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Bilateral 0.19 mg Fluocinolone Acetonide Intravitreal Implant in the Successful Treatment of Juvenile Idiopathic Arthritis-Associated Uveitis and Secondary Macular Oedema: A Case Report and Review of Intravitreal Therapies. Ansari, AS., Amir, Z., Williams, GS.[2021]

In a study of 28 children with juvenile idiopathic arthritis-associated uveitis (JIA-U), adalimumab was found to be well tolerated, and most participants (92%) resumed treatment after initial withdrawal due to active disease.

However, the remission achieved with adalimumab did not last after stopping the medication, indicating that ongoing treatment may be necessary to maintain control of JIA-U.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adalimumab in Juvenile Idiopathic Arthritis-Associated Uveitis: 5-Year Follow-up of the Bristol Participants of the SYCAMORE Trial. Horton, S., Jones, AP., Guly, CM., et al.[2020]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Odevixibat: First Approval. [2022]

2. China pubmed.ncbi.nlm.nih.gov

Adjuvant treatments for biliary atresia. [2020]

3. Netherlands pubmed.ncbi.nlm.nih.gov

Steroids in biliary atresia: single surgeon, single centre, prospective study. [2022]

4. Greece pubmed.ncbi.nlm.nih.gov

Effectiveness of bezafibrate and ursodeoxycholic acid in patients with primary biliary cholangitis: a meta-analysis of randomized controlled trials. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

Social and Educational Outcomes in Patients With Biliary Atresia: A Systematic Review. [2023]

6. England pubmed.ncbi.nlm.nih.gov

Bilateral 0.19 mg Fluocinolone Acetonide Intravitreal Implant in the Successful Treatment of Juvenile Idiopathic Arthritis-Associated Uveitis and Secondary Macular Oedema: A Case Report and Review of Intravitreal Therapies. [2021]

7. Netherlands pubmed.ncbi.nlm.nih.gov

Adalimumab for the treatment of refractory noninfectious paediatric uveitis. [2022]

8. England pubmed.ncbi.nlm.nih.gov

Drug monitoring in long-term treatment with adalimumab for juvenile idiopathic arthritis-associated uveitis. [2020]

9. England pubmed.ncbi.nlm.nih.gov

Adalimumab in juvenile idiopathic arthritis-associated chronic anterior uveitis. [2022]

10. United States pubmed.ncbi.nlm.nih.gov

Adalimumab in Juvenile Idiopathic Arthritis-Associated Uveitis: 5-Year Follow-up of the Bristol Participants of the SYCAMORE Trial. [2020]