Evaluation of Maralixibat in Biliary Atresia Response Pos... | NCT04524390

Label	Type	Description	Intervention Names
Double Blind - Maralixibat	Experimental	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.	Drug: Maralixibat
Double Blind - Placebo	Placebo Comparator	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.	Other: Placebo
Open Label - Maralixibat	Experimental	The Open-Label period comprised of 4-8 weeks of dose escalation followed by 70 - 74 weeks of stable dosing treatment. During the OLE, all participants, regardless of treatment assignment in the double-blind period, received maralixibat.	Drug: Maralixibat

Name	Type	Description	Arm Group Labels	Other Names
Maralixibat	Drug	A small molecule inhibitor of the ileal bile acid transporter (IBAT)	Double Blind - Maralixibat Open Label - Maralixibat

Measure	Description	Time Frame
Mean Change in Total Serum Bilirubin Levels		From baseline to Week 26

Measure	Description	Time Frame
Mean Change in Total Serum Bile Acids		From baseline to Week 26
Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26		From baseline to Week 26

Facility	Status	City	State	ZIP	Country	Contacts
Phoenix Children's Division of Gastroenterology & Hepatology		Phoenix	Arizona	85016	United States

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Double Blind - Maralixibat	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
FG001	Double Blind - Placebo

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	May 10, 2023	Dec 20, 2024

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Mean Change in Total Serum Bilirubin Levels

Posted

Least Squares Mean

Standard Error

mg/dl

From baseline to Week 26

ID	Title	Description
OG000	Double Blind - Maralixibat	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
OG001	Double Blind - Placebo	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.

Units	Counts
Participants	OG00040 OG00135

Title

Denominators

Categories

Title	Measurements
	OG000-3.5± 0.853 OG001-3.11± 0.947

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000 OG001		MMRM		0.7419		Least-Square mean	-0.39	Standard Error of the Mean	1.182	2-Sided	95	-2.76	1.97					Superiority

Secondary

Mean Change in Total Serum Bile Acids

Posted

Least Squares Mean

Standard Error

umol/L

From baseline to Week 26

ID	Title	Description
OG000	Double Blind - Maralixibat	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
OG001	Double Blind - Placebo	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.

Units	Counts
Participants	OG000

Secondary

Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26

Posted

Count of Participants

Participants

From baseline to Week 26

ID	Title	Description
OG000	Double Blind - Maralixibat	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
OG001	Double Blind - Placebo	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.

Units	Counts
Participants	OG000

Secondary

Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death.

Liver decompensation (hepatic encephalopathy, variceal bleeding, new persistent ascites)

Posted

Count of Participants

Participants

From Baseline to Week 26

ID	Title	Description
OG000	Double Blind - Maralixibat	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
OG001	Double Blind - Placebo	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.

Units	Counts
Participants	OG000

Secondary

Proportion of Participants Undergoing Liver Transplantation or Death

Posted

Count of Participants

Participants

From Baseline to Week 26

ID	Title	Description
OG000	Double Blind - Maralixibat	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
OG001	Double Blind - Placebo	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.

Units	Counts
Participants	OG000

Secondary

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.

Splenomegaly => (spleen size >2 cm below the costal margin palpated on physical examination)

Posted

Count of Participants

Participants

From Baseline to Week 26

ID	Title	Description
OG000	Double Blind - Maralixibat	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
OG001	Double Blind - Placebo	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.

Units	Counts
Participants	OG000

Secondary

Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL

Posted

Count of Participants

Participants

From Baseline to Week 26

ID	Title	Description
OG000	Double Blind - Maralixibat	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
OG001	Double Blind - Placebo	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.

Units	Counts
Participants	OG000

Secondary

Proportion of Participants With Mean sBA Levels ≤40 mmol/L

Posted

Count of Participants

Participants

From Baseline to Week 26

ID	Title	Description
OG000	Double Blind - Maralixibat	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
OG001	Double Blind - Placebo	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.

Units	Counts
Participants	OG000

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Alanine aminotransferase increased	Investigations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0011 events1 affected35 at risk EG0020 events0 affected52 at risk
Hepatic enzyme increased	Investigations	MedDRA 26.1	Systematic Assessment	EG0002 events2 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Accidental exposure to product by child	Injury, poisoning and procedural complications	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Procedural complication	Injury, poisoning and procedural complications	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Coagulopathy	Blood and lymphatic system disorders	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0011 events1 affected35 at risk EG0020 events0 affected52 at risk
Pyrexia	General disorders	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0011 events1 affected35 at risk EG0021 events1 affected52 at risk
Systemic inflammatory response syndrome	General disorders	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Drug hypersensitivity	Immune system disorders	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 26.1	Systematic Assessment	EG0002 events2 affected40 at risk EG0010 events0 affected35 at risk EG0020 events0 affected52 at risk
Faeces discoloured	Gastrointestinal disorders	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0010 events0 affected35 at risk EG0020 events0 affected52 at risk
Gastrointestinal haemorrhage	Gastrointestinal disorders	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0010 events0 affected35 at risk EG0020 events0 affected52 at risk
Ileus	Gastrointestinal disorders	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Mouth ulceration	Gastrointestinal disorders	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0011 events1 affected35 at risk EG0020 events0 affected52 at risk
Stomatitis	Gastrointestinal disorders	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0010 events0 affected35 at risk EG0020 events0 affected52 at risk
Cholangitis	Hepatobiliary disorders	MedDRA 26.1	Systematic Assessment	EG00023 events15 affected40 at risk EG00125 events17 affected35 at risk EG00210 events8 affected52 at risk
Cholangitis acute	Hepatobiliary disorders	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0011 events1 affected35 at risk EG0020 events0 affected52 at risk
Drug-induced liver injury	Hepatobiliary disorders	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Hyperbilirubinaemia	Hepatobiliary disorders	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0011 events1 affected35 at risk EG0020 events0 affected52 at risk
Jaundice	Hepatobiliary disorders	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0010 events0 affected35 at risk EG0020 events0 affected52 at risk
Liver injury	Hepatobiliary disorders	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Urticaria	Skin and subcutaneous tissue disorders	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Adenovirus infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0022 events2 affected52 at risk
Bronchiolitis	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0002 events2 affected40 at risk EG0011 events1 affected35 at risk EG0021 events1 affected52 at risk
COVID-19	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0013 events3 affected35 at risk EG0020 events0 affected52 at risk
Cytomegalovirus infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0002 events1 affected40 at risk EG0010 events0 affected35 at risk EG0020 events0 affected52 at risk
Gastroenteritis	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0022 events2 affected52 at risk
Gastrointestinal infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0011 events1 affected35 at risk EG0021 events1 affected52 at risk
Otitis media	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Parainfluenzae virus infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0002 events2 affected40 at risk EG0010 events0 affected35 at risk EG0020 events0 affected52 at risk
Pertussis	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Pharyngitis	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0011 events1 affected35 at risk EG0020 events0 affected52 at risk
Pneumonia	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG00013 events8 affected40 at risk EG0015 events4 affected35 at risk EG0022 events2 affected52 at risk
Respiratory syncytial virus bronchiolitis	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0011 events1 affected35 at risk EG0020 events0 affected52 at risk
Respiratory syncytial virus infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0010 events0 affected35 at risk EG0020 events0 affected52 at risk
Respiratory tract infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0011 events1 affected35 at risk EG0020 events0 affected52 at risk
Rhinovirus infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0010 events0 affected35 at risk EG0020 events0 affected52 at risk
Rotavirus infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Sepsis	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0012 events2 affected35 at risk EG0020 events0 affected52 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Urinary tract infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Viral infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Hyperinsulinism	Metabolism and nutrition disorders	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0011 events1 affected35 at risk EG0020 events0 affected52 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Alanine aminotransferase increased	Investigations	MedDRA 26.1	Systematic Assessment	EG0005 events5 affected40 at risk EG0015 events5 affected35 at risk EG0026 events4 affected52 at risk
Blood bilirubin increased	Investigations	MedDRA 26.1	Systematic Assessment	EG0003 events1 affected40 at risk EG0012 events2 affected35 at risk EG0021 events1 affected52 at risk
Hepatic enzyme increased	Investigations	MedDRA 26.1	Systematic Assessment	EG0003 events3 affected40 at risk EG0010 events0 affected35 at risk EG0021 events1 affected52 at risk
Spleen palpable	Investigations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0012 events2 affected35 at risk EG0021 events1 affected52 at risk
Vitamin A decreased	Investigations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0012 events2 affected35 at risk EG0021 events1 affected52 at risk
Scratch	Injury, poisoning and procedural complications	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0012 events2 affected35 at risk EG0020 events0 affected52 at risk
Anaemia	Blood and lymphatic system disorders	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0015 events3 affected35 at risk EG0023 events3 affected52 at risk
Coagulopathy	Blood and lymphatic system disorders	MedDRA 26.1	Systematic Assessment	EG0003 events2 affected40 at risk EG0014 events3 affected35 at risk EG0023 events2 affected52 at risk
Splenomegaly	Blood and lymphatic system disorders	MedDRA 26.1	Systematic Assessment	EG0002 events2 affected40 at risk EG0011 events1 affected35 at risk EG0021 events1 affected52 at risk
Pyrexia	General disorders	MedDRA 26.1	Systematic Assessment	EG00019 events14 affected40 at risk EG0018 events8 affected35 at risk EG00218 events12 affected52 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 26.1	Systematic Assessment	EG00019 events13 affected40 at risk EG0018 events8 affected35 at risk EG00216 events13 affected52 at risk
Vomiting	Gastrointestinal disorders	MedDRA 26.1	Systematic Assessment	EG0004 events4 affected40 at risk EG0018 events6 affected35 at risk EG0022 events2 affected52 at risk
Dyspepsia	Gastrointestinal disorders	MedDRA 26.1	Systematic Assessment	EG0002 events2 affected40 at risk EG0010 events0 affected35 at risk EG0020 events0 affected52 at risk
Haematochezia	Gastrointestinal disorders	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0014 events2 affected35 at risk EG0020 events0 affected52 at risk
Cholangitis	Hepatobiliary disorders	MedDRA 26.1	Systematic Assessment	EG0008 events6 affected40 at risk EG0016 events6 affected35 at risk EG0023 events3 affected52 at risk
Hepatomegaly	Hepatobiliary disorders	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0012 events2 affected35 at risk EG0020 events0 affected52 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 26.1	Systematic Assessment	EG0009 events8 affected40 at risk EG0016 events5 affected35 at risk EG0029 events8 affected52 at risk
Dermatitis diaper	Skin and subcutaneous tissue disorders	MedDRA 26.1	Systematic Assessment	EG0006 events3 affected40 at risk EG0010 events0 affected35 at risk EG0028 events5 affected52 at risk
Eczema	Skin and subcutaneous tissue disorders	MedDRA 26.1	Systematic Assessment	EG0009 events6 affected40 at risk EG0014 events4 affected35 at risk EG0021 events1 affected52 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA 26.1	Systematic Assessment	EG0003 events3 affected40 at risk EG0013 events3 affected35 at risk EG0022 events2 affected52 at risk
Urticaria	Skin and subcutaneous tissue disorders	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0010 events0 affected35 at risk EG0024 events3 affected52 at risk
Pruritus	Skin and subcutaneous tissue disorders	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0012 events2 affected35 at risk EG0020 events0 affected52 at risk
Bronchitis	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0003 events3 affected40 at risk EG0013 events3 affected35 at risk EG0025 events4 affected52 at risk
COVID-19	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0003 events3 affected40 at risk EG0015 events5 affected35 at risk EG0022 events2 affected52 at risk
Nasopharyngitis	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0011 events1 affected35 at risk EG0027 events3 affected52 at risk
Otitis media	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0012 events1 affected35 at risk EG0023 events3 affected52 at risk
Pharyngitis	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0002 events2 affected40 at risk EG0011 events1 affected35 at risk EG0021 events1 affected52 at risk
Pneumonia	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0008 events6 affected40 at risk EG0018 events5 affected35 at risk EG0026 events5 affected52 at risk
Respiratory tract infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0012 events2 affected35 at risk EG0022 events2 affected52 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0008 events7 affected40 at risk EG0015 events3 affected35 at risk EG00223 events12 affected52 at risk
Viral upper respiratory tract infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0001 events1 affected40 at risk EG0015 events4 affected35 at risk EG0026 events4 affected52 at risk
Cytomegalovirus infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0002 events2 affected40 at risk EG0011 events1 affected35 at risk EG0020 events0 affected52 at risk
Gastroenteritis	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0023 events3 affected52 at risk
Influenza	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0000 events0 affected40 at risk EG0010 events0 affected35 at risk EG0023 events3 affected52 at risk
Parainfluenzae virus infection	Infections and infestations	MedDRA 26.1	Systematic Assessment	EG0002 events2 affected40 at risk EG0010 events0 affected35 at risk EG0020 events0 affected52 at risk
Vitamin A deficiency	Metabolism and nutrition disorders	MedDRA 26.1	Systematic Assessment	EG0003 events3 affected40 at risk EG0017 events7 affected35 at risk EG0023 events3 affected52 at risk
Vitamin D deficiency	Metabolism and nutrition disorders	MedDRA 26.1	Systematic Assessment	EG0007 events7 affected40 at risk EG0019 events9 affected35 at risk EG0028 events8 affected52 at risk
Vitamin E deficiency	Metabolism and nutrition disorders	MedDRA 26.1	Systematic Assessment	EG0002 events2 affected40 at risk EG0014 events4 affected35 at risk EG0024 events4 affected52 at risk
Zinc deficiency	Metabolism and nutrition disorders	MedDRA 26.1	Systematic Assessment	EG0005 events5 affected40 at risk EG0016 events5 affected35 at risk EG0022 events2 affected52 at risk

ID	Title	Description
BG000	Double Blind Period - Placebo	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment.
BG001	Double Blind - Maralixibat	The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment.
BG002	Total	Total of all reporting groups

ID	Term
D001656	Biliary Atresia
D001660	Biliary Tract Diseases
D001649	Bile Duct Diseases
D000013	Congenital Abnormalities

ID	Term
D004066	Digestive System Diseases
D004065	Digestive System Abnormalities
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

ID	Title	Description	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Double Blind - Maralixibat	All participants who receive at least 1 dose of study medication during the double-blinded Maralixibat Period.	40	26	40	38	40
EG001	Double Blind - Placebo	All participants who receive at least 1 dose of study medication during the double-blinded Placebo Period.	35	25	35	33	35
EG002	Open Label - Maralixibat	All participants who receive at least 1 dose of study medication during the Open-Label Maralixibat Period.	52	21	52	47	52