Clinical trial NCT04524390: Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Biliary Atresia
Keywords: Biliary Atresia
Kasai
Biliary Tract Diseases
Bile Duct Diseases
Congenital Abnormalities
Lead sponsor: Mirum Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2020-08-24
Enrollment: 75
Sex: ALL
Minimum age (years): 21
Maximum age (years): 111
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).

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.

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)

Primary outcomes:
measure: Mean Change in Total Serum Bilirubin Levels
timeFrame: From baseline to Week 26
Secondary outcomes:
measure: Mean Change in Total Serum Bile Acids
timeFrame: From baseline to Week 26
measure: Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26
timeFrame: From baseline to Week 26
measure: Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death.
timeFrame: From Baseline to Week 26
measure: Proportion of Participants Undergoing Liver Transplantation or Death
timeFrame: From Baseline to Week 26
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.
timeFrame: From Baseline to Week 26
measure: Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL
timeFrame: From Baseline to Week 26
measure: Proportion of Participants With Mean sBA Levels ≤40 mmol/L
timeFrame: From Baseline to Week 26
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Division of Gastroenterology & Hepatology
state: Arizona
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta - Emory University School of Medicine
state: Georgia
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: New York
country: United States
facility: NYU Grossman School of Medicine
state: New York
city: New York
country: United States
facility: New York-Presbyterian - Columbia University Medical Center
state: New York
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Beijing
country: China
facility: Beijing Pediatric Research Institute
state: Beijing
city: Guangzhou
country: China
facility: Guangzhou Women and Children's Medical Center
state: Guangdong
city: Hanzhou
country: China
facility: The Children's Hospital, Zhejiang University School of Medicine
state: Zhejiang
city: Shanghai
country: China
facility: Children's hospital of Shanghai
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
city: Hanover
country: Germany
facility: Hannover Medical School
city: Warsaw
country: Poland
facility: Instytut Pomnik-Centrum Zdrowia Dziecka
city: Bukit Timah
country: Singapore
facility: KK women's and Children's hospital
city: Taichung
country: Taiwan
facility: Taichung Veterans General Hospital
city: Taoyuan
country: Taiwan
facility: Linkou Chang Gung Memorial Hospital
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: London
country: United Kingdom
facility: King's College Hospital NHS
city: Huế
country: Vietnam
facility: Hue Central Hospital
state: Thừa Thiên Huế
city: Hanoi
country: Vietnam
facility: Vietnam National Children's Hospital
city: Ho Chi Minh City
country: Vietnam
facility: Children's Hospital No. 1
Study officials:
References: