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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06121375




Registration number
NCT06121375
Ethics application status
Date submitted
2/11/2023
Date registered
7/11/2023

Titles & IDs
Public title
Study to Assess Efficacy, Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Obeticholic Acid (OCA) Compared to Placebo in Pediatric Participants With Biliary Atresia, Post-hepatoportoenterostomy
Scientific title
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
Secondary ID [1] 0 0
747-308
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Biliary Atresia 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - OCA
Treatment: Drugs - Matching Placebo

Active comparator: Participants receiving OCA - Participants will be randomized to receive OCA (starting at 1.5 milligrams \[mg\] adult equivalent dose \[AED\]) orally, with water, once daily. Dose will be titrated every 2 weeks in a stepwise manner for the first 6 weeks, starting at 1.5 mg AED and titrating through 3 mg AED to a maximum of 5 mg AED, as tolerated; a discussion with the Medical Monitor is encouraged when determining uptitration if considerable signs or symptoms have arisen. Following the 6-week dose titration phase, participants will continue at the tolerated dose for approximately 24 months in Age Expansion Treatment Phase.

Placebo comparator: Participants receiving Matching placebo - Participants will be randomized to receive matching placebo orally, with water, once daily. Dose will be titrated every 2 weeks in a stepwise manner for the first 6 weeks, starting at 1.5 mg AED and titrating through 3 mg AED to a maximum of 5 mg AED, as tolerated; a discussion with the Medical Monitor is encouraged when determining uptitration if considerable signs or symptoms have arisen. Following the 6-week dose titration phase, participants will continue at the tolerated dose for approximately 24 months in Age Expansion Treatment Phase.


Treatment: Drugs: OCA
OCA will be administered.

Treatment: Drugs: Matching Placebo
Matching Placebo will be administered.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Time to the First Occurrence of Composite Endpoint
Timepoint [1] 0 0
Up to Week 64
Secondary outcome [1] 0 0
Plasma concentration of unconjugated OCA (parent), glyco-OCA, tauro-OCA, and total OCA (molar sum of OCA and its active conjugates)
Timepoint [1] 0 0
Up to Week 64
Secondary outcome [2] 0 0
Change from Baseline in Gamma Glutamyl Transferase (GGT)
Timepoint [2] 0 0
Baseline and up to Week 64
Secondary outcome [3] 0 0
Change from Baseline in total and direct (conjugated) bilirubin
Timepoint [3] 0 0
Baseline and up to Week 64
Secondary outcome [4] 0 0
Change from Baseline in Fibroblast Growth Factor-19 (FGF-19)
Timepoint [4] 0 0
Baseline and up to Week 64
Secondary outcome [5] 0 0
Change from Baseline in 7-hydroxyl-4-cholesten-3-one (C4)
Timepoint [5] 0 0
Baseline and up to Week 64
Secondary outcome [6] 0 0
Change from Baseline in endogenous bile acids
Timepoint [6] 0 0
Baseline and up to Week 64
Secondary outcome [7] 0 0
Change from Baseline in liver stiffness as assessed by transient elastography
Timepoint [7] 0 0
Baseline and up to Week 64
Secondary outcome [8] 0 0
Change from Baseline in plasma levels of fat-soluble vitamins (D and K)
Timepoint [8] 0 0
Baseline and up to Week 64
Secondary outcome [9] 0 0
Safety and tolerability as assessed by the incidence of treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs)
Timepoint [9] 0 0
Up to Week 64

Eligibility
Key inclusion criteria
Inclusion criteria:

* Male or female pediatric participants from birth to <18 years old. Note: Participants aged <2 years old will not be enrolled until after review of safety data during the planned interim analysis and agreement from the Data Safety Monitoring Board (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 milligrams per deciliter (mg/dL) (34.2 micromoles per liter [µmol/L]) at least 3 months post-HPE procedure.
Minimum age
1 Day
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion criteria:

* Prior liver transplant or active status on transplant list.
* Participants 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 mol/L).
* Platelets <120,000/µL
* International normalized ratio (INR) =1.5.
* Current or history of complications of decompensated chronic liver disease including:

1. Gastroesophageal varices and/or variceal bleeding
2. Clinically evident ascites related to portal hypertension
3. Hepatic encephalopathy
4. Prior placement of portosystemic shunt
5. Hepatopulmonary syndrome or portopulmonary hypertension
6. Hepatorenal syndrome
7. Any evidence of portal hypertension based on imaging (e.g., cavernous transformation of portal vein, abdominal varices, etc.)
8. Hepatocellular carcinoma
9. Childs-Pugh B or C
* Height and weight Z-score <-2 per site-specific reference ranges.
* Acholic (pale) stools.
* Aspartate aminotransferase (AST) >4x ULN.
* Alanine aminotransferase >4x ULN
* GGT >500 Units per Liter (U/L)
* On anticoagulation therapy
* Albumin <3.5 grams per deciliter (g/dL).
* Inability to swallow tablets (i.e., tablet or mini-tablet formulations).

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
2/09/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2027
Actual
Sample size
Target
144
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Royal Childrens Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3104 - Parkville
Recruitment outside Australia
Country [1] 0 0
Malaysia
State/province [1] 0 0
Kelantan
Country [2] 0 0
Malaysia
State/province [2] 0 0
Kuala Lumpur
Country [3] 0 0
New Zealand
State/province [3] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Intercept Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Lynda Szczech, MD
Address 0 0
Intercept Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Scott Birnbaum
Address 0 0
Country 0 0
Phone 0 0
6197572331
Fax 0 0
Email 0 0
scott.birnbaum@interceptpharma.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06121375