Trial Outcomes & Findings for 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). (NCT NCT04729751)

NCT ID: NCT04729751

Last Updated: 2026-06-02

Results Overview

TEAEs = Treatment-emergent Adverse Events

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

27 participants

Primary outcome timeframe

From Baseline through to Week 13

Results posted on

2026-06-02

Participant Flow

A total of 27 participants were enrolled in the study, across 10 sites in 6 countries (Belgium, Brazil, France, Poland, United Kingdom, and United States), 17 patients in the ALGS Cohort and 10 in the PFIC Cohort.

The screening period starts when informed consent (by the legally authorized representative) is signed. The duration of the screening period is up to 4 weeks, during which all procedures listed for the screening visit in the schedule of assessment must be completed.

Participant milestones

Participant milestones
Measure	Maralixibat: ALGS Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 μg/kg QD, 300 μg/kg BID or 600 µg/kg BID.
Overall Study STARTED	17	10
Overall Study COMPLETED	16	9
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Maralixibat: ALGS Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 μg/kg QD, 300 μg/kg BID or 600 µg/kg BID.
Overall Study Withdrawal by Subject	1	0
Overall Study Adverse Event	0	1

Baseline Characteristics

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).

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Maralixibat :ALGS n=17 Participants Participants will receive up to 400 μg/kg once daily (ALGS) over 13 weeks in the core study and for the duration of the Long Term Extension (LTE) where applicable. Maralixibat: Maralixibat chloride provided in the form of an oral solution (i.e., 5, 10, 15, and 20 mg/mL) \*400 μg/kg maralixibat chloride is equivalent to 380 µg/kg maralixibat free base	Maralixibat: PFIC n=10 Participants Participants will receive up to 600 μg/kg twice daily (PFIC) over 13 weeks in the core study and for the duration of the Long Term Extension (LTE) where applicable. Maralixibat: Maralixibat chloride provided in the form of an oral solution (i.e., 5, 10, 15, and 20 mg/mL) \*600 μg/kg maralixibat chloride is equivalent to 570 µg/kg maralixibat free base	Total n=27 Participants Total of all reporting groups
Age, Customized Preterm newborn - gestational age < 37 weeks	0 Participants n=9 Participants	0 Participants n=27 Participants	0 Participants n=267 Participants
Age, Customized Newborns (0-27 days)	0 Participants n=9 Participants	0 Participants n=27 Participants	0 Participants n=267 Participants
Age, Customized Infants and toddlers (28 days-23 months)	17 Participants n=9 Participants	10 Participants n=27 Participants	27 Participants n=267 Participants
Sex: Female, Male Female	3 Participants n=9 Participants	3 Participants n=27 Participants	6 Participants n=267 Participants
Sex: Female, Male Male	14 Participants n=9 Participants	7 Participants n=27 Participants	21 Participants n=267 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=9 Participants	0 Participants n=27 Participants	0 Participants n=267 Participants
Race (NIH/OMB) Asian	0 Participants n=9 Participants	0 Participants n=27 Participants	0 Participants n=267 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=9 Participants	0 Participants n=27 Participants	0 Participants n=267 Participants
Race (NIH/OMB) Black or African American	0 Participants n=9 Participants	0 Participants n=27 Participants	0 Participants n=267 Participants
Race (NIH/OMB) White	7 Participants n=9 Participants	6 Participants n=27 Participants	13 Participants n=267 Participants
Race (NIH/OMB) More than one race	1 Participants n=9 Participants	0 Participants n=27 Participants	1 Participants n=267 Participants
Race (NIH/OMB) Unknown or Not Reported	9 Participants n=9 Participants	4 Participants n=27 Participants	13 Participants n=267 Participants
Region of Enrollment Belgium	1 participants n=9 Participants	0 participants n=27 Participants	1 participants n=267 Participants
Region of Enrollment United States	6 participants n=9 Participants	1 participants n=27 Participants	7 participants n=267 Participants
Region of Enrollment Poland	4 participants n=9 Participants	3 participants n=27 Participants	7 participants n=267 Participants
Region of Enrollment Brazil	4 participants n=9 Participants	0 participants n=27 Participants	4 participants n=267 Participants
Region of Enrollment United Kingdom	2 participants n=9 Participants	3 participants n=27 Participants	5 participants n=267 Participants
Region of Enrollment France	2 participants n=9 Participants	1 participants n=27 Participants	3 participants n=267 Participants

PRIMARY outcome

Timeframe: From Baseline through to Week 13

Population: Includes all participants with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.

TEAEs = Treatment-emergent Adverse Events

Outcome measures

Outcome measures
Measure	Maralixibat: ALGS n=17 Participants Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC n=10 Participants Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 µg/kg QD, 300 µg/kg BID, or 600 µg/kg BID.
Frequency of Treatment-emergent Adverse Events [TEAEs] At least one TEAE	16 Participants	10 Participants
Frequency of Treatment-emergent Adverse Events [TEAEs] TEAE related to study drug	4 Participants	3 Participants
Frequency of Treatment-emergent Adverse Events [TEAEs] Grade ≥3 TEAE	6 Participants	1 Participants
Frequency of Treatment-emergent Adverse Events [TEAEs] Grade ≥3 TEAE related to study drug	0 Participants	0 Participants
Frequency of Treatment-emergent Adverse Events [TEAEs] Serious TEAE	6 Participants	2 Participants
Frequency of Treatment-emergent Adverse Events [TEAEs] Serious TEAE related to study drug	0 Participants	0 Participants
Frequency of Treatment-emergent Adverse Events [TEAEs] TEAE that led to study discontinuation	0 Participants	1 Participants
Frequency of Treatment-emergent Adverse Events [TEAEs] TEAE that led to death	0 Participants	0 Participants

SECONDARY outcome

Timeframe: From Baseline through to Week 13

Population: Includes all participants with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.

sBA = serum bile acid

Outcome measures

Outcome measures
Measure	Maralixibat: ALGS n=17 Participants Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC n=10 Participants Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 µg/kg QD, 300 µg/kg BID, or 600 µg/kg BID.
Change in Fasting Serum Bile Acid (sBA) Levels Baseline	292.81 µmol/L Standard Deviation 218.756	228.32 µmol/L Standard Deviation 107.52
Change in Fasting Serum Bile Acid (sBA) Levels Week 13	172.49 µmol/L Standard Deviation 109.924	138.90 µmol/L Standard Deviation 91.610
Change in Fasting Serum Bile Acid (sBA) Levels Change from Baseline	-75.68 µmol/L Standard Deviation 115.669	-77.49 µmol/L Standard Deviation 128.616

SECONDARY outcome

Timeframe: From Baseline through to Week 13

Population: Includes all participants with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.

ALT= alanine aminotransferase.

Outcome measures

Outcome measures
Measure	Maralixibat: ALGS n=17 Participants Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC n=10 Participants Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 µg/kg QD, 300 µg/kg BID, or 600 µg/kg BID.
To Evaluate the Effect on Liver Enzymes (ALT) ALT, U/L Baseline	167.35 U/L Standard Deviation 106.018	149.00 U/L Standard Deviation 110.108
To Evaluate the Effect on Liver Enzymes (ALT) ALT, U/L Week 13	256.87 U/L Standard Deviation 190.732	80.50 U/L Standard Deviation 41.946
To Evaluate the Effect on Liver Enzymes (ALT) ALT, U/L Change from Baseline	99.00 U/L Standard Deviation 190.981	-27.00 U/L Standard Deviation 25.031

SECONDARY outcome

Timeframe: From Baseline through to Week 13

Population: Includes all participants with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.

Change from baseline to Week 13 in vitamin E.

Outcome measures

Outcome measures
Measure	Maralixibat: ALGS n=17 Participants Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC n=10 Participants Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 µg/kg QD, 300 µg/kg BID, or 600 µg/kg BID.
Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamin E Vitamin E mg/L Week 13	5.99 mg/L Standard Deviation 5.084	3.98 mg/L Standard Deviation 3.658
Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamin E Vitamin E mg/L Change from Baseline	-0.46 mg/L Standard Deviation 2.214	0.92 mg/L Standard Deviation 1.422
Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamin E Vitamin E mg/L Baseline	6.54 mg/L Standard Deviation 5.351	2.78 mg/L Standard Deviation 2.149

SECONDARY outcome

Timeframe: At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit

Population: Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat.

BID=twice daily; QD=once daily. Systemic concentrations of maralixibat in plasma were determined at the following visits: * Approximately 2.5 hours after the morning dose at Week 0/Day 1 of dosing * Before dosing and \~2.5 hours after the morning dose at Week 6, Week 10, and Week 13 Stable dosing occurred at 400 μg/kg QD for ALGS or at the highest tolerated dose. Note: The values added in section Measured Values are for maralixibat dose of 400 µg/kg QD.

Outcome measures

Outcome measures
Measure	Maralixibat: ALGS n=17 Participants Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 µg/kg QD, 300 µg/kg BID, or 600 µg/kg BID.
Maximum Level of Maralixibat Concentration in Plasma From Baseline to Week 13 for ALGS	0.5576 ng/mL Standard Deviation 0.6107	—

SECONDARY outcome

Timeframe: From Baseline through to Week 13.

Population: Includes all participants with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.

AST= aspartate aminotransferase

Outcome measures

Outcome measures
Measure	Maralixibat: ALGS n=17 Participants Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC n=10 Participants Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 µg/kg QD, 300 µg/kg BID, or 600 µg/kg BID.
To Evaluate the Effect on Liver Enzymes (AST) AST, U/L Baseline	184.12 U/L Standard Deviation 105.42	159.10 U/L Standard Deviation 97.034
To Evaluate the Effect on Liver Enzymes (AST) AST, U/L Week 13	236.8 U/L Standard Deviation 142.540	85.71 U/L Standard Deviation 36.068
To Evaluate the Effect on Liver Enzymes (AST) AST, U/L Change from Baseline	69.27 U/L Standard Deviation 136.827	-30.43 U/L Standard Deviation 32.264

SECONDARY outcome

Timeframe: From Baseline through to Week 13

Population: Includes all participants with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.

Bilirubin

Outcome measures

Outcome measures
Measure	Maralixibat: ALGS n=17 Participants Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC n=10 Participants Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 µg/kg QD, 300 µg/kg BID, or 600 µg/kg BID.
To Evaluate the Effect on Bilirubin Total bilirubin, µmol/L Baseline	142.54 µmol/L Standard Deviation 102.323	41.02 µmol/L Standard Deviation 23.651
To Evaluate the Effect on Bilirubin Total bilirubin, µmol/L Week 13	114.89 µmol/L Standard Deviation 102.789	18.51 µmol/L Standard Deviation 16.720
To Evaluate the Effect on Bilirubin Total bilirubin, µmol/L Change from Baseline	-22.85 µmol/L Standard Deviation 46.252	-18.01 µmol/L Standard Deviation 18.781

SECONDARY outcome

Timeframe: From Baseline through to Week 13

Population: Includes all participants with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.

Change from baseline to Week 13 in vitamin A

Outcome measures

Outcome measures
Measure	Maralixibat: ALGS n=17 Participants Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC n=10 Participants Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 µg/kg QD, 300 µg/kg BID, or 600 µg/kg BID.
Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamin A Vitamin A umol/L Baseline	1.78 umol/L Standard Deviation 1.189	1.50 umol/L Standard Deviation 0.991
Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamin A Vitamin A umol/L Week 13	2.08 umol/L Standard Deviation 1.031	2.32 umol/L Standard Deviation 0.884
Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamin A Vitamin A umol/L Change from Baseline	0.45 umol/L Standard Deviation 0.678	0.61 umol/L Standard Deviation 0.878

SECONDARY outcome

Timeframe: From Baseline through to Week 13

Population: Includes all participants with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.

Change from baseline to Week 13 in vitamins D and K.

Outcome measures

Outcome measures
Measure	Maralixibat: ALGS n=17 Participants Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC n=10 Participants Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 µg/kg QD, 300 µg/kg BID, or 600 µg/kg BID.
Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamins D and K Vitamin K nmol/L Change from Baseline	-241.14 nmol/L Standard Deviation NA Not applicable as Standard Deviation could not be calculated due to the analyzed number of participants at week 13. Only a single data point was available.	—
Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamins D and K Vitamin D nmol/L Baseline	62.64 nmol/L Standard Deviation 58.563	62.87 nmol/L Standard Deviation 60.308
Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamins D and K Vitamin D nmol/L Week 13	78.42 nmol/L Standard Deviation 58.952	94.84 nmol/L Standard Deviation 56.849
Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamins D and K Vitamin D nmol/L Change from Baseline	7.63 nmol/L Standard Deviation 79.383	20.65 nmol/L Standard Deviation 44.394
Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamins D and K Vitamin K nmol/L Baseline	31.22 nmol/L Standard Deviation 84.315	0.21 nmol/L Standard Deviation 0.133
Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamins D and K Vitamin K nmol/L Week 13	14.64 nmol/L Standard Deviation NA Not applicable as Standard Deviation could not be calculated due to the analyzed number of participants at week 13. Only a single data point was available.	—

SECONDARY outcome

Timeframe: At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit

Population: Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.

BID=twice daily; QD=once daily. Systemic concentrations of maralixibat in plasma were determined at the following visits: * Approximately 2.5 hours after the morning dose at Week 0/Day 1 of dosing * Before dosing and \~2.5 hours after the morning dose at Week 6, Week 10, and Week 13 Stable dosing occurred at 300 μg/kg QD, 300 μg/kg BID and 600 μg/kg BID for PFIC or at the highest tolerated dose. Note: The values added in section Measured Values are for maralixibat dose of 600 µg/kg BID.

Outcome measures

Outcome measures
Measure	Maralixibat: ALGS n=10 Participants Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 µg/kg QD, 300 µg/kg BID, or 600 µg/kg BID.
Maximum Level of Maralixibat Concentration in Plasma From Baseline to Week 13 for PFIC	0.153 ng/mL Standard Deviation 0.2629	—

Adverse Events

Maralixibat: ALGS

Serious events: 6 serious events

Other events: 16 other events

Deaths: 0 deaths

Maralixibat: PFIC

Serious events: 2 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Mirum Clinical Trials

Mirum Pharmaceuticals, Inc

Phone: 16506674085

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60

Measure	Maralixibat: ALGS n=17 participants at risk Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC n=10 participants at risk Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 μg/kg QD, 300 μg/kg BID or 600 µg/kg BID.
General disorders Crying	5.9% 1/17 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
General disorders Pyrexia	5.9% 1/17 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
Gastrointestinal disorders Diarrhoea	5.9% 1/17 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
General disorders Infantile colic	5.9% 1/17 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
Gastrointestinal disorders Intestinal obstruction	0.00% 0/17 • The timeframe for reporting adverse events was from Baseline to study week 147.	10.0% 1/10 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Abdominal sepsis	0.00% 0/17 • The timeframe for reporting adverse events was from Baseline to study week 147.	10.0% 1/10 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Adenovirus infection	0.00% 0/17 • The timeframe for reporting adverse events was from Baseline to study week 147.	10.0% 1/10 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Appendicitis perforated	0.00% 0/17 • The timeframe for reporting adverse events was from Baseline to study week 147.	10.0% 1/10 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Corona virus infection	5.9% 1/17 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Gastroenteritis adenovirus	0.00% 0/17 • The timeframe for reporting adverse events was from Baseline to study week 147.	10.0% 1/10 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Influenza	0.00% 0/17 • The timeframe for reporting adverse events was from Baseline to study week 147.	10.0% 1/10 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Otitis media acute	5.9% 1/17 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Pneumonia	5.9% 1/17 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Sepsis	5.9% 1/17 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Varicella	5.9% 1/17 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Viral infection	5.9% 1/17 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.

Measure	Maralixibat: ALGS n=17 participants at risk Includes all participants with Alagille Syndrome (ALGS) who received at least one dose of maralixibat. Participants with ALGS received maralixibat doses of either: 200 µg/kg or 400 µg/kg QD.	Maralixibat: PFIC n=10 participants at risk Includes all participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat. Participants with PFIC received maralixibat doses of either: 300 μg/kg QD, 300 μg/kg BID or 600 µg/kg BID.
Injury, poisoning and procedural complications Injury, poisoning and procedural complications	5.9% 1/17 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.	20.0% 2/10 • Number of events 4 • The timeframe for reporting adverse events was from Baseline to study week 147.
Nervous system disorders Nervous system disorders	11.8% 2/17 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.	20.0% 2/10 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.
Blood and lymphatic system disorders Coagulopathy	0.00% 0/17 • The timeframe for reporting adverse events was from Baseline to study week 147.	30.0% 3/10 • Number of events 6 • The timeframe for reporting adverse events was from Baseline to study week 147.
General disorders Pyrexia	17.6% 3/17 • Number of events 8 • The timeframe for reporting adverse events was from Baseline to study week 147.	20.0% 2/10 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.
Gastrointestinal disorders Diarrhoea	41.2% 7/17 • Number of events 15 • The timeframe for reporting adverse events was from Baseline to study week 147.	40.0% 4/10 • Number of events 8 • The timeframe for reporting adverse events was from Baseline to study week 147.
Gastrointestinal disorders Vomiting	11.8% 2/17 • Number of events 5 • The timeframe for reporting adverse events was from Baseline to study week 147.	50.0% 5/10 • Number of events 8 • The timeframe for reporting adverse events was from Baseline to study week 147.
Gastrointestinal disorders Teething	17.6% 3/17 • Number of events 7 • The timeframe for reporting adverse events was from Baseline to study week 147.	20.0% 2/10 • Number of events 3 • The timeframe for reporting adverse events was from Baseline to study week 147.
Gastrointestinal disorders Abdominal pain	17.6% 3/17 • Number of events 4 • The timeframe for reporting adverse events was from Baseline to study week 147.	10.0% 1/10 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.
Respiratory, thoracic and mediastinal disorders Cough	23.5% 4/17 • Number of events 5 • The timeframe for reporting adverse events was from Baseline to study week 147.	20.0% 2/10 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	11.8% 2/17 • Number of events 3 • The timeframe for reporting adverse events was from Baseline to study week 147.	30.0% 3/10 • Number of events 4 • The timeframe for reporting adverse events was from Baseline to study week 147.
Respiratory, thoracic and mediastinal disorders Nasal congestion	11.8% 2/17 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders	23.5% 4/17 • Number of events 5 • The timeframe for reporting adverse events was from Baseline to study week 147.	20.0% 2/10 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Nasopharyngitis	35.3% 6/17 • Number of events 9 • The timeframe for reporting adverse events was from Baseline to study week 147.	40.0% 4/10 • Number of events 6 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Upper respiratory tract infection	23.5% 4/17 • Number of events 12 • The timeframe for reporting adverse events was from Baseline to study week 147.	40.0% 4/10 • Number of events 5 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Viral infection	23.5% 4/17 • Number of events 4 • The timeframe for reporting adverse events was from Baseline to study week 147.	40.0% 4/10 • Number of events 7 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Corona virus infection	17.6% 3/17 • Number of events 3 • The timeframe for reporting adverse events was from Baseline to study week 147.	20.0% 2/10 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Rhinitis	17.6% 3/17 • Number of events 3 • The timeframe for reporting adverse events was from Baseline to study week 147.	20.0% 2/10 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Ear infection	23.5% 4/17 • Number of events 8 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Gastroenteritis	11.8% 2/17 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.	10.0% 1/10 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Influenza	5.9% 1/17 • Number of events 1 • The timeframe for reporting adverse events was from Baseline to study week 147.	10.0% 1/10 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Conjunctivitis	11.8% 2/17 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Otitis media	11.8% 2/17 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
Infections and infestations Respiratory tract infection viral	11.8% 2/17 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
Metabolism and nutrition disorders Hypervitaminosis D	11.8% 2/17 • Number of events 2 • The timeframe for reporting adverse events was from Baseline to study week 147.	0.00% 0/10 • The timeframe for reporting adverse events was from Baseline to study week 147.
Investigations Investigations	17.6% 3/17 • Number of events 5 • The timeframe for reporting adverse events was from Baseline to study week 147.	30.0% 3/10 • Number of events 6 • The timeframe for reporting adverse events was from Baseline to study week 147.