Clinical trial NCT04729751: A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
Official title: Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Progressive Familial Intrahepatic Cholestasis
Alagille Syndrome
Cholestatic Liver Disease
Keywords: PFIC
ALGS
Maralixibat
Bile Duct Diseases
Liver Diseases
Biliary Tract Diseases
Pediatric
Lead sponsor: Mirum Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2021-01-28
Enrollment: 27
Sex: ALL
Minimum age (years): 0
Maximum age (years): 364
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single group with 2 cohorts - ≥ 6 participants each from ALGS and PFIC.
Primary purpose: TREATMENT

Brief summary: This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children \<12 months of age with Alagille Syndrome \[ALGS\] or Progressive Familial Intrahepatic Cholestasis \[PFIC\].

Detailed description: This is an open label study where all participants will receive maralixibat treatment.

Eligibility criteria: Inclusion Criteria:

1. Body weight of ≥2.5 kg
2. \<12 months of age at the baseline visit (ROW). \>31 days and \<12 months of age at the baseline visit (US).
3. Gestational age ≥36 weeks at birth. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required.
4. Diagnosis of PFIC or ALGS

Exclusion criteria:

1. Predicted complete absence of bile salt excretion pump (BSEP) function
2. History of surgical disruption of the enterohepatic circulation
3. History of liver transplant or imminent need for liver transplant
4. Decompensated cirrhosis
5. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per investigator discretion
6. Presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or medical monitor, may compromise the safety of the participant or interfere with the participant's participation in or completion of the study

Primary outcomes:
measure: Frequency of treatment-emergent adverse events [TEAEs]
timeFrame: From Baseline through to Week 13
Secondary outcomes:
measure: Change in fasting serum bile acid (sBA) levels
timeFrame: From Baseline through to Week 13
measure: To evaluate the effect on liver enzymes (ALT, AST) and bilirubin
timeFrame: From Baseline through to Week 13
measure: To evaluate the effect on LSVs
timeFrame: From Baseline through to Week 13
measure: To assess the plasma level of maralixibat in infant participants
timeFrame: At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children Hospital LA
state: California
city: San Francisco
country: United States
facility: University of California - San Francisco
state: California
city: Washington
country: United States
facility: Medstar Georgetown University Hospital
state: District of Columbia
city: New Orleans
country: United States
facility: Ochsner Hospital for Children
state: Louisiana
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Brussels
country: Belgium
facility: Cliniques Universitaires Saint-Luc
city: São Paulo
country: Brazil
facility: Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês
city: Le Kremlin-Bicêtre
country: France
facility: Hôpital Kremlin Bicêtre
city: Paris
country: France
facility: Hopital Necker
city: Zapopan
country: Mexico
facility: Consultorio de Joshue David Covarrubias Esquer
city: Warsaw
country: Poland
facility: Instytut Pomnik-Centrum Zdrowia Dziecka
city: London
country: United Kingdom
facility: King's College Hospital
Study officials:
References: