BridgeMD
A Randomized, Double-blind, Placebo-controlled, Phase 2/3 Study to Assess the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Obeticholic Acid Compared to Placebo in Pediatric Subjects with Biliary Atresia, Post-hepatoportoenterostomy
EU CTIS ID: 2023-503926-37-00
What this study is testing
To evaluate the effect of OCA compared to placebo in conjunction with established local standard of care on clinical outcomes in subjects with biliary atresia who have had a successful Kasai procedure as measured by time to first occurrence of any component of a composite endpoint comprised of the following adjudicated events: Death (all-cause) Liver transplant Pediatric end-stage liver disease (PELD) score ≥17/model of end stage liver disease (MELD) ≥15 Hospitalization (as defined by a stay of 24 hours or greater) for new onset or recurrence of: − Variceal bleed − Hepatic encephalopathy (as defined by a West Haven score of ≥2) − Spontaneous bacterial peritonitis (confirmed by diagnostic paracentesis) Clinically evident ascites related to portal hypertension (diuretic-resistant ascites requiring therapeutic paracentesis at a frequency of at least twice in a month)
- Phase II and Phase III (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male or female pediatric subjects from birth to <18 years old Note: Subjects aged <2 years old will not be enrolled until after review of safety data during the planned interim analysis and agreement from the DSMB that there is sufficient safety data to enroll this age group.
- Diagnosis of non-syndromic biliary atresia
- Demonstrated successful HPE as defined by total bilirubin <2 mg/dL (34.2 μmol/L) at least 3 months post-HPE procedure.
- Female subjects of childbearing potential must use ≥1 highly effective method (≤1% failure rate) of contraception from the initiation of Screening and until at least 20 days (5 half-lives), after the last dose of investigational product. Highly effective methods of contraception are considered to be those listed below: • Surgical sterilization (bilateral tubal occlusion, etc.) • Placement of an intrauterine device (IUD) or intrauterine system (e.g., intrauterine hormone-releasing system [IUS]). • Combined (estrogen and progesterone containing) hormonal contraceptive associated with inhibition of ovulation: − Oral − Intravaginal − Transdermal • Progesterone-only hormonal contraception associated with inhibition of ovulation: − Oral − Injectable − Implantable • In the context of this study, the goal of using a highly effective contraception method is to avoid the potential embryofetal risks from exposure to investigational medicinal products. Sexual abstinence, as a form of highly effective contraception, is defined as avoiding all types of sexual activity that could result in pregnancy during the entire period of the study treatment until at least 20 days (5 half-lives), after the last dose of investigational product. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical study.
- Male subjects who are sexually active with female partners of childbearing potential must agree to use a condom with spermicide and to use one other approved method of highly effective contraception from the time of initiation of the investigational product administration and until at least 20 days (5 half-lives), after the last dose of investigational product.
- Male subjects, if permitted to donate sperm per local regulations, must refrain from sperm donation from Screening through at least 20 days (5 half-lives) after the last dose of investigational product.
You likely can't join if
- Prior liver transplant or active status on transplant list
- International normalized ratio (INR) ≥1.5
- Current or history of complications of decompensated chronic liver disease including: a.) gastroesophageal varices and/or variceal bleeding; b.) clinically evident ascites related to portal hypertension; c.) hepatic encephalopathy; d.) prior placement of portosystemic shunt; e.) hepatopulmonary syndrome or portopulmonary hypertension; f.) hepatorenal syndrome; g.) any evidence of portal hypertension based on imaging (e.g., cavernous transformation of portal vein, abdominal varices, etc.); h.) Hepatocellular carcinoma; i.) Childs-Pugh B or C
- Height and weight Z-score <-2 per site-specific reference ranges
- Acholic (pale) stools
- Aspartate aminotransferase (AST) >4x ULN
See the full eligibility criteria
Who can join
- Male or female pediatric subjects from birth to <18 years old Note: Subjects aged <2 years old will not be enrolled until after review of safety data during the planned interim analysis and agreement from the DSMB that there is sufficient safety data to enroll this age group.
- Diagnosis of non-syndromic biliary atresia
- Demonstrated successful HPE as defined by total bilirubin <2 mg/dL (34.2 μmol/L) at least 3 months post-HPE procedure.
- Female subjects of childbearing potential must use ≥1 highly effective method (≤1% failure rate) of contraception from the initiation of Screening and until at least 20 days (5 half-lives), after the last dose of investigational product. Highly effective methods of contraception are considered to be those listed below: • Surgical sterilization (bilateral tubal occlusion, etc.) • Placement of an intrauterine device (IUD) or intrauterine system (e.g., intrauterine hormone-releasing system [IUS]). • Combined (estrogen and progesterone containing) hormonal contraceptive associated with inhibition of ovulation: − Oral − Intravaginal − Transdermal • Progesterone-only hormonal contraception associated with inhibition of ovulation: − Oral − Injectable − Implantable • In the context of this study, the goal of using a highly effective contraception method is to avoid the potential embryofetal risks from exposure to investigational medicinal products. Sexual abstinence, as a form of highly effective contraception, is defined as avoiding all types of sexual activity that could result in pregnancy during the entire period of the study treatment until at least 20 days (5 half-lives), after the last dose of investigational product. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical study.
- Male subjects who are sexually active with female partners of childbearing potential must agree to use a condom with spermicide and to use one other approved method of highly effective contraception from the time of initiation of the investigational product administration and until at least 20 days (5 half-lives), after the last dose of investigational product.
- Male subjects, if permitted to donate sperm per local regulations, must refrain from sperm donation from Screening through at least 20 days (5 half-lives) after the last dose of investigational product.
- Parent or guardian is willing to provide written informed consent and when appropriate, the subject is willing to assent and agree to comply with the study protocol.
What rules you out
- Prior liver transplant or active status on transplant list
- International normalized ratio (INR) ≥1.5
- Current or history of complications of decompensated chronic liver disease including: a.) gastroesophageal varices and/or variceal bleeding; b.) clinically evident ascites related to portal hypertension; c.) hepatic encephalopathy; d.) prior placement of portosystemic shunt; e.) hepatopulmonary syndrome or portopulmonary hypertension; f.) hepatorenal syndrome; g.) any evidence of portal hypertension based on imaging (e.g., cavernous transformation of portal vein, abdominal varices, etc.); h.) Hepatocellular carcinoma; i.) Childs-Pugh B or C
- Height and weight Z-score <-2 per site-specific reference ranges
- Acholic (pale) stools
- Aspartate aminotransferase (AST) >4x ULN
- History of known or suspected clinically significant hypersensitivity to OCA or any of its excipients
- If female, known pregnancy, or has a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating
- Subjects who are committed to an institution by virtue of an order issued either by the judicial or administrative authorities per local regulations
- Subjects who are children of or related to investigational site staff members, site staff members otherwise supervised by the investigator, or sponsor employees directly involved in the conduct of the study
- Alanine aminotransferase >4x ULN
- GGT >500 U/L
- Anticoagulation therapy
- Albumin <3.5 g/dL
- Inability to swallow tablets (i.e., tablet or mini-tablet formulations)
- Subjects diagnosed with biliary atresia splenic malformation (BASM)
- Conjugated (direct) bilirubin ≥ upper limit of normal (ULN) of site-specific reference range. If conjugated bilirubin is not available: total bilirubin ≥2 mg/dL (34.2 μmol/L)
- Platelets <120,000/μL
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- Singapore
- New Zealand
- Australia
- China
- Hong Kong
- Malaysia
- Canada
- Turkey
- Vietnam
- Taiwan
- Israel
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; Singapore; New Zealand; Australia; China; Hong Kong and 6 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.