Brief Summary
A study to evaluate the efficacy and safety of maralixibat in infants with Biliary Atresia (BA) after Hepatoportoenterostomy (HPE, also known as the Kasai procedure).
Brief Title
Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
Detailed Description
This is a double-blind randomized, placebo-controlled study in subjects with Biliary Atresia with a primary endpoint at Week 26 followed by long-term open-label period during which all subjects will receive maralixibat to Week 104.
Completion Date
Completion Date Type
Actual
Conditions
Biliary Atresia
Eligibility Criteria
Inclusion Criteria:
1. Male or female subjects with body weight ≥2500 g, who are ≥21 days old and \<90 days old at the time of HPE (Kasai)
2. HPE or Kasai Procedure within 3 weeks prior to randomization
3. Clinical diagnosis of biliary atresia
Exclusion Criteria:
1. Subjects with intractable chronic diarrhea at randomization
2. Subjects not tolerating enteral feeds at randomization
3. History of ileal resection
4. Diagnosis of biliary atresia splenic malformation syndrome or cystic biliary atresia
5. Evidence of another non-biliary atresia pathology involving the intrahepatic bile duct (e.g., paucity, sclerosing cholangitis)
6. Evidence of liver failure (e.g. significant ascites)
1. Male or female subjects with body weight ≥2500 g, who are ≥21 days old and \<90 days old at the time of HPE (Kasai)
2. HPE or Kasai Procedure within 3 weeks prior to randomization
3. Clinical diagnosis of biliary atresia
Exclusion Criteria:
1. Subjects with intractable chronic diarrhea at randomization
2. Subjects not tolerating enteral feeds at randomization
3. History of ileal resection
4. Diagnosis of biliary atresia splenic malformation syndrome or cystic biliary atresia
5. Evidence of another non-biliary atresia pathology involving the intrahepatic bile duct (e.g., paucity, sclerosing cholangitis)
6. Evidence of liver failure (e.g. significant ascites)
Inclusion Criteria
Inclusion Criteria:
1. Male or female subjects with body weight ≥2500 g, who are ≥21 days old and \<90 days old at the time of HPE (Kasai)
2. HPE or Kasai Procedure within 3 weeks prior to randomization
3. Clinical diagnosis of biliary atresia
1. Male or female subjects with body weight ≥2500 g, who are ≥21 days old and \<90 days old at the time of HPE (Kasai)
2. HPE or Kasai Procedure within 3 weeks prior to randomization
3. Clinical diagnosis of biliary atresia
Gender
All
Gender Based
false
Keywords
Biliary Atresia
Kasai
Biliary Tract Diseases
Bile Duct Diseases
Congenital Abnormalities
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
111 Days
Minimum Age
21 Days
NCT Id
NCT04524390
Org Class
Industry
Org Full Name
Mirum Pharmaceuticals, Inc.
Org Study Id
MRX-701
Overall Status
Completed
Phases
Phase 2
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Biliary Atresia After Hepatoportoenterostomy
Primary Outcomes
Outcome Measure
Mean Change in Total Serum Bilirubin Levels
Outcome Time Frame
From baseline to Week 26
Secondary Outcomes
Outcome Time Frame
From baseline to Week 26
Outcome Measure
Mean Change in Total Serum Bile Acids
Outcome Time Frame
From baseline to Week 26
Outcome Measure
Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26
Outcome Description
Liver decompensation (hepatic encephalopathy, variceal bleeding, new persistent ascites)
Outcome Time Frame
From Baseline to Week 26
Outcome Measure
Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death.
Outcome Time Frame
From Baseline to Week 26
Outcome Measure
Proportion of Participants Undergoing Liver Transplantation or Death
Outcome Description
Splenomegaly =\> (spleen size \>2 cm below the costal margin palpated on physical examination)
Outcome Time Frame
From Baseline to Week 26
Outcome Measure
Proportion of Participants Observed to Develop Clinically Evident Portal Hypertension Defined as Splenomegaly and Thrombocytopenia (Platelet Count <150 x 109/L) or Clinically Evident Ascites or Endoscopic Evidence of Esophageal or Gastric Varices.
Outcome Time Frame
From Baseline to Week 26
Outcome Measure
Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL
Outcome Time Frame
From Baseline to Week 26
Outcome Measure
Proportion of Participants With Mean sBA Levels ≤40 mmol/L
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
Lidija Palezac
Investigator Email
lpalezac@montefiore.org