Odevixibat for Biliary Atresia

(BOLD-EXT Trial)

Recruiting at 30 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Albireo, an Ipsen Company

Disqualifiers: Non-compliance, Hypersensitivity, Liver transplant, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 104 weeks

180 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a medication called odevixibat in children with a liver condition called Biliary Atresia. The medication aims to lower bile acids in the liver and intestines to help protect the liver and improve symptoms. Children who completed a previous related study can join this study to see if the medication is safe and effective over time.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is Odevixibat safe for humans?

Odevixibat has been studied in children with progressive familial intrahepatic cholestasis (PFIC) and was found to be safe, with no adverse drug events reported in the studies. It is approved for use in the EU and USA for certain conditions, indicating a recognized safety profile. ^{1 2 3 4 5}

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

Odevixibat is unique because it is an oral drug that works by inhibiting the ileal bile acid transporter, reducing bile acid reuptake in the intestines, which is a novel approach compared to the primarily surgical treatments for biliary atresia. This mechanism is different from traditional treatments like surgery or liver transplantation, offering a non-invasive option for managing bile acid levels. ^{1 2 3 4 5}

Eligibility Criteria

This trial is for children with Biliary Atresia who completed a previous 104-week study of Odevixibat. They must have consent from a caregiver and cannot join if they're allergic to Odevixibat, need an imminent liver transplant, were non-compliant in the prior study, or have conditions that may risk their safety or study participation.

Inclusion Criteria

Signed informed consent by caregiver

Completion of the 104-week Treatment Period of Study A4250-011

Exclusion Criteria

Any conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study

Patients who were not compliant with study drug treatment or procedures in Study A4250-011 as per the investigator's discretion

You are in need of a liver transplant or are scheduled to receive one soon according to the investigator's judgement.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive odevixibat for 104 weeks to evaluate long-term efficacy and safety

104 weeks

Regular visits as per study protocol

Safety Follow-up

Participants are monitored for safety after the treatment period

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

Long-term

Treatment Details

Interventions

• Odevixibat

Trial Overview The trial tests the long-term effects and safety of Odevixibat on children with Biliary Atresia. It's an extension of a prior study where participants continue to receive this medication under open-label conditions, meaning both researchers and participants know what treatment is being given.

Participant Groups

Group I: Odevixibat (A4250) Experimental Treatment 1 Intervention

Capsules for oral administration 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]

Odevixibat is a novel treatment for cholestatic diseases like progressive familial intrahepatic cholestasis (PFIC), approved in the EU and USA in 2021, that works by inhibiting the ileal bile acid transporter, leading to significant reductions in bile acid levels in the body.

In clinical studies, a daily dose of 3 mg of odevixibat resulted in a 56% reduction in bile acid levels, demonstrating its efficacy in managing cholestatic pruritus and liver disease development.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Odevixibat: A Review of a Bioactive Compound for the Treatment of Pruritus Approved by the FDA. Porwal, M., Kumar, A., Rastogi, V., et al.[2023]

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. Switzerland pubmed.ncbi.nlm.nih.gov

Case Report: Add-on treatment with odevixibat in a new subtype of progressive familial intrahepatic cholestasis broadens the therapeutic horizon of genetic cholestasis. [2023]

4. United Arab Emirates pubmed.ncbi.nlm.nih.gov

Odevixibat: A Review of a Bioactive Compound for the Treatment of Pruritus Approved by the FDA. [2023]

5. England pubmed.ncbi.nlm.nih.gov

Odevixibat and partial external biliary diversion showed equal improvement of cholestasis in a patient with progressive familial intrahepatic cholestasis. [2021]