Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
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| ClinicalTrials.gov Identifier: NCT04336722 |
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Recruitment Status :
Active, not recruiting
First Posted : April 7, 2020
Last Update Posted : November 25, 2022
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Sponsor:
Albireo
Information provided by (Responsible Party):
Albireo
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Brief Summary:
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in children with biliary atresia who have undergone a Kasai hepatoportoenterostomy.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Biliary Atresia | Drug: Odevixibat Drug: Placebo | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 205 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Triple (Participant, Care Provider, Investigator) |
| Primary Purpose: | Treatment |
| Official Title: | A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy |
| Actual Study Start Date : | July 10, 2020 |
| Estimated Primary Completion Date : | May 2024 |
| Estimated Study Completion Date : | June 2024 |
Resource links provided by the National Library of Medicine
Drug Information available for:
Odevixibat
| Arm | Intervention/treatment |
|---|---|
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Experimental: Odevixibat (A4250)
Capsules for oral administration once daily for 104 weeks.
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Drug: Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT. |
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Placebo Comparator: Placebo
Capsules for oral administration (to match active) once daily for 104 weeks.
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Drug: Placebo
Placebo identical in appearance to experimental drug (odevixibat). |
Primary Outcome Measures :
- Proportion of patients with liver transplant [ Time Frame: From baseline to Week 104 ]Proportion of patients who are alive and have not undergone a liver transplant after 104 weeks of study treatment.
Secondary Outcome Measures :
- Time to onset of any sentinel events [ Time Frame: From baseline to Week 104 ]Time to onset of any sentinel events
- Time to pediatric end-stage liver disease (PELD) score >15 [ Time Frame: From baseline to Week 104 ]Time to pediatric end-stage liver disease (PELD) score >15
- Total bilirubin levels [ Time Frame: From baseline to Weeks 13, 26, 52 and 104 ]Total bilirubin level after 13, 26, 52, and 104 weeks of study treatment
- Serum bile acid levels [ Time Frame: From baseline to Weeks 13, 26, 52 and 104 ]Serum bile acid level after 13, 26, 52, and 104 weeks of study treatment
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| Ages Eligible for Study: | up to 111 Days (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- A male or female patient with a clinical diagnosis of BA
- Age at Kasai HPE ≤90 days
- Eligible to start study treatment within 3 weeks post-Kasai HPE
Key Exclusion Criteria:
- Patients with intractable ascites
- Ileal resection surgery
- ALT ≥10× upper limit of normal (ULN) at screening
- Patients reliant only on total parenteral nutrition, or not able to take study medication orally, at randomization
- Acute ascending cholangitis (patients may be randomized after resolution of acute ascending cholangitis)
- Choledochal cystic disease
- INR >1.6 (the patient may be treated with Vitamin K intravenously; sample may be redrawn and if INR is ≤1.6 at resampling the patient may be randomized)
- Any other conditions or abnormalities, including congenital abnormalities, major cardiac surgery, hepatic, biliary, or GI disease which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the patient, the integrity of study results, or patient compliance with study requirements
- Weight <3.5kg at randomization
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04336722
Locations
Show 71 study locations
Sponsors and Collaborators
Albireo
| Responsible Party: | Albireo |
| ClinicalTrials.gov Identifier: | NCT04336722 |
| Other Study ID Numbers: |
A4250-011 |
| First Posted: | April 7, 2020 Key Record Dates |
| Last Update Posted: | November 25, 2022 |
| Last Verified: | November 2022 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Albireo:
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Kasai |
Additional relevant MeSH terms:
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Biliary Atresia Bile Duct Diseases Biliary Tract Diseases |
Digestive System Diseases Digestive System Abnormalities Congenital Abnormalities |