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.
Brief Title
Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
Detailed Description
Up to 70 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.
Completion Date
Completion Date Type
Estimated
Conditions
Biliary Atresia
Eligibility 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
* 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
Inclusion 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
* 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
Gender
All
Gender Based
false
Keywords
Kasai
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
111 Days
NCT Id
NCT04336722
Org Class
Industry
Org Full Name
Ipsen
Org Study Id
A4250-011
Overall Status
Active, not recruiting
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Estimated
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
Primary Outcomes
Outcome Measure
Time from randomization to first occurrence of liver transplant, or death
Outcome Time Frame
From baseline to Week 104
Secondary Ids
Secondary Id
2019-003807-37
Secondary Id
2024-512086-14-00
Secondary Outcomes
Outcome Description
Proportion of patients who are alive and have not undergone a liver transplant
Outcome Time Frame
From baseline to Week 104
Outcome Measure
Proportion of patients with liver transplant
Outcome Description
Time to onset of any sentinel events
Outcome Time Frame
From baseline to Week 104
Outcome Measure
Time to onset of any sentinel events
Outcome Description
Total bilirubin level after 13, 26, 52, and 104 weeks of study treatment
Outcome Time Frame
From baseline to Weeks 13, 26, 52 and 104
Outcome Measure
Total bilirubin levels
Outcome Description
Serum bile acid level after 13, 26, 52, and 104 weeks of study treatment
Outcome Time Frame
From baseline to Weeks 13, 26, 52 and 104
Outcome Measure
Serum bile acid levels
Outcome Description
Time to pediatric end-stage liver disease (PELD) score \>15
Outcome Time Frame
From baseline to Week 104
Outcome Measure
Time to pediatric end-stage liver disease (PELD) score >15
Outcome Description
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
Outcome Time Frame
From baseline to Week 104
Outcome Measure
Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
Outcome Description
Percentage of participants with clinically significant changes in physical examination findings will be reported. The clinical significance will be graded by the investigator.
Outcome Time Frame
From baseline to Week 104
Outcome Measure
Percentage of participants with clinically significant changes in Physical Examination
Outcome Description
Percentage of participants with clinically significant change in laboratory parameters (blood chemistry, hematology and coagulation) will be reported. The clinical significance will be decided by the investigator.
Outcome Time Frame
From baseline to Week 104
Outcome Measure
Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)
Outcome Description
Percentage of participants with clinically significant change in Abdominal Ultrasound findings will be reported. The clinical significance will be decided by the investigator.
Outcome Time Frame
From baseline to Week 26 and Week 104
Outcome Measure
Percentage of participants with clinically significant changes in Abdominal Ultrasound findings
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Child
Locked Fields
Render the field
Maximum Age Number (converted to Years and rounded down)
0
Minimum Age Number (converted to Years and rounded down)
0
Investigators
Investigator Type
Principal Investigator
Investigator Name
Debra Pan
Investigator Email
dpan@montefiore.org
Investigator Phone
718-741-2332