Trial Outcomes & Findings for An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC) (NCT NCT04185363)

NCT ID: NCT04185363

Last Updated: 2026-06-23

Results Overview

The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch)

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

84 participants

Primary outcome timeframe

From Baseline to Weeks 75-78

Results posted on

2026-06-23

Participant Flow

A total of 84 participants were enrolled at 28 sites across 16 countries (Argentina, Austria, Belgium, Brazil, Canada, Colombia, France, Germany, Italy, Lebanon, Mexico, Poland, Singapore, Turkey, United Kingdom, United States)

MRX-503, screening starts after signing the ICF and confirming eligibility. It overlaps with the MRX-502 end-of-treatment (EOT) visit: if the MRX-503 baseline visit occurs the same day or within 30 days of the MRX-502 EOT visit, those prior evaluations count as the MRX-503 baseline assessments.

Participant milestones

Participant milestones
Measure	Maralixibat All participants were assigned to a single treatment arm and received maralixibat oral solution. Participants underwent an initial dose-titration period (4-6 weeks), during which maralixibat doses were escalated within participants from 150 microgram per kilogram (mcg/kg) twice daily up to a maximum tolerated dose of 600 mcg/kg twice daily, followed by a stable-dosing period and safety follow-up period. Subjects were analyzed in two cohorts: * The Primary Cohort (non-truncated PFIC2) * The PFIC Cohort (other PFIC subtypes, including PFIC1, PFIC3, truncated PFIC2, or incomplete response after Partial external biliary diversion)
Overall Study STARTED	84
Overall Study COMPLETED	45
Overall Study NOT COMPLETED	39

Reasons for withdrawal

Reasons for withdrawal
Measure	Maralixibat All participants were assigned to a single treatment arm and received maralixibat oral solution. Participants underwent an initial dose-titration period (4-6 weeks), during which maralixibat doses were escalated within participants from 150 microgram per kilogram (mcg/kg) twice daily up to a maximum tolerated dose of 600 mcg/kg twice daily, followed by a stable-dosing period and safety follow-up period. Subjects were analyzed in two cohorts: * The Primary Cohort (non-truncated PFIC2) * The PFIC Cohort (other PFIC subtypes, including PFIC1, PFIC3, truncated PFIC2, or incomplete response after Partial external biliary diversion)
Overall Study Withdrawal by Subject	7
Overall Study Physician Decision	3
Overall Study Adverse Event	8
Overall Study Liver Transplant	16
Overall Study Lost to Follow-up	1
Overall Study Disease progression	2
Overall Study Rolled over under drug interruption	1
Overall Study Initiation/Dose Increase of Medication to treat Pruritus or Cholestatic Liver Disease	1

Baseline Characteristics

An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Maralixibat n=84 Participants This open-label, extension study comprised one continuous treatment period, including: * Dose-escalation (4-6 weeks) * Stable-dosing (until the sponsor or the subject terminates the study, discontinues/ transitions to commercial product) * Safety follow-up phases. Subjects were analyzed in two cohorts: * The Primary Cohort (non-truncated PFIC2) * The PFIC Cohort (other PFIC subtypes, including PFIC1, PFIC3, truncated PFIC2, or incomplete response after Partial external biliary diversion)
Age, Customized Infants and toddlers (28 days - 23 months)	18 Participants n=20 Participants
Age, Customized Children (2-11 years)	62 Participants n=20 Participants
Age, Customized Adolescents (12-17 years)	4 Participants n=20 Participants
Sex: Female, Male Female	48 Participants n=20 Participants
Sex: Female, Male Male	36 Participants n=20 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	33 Participants n=20 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	48 Participants n=20 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=20 Participants
Region of Enrollment Colombia	5 participants n=20 Participants
Region of Enrollment Argentina	3 participants n=20 Participants
Region of Enrollment Singapore	2 participants n=20 Participants
Region of Enrollment United States	18 participants n=20 Participants
Region of Enrollment United Kingdom	2 participants n=20 Participants
Region of Enrollment Lebanon	13 participants n=20 Participants
Region of Enrollment Canada	2 participants n=20 Participants
Region of Enrollment Austria	2 participants n=20 Participants
Region of Enrollment Turkey	2 participants n=20 Participants
Region of Enrollment Belgium	2 participants n=20 Participants
Region of Enrollment Poland	5 participants n=20 Participants
Region of Enrollment Brazil	10 participants n=20 Participants
Region of Enrollment Mexico	8 participants n=20 Participants
Region of Enrollment Italy	5 participants n=20 Participants
Region of Enrollment France	3 participants n=20 Participants
Region of Enrollment Germany	2 participants n=20 Participants

PRIMARY outcome

Timeframe: From Baseline to Weeks 75-78

Population: Change from baseline values were calculated only for participants with both a non-missing baseline value and a non-missing post-baseline value at the specified visit/time period. Participants without a post-baseline assessment at that visit were not included in the analysis for that time point. Therefore, the number of participants analyzed varies by visit and may be less than the total number in the analysis population.

The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch)

Outcome measures

Outcome measures
Measure	The Primary Cohort n=21 Participants The Primary Cohort in MRX-503 includes subjects with non-truncated PFIC2 (nt-PFIC2) mutations previously enrolled in MRX-502.	PFIC Cohort n=41 Participants The PFIC (All PFIC) Cohort in MRX-503 combines the nt-PFIC2 primary cohort with other PFIC types to evaluate safety and efficacy across the wider PFIC population.
Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Severity Score	-1.633 score on a scale Standard Deviation 1.4416	-1.602 score on a scale Standard Deviation 1.3649

SECONDARY outcome

Timeframe: Week 15 - 26

Maintenance of treatment effect is defined as the proportion of participants in the MRX-MRX treatment group (MRX-502 and MRX-503 data for subjects on MRX in the MRX-502 study) who obtain an ItchRO (Obs) response from Week 15 to Week 26 in Study MRX-503 in the primary cohort and PFIC cohort.

Outcome measures

Outcome measures
Measure	The Primary Cohort n=14 Participants The Primary Cohort in MRX-503 includes subjects with non-truncated PFIC2 (nt-PFIC2) mutations previously enrolled in MRX-502.	PFIC Cohort n=33 Participants The PFIC (All PFIC) Cohort in MRX-503 combines the nt-PFIC2 primary cohort with other PFIC types to evaluate safety and efficacy across the wider PFIC population.
Maintenance of ItchRO(Obs) Response (Weeks 15 - 26) Week 15-18	71.4 percentage of participants	69.7 percentage of participants
Maintenance of ItchRO(Obs) Response (Weeks 15 - 26) Week 19-22	64.3 percentage of participants	72.7 percentage of participants
Maintenance of ItchRO(Obs) Response (Weeks 15 - 26) Week 23-26	64.3 percentage of participants	69.7 percentage of participants

SECONDARY outcome

Timeframe: Baseline to EOT

Proportion of ItchRO responders over time at each study visit using the 4-week study period prior to the visit as per ItchRO(Obs) responder definition.

Outcome measures

Outcome measures
Measure	The Primary Cohort n=28 Participants The Primary Cohort in MRX-503 includes subjects with non-truncated PFIC2 (nt-PFIC2) mutations previously enrolled in MRX-502.	PFIC Cohort n=60 Participants The PFIC (All PFIC) Cohort in MRX-503 combines the nt-PFIC2 primary cohort with other PFIC types to evaluate safety and efficacy across the wider PFIC population.
Proportion of ItchRO(Obs) Responders Over Time Week 63-66	57.1 percentage of participants	58.3 percentage of participants
Proportion of ItchRO(Obs) Responders Over Time Week 75-78	57.1 percentage of participants	53.3 percentage of participants
Proportion of ItchRO(Obs) Responders Over Time Week 87-90	50 percentage of participants	48.3 percentage of participants
Proportion of ItchRO(Obs) Responders Over Time Week 99-102	57.1 percentage of participants	46.7 percentage of participants
Proportion of ItchRO(Obs) Responders Over Time Week 111-114	21.4 percentage of participants	26.7 percentage of participants
Proportion of ItchRO(Obs) Responders Over Time Week 123-126	14.3 percentage of participants	20 percentage of participants
Proportion of ItchRO(Obs) Responders Over Time Week 23-26	64.3 percentage of participants	63.3 percentage of participants
Proportion of ItchRO(Obs) Responders Over Time Week 39-42	78.6 percentage of participants	71.7 percentage of participants
Proportion of ItchRO(Obs) Responders Over Time Week 51-54	71.4 percentage of participants	66.7 percentage of participants
Proportion of ItchRO(Obs) Responders Over Time Week 1-6	57.1 percentage of participants	50 percentage of participants
Proportion of ItchRO(Obs) Responders Over Time Week 7-10	67.9 percentage of participants	65 percentage of participants
Proportion of ItchRO(Obs) Responders Over Time Week 11-14	67.9 percentage of participants	63.3 percentage of participants
Proportion of ItchRO(Obs) Responders Over Time Week 15-18	75 percentage of participants	66.7 percentage of participants
Proportion of ItchRO(Obs) Responders Over Time Week 19-22	67.9 percentage of participants	68.3 percentage of participants

SECONDARY outcome

Timeframe: From Baseline to Weeks 75-78

The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch)

Outcome measures

Outcome measures
Measure	The Primary Cohort n=21 Participants The Primary Cohort in MRX-503 includes subjects with non-truncated PFIC2 (nt-PFIC2) mutations previously enrolled in MRX-502.	PFIC Cohort n=41 Participants The PFIC (All PFIC) Cohort in MRX-503 combines the nt-PFIC2 primary cohort with other PFIC types to evaluate safety and efficacy across the wider PFIC population.
Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Frequency Score	-1.644 score on a scale Standard Deviation 1.4451	-1.608 score on a scale Standard Deviation 1.3848

SECONDARY outcome

Timeframe: Baseline to week 70

Mean change from baseline over time in total serum bile acid (sBA) levels

Outcome measures

Outcome measures
Measure	The Primary Cohort n=20 Participants The Primary Cohort in MRX-503 includes subjects with non-truncated PFIC2 (nt-PFIC2) mutations previously enrolled in MRX-502.	PFIC Cohort n=44 Participants The PFIC (All PFIC) Cohort in MRX-503 combines the nt-PFIC2 primary cohort with other PFIC types to evaluate safety and efficacy across the wider PFIC population.
Mean Change From Baseline Over Time in Total Serum Bile Acid (sBA) Levels	-150.064 umol/L Standard Deviation 213.7437	-111.473 umol/L Standard Deviation 188.1880

SECONDARY outcome

Timeframe: From Week 18 to Week 26

Proportion of subjects who experience an sBA control over time from Week 18 to Week 26

Outcome measures

Outcome measures
Measure	The Primary Cohort n=28 Participants The Primary Cohort in MRX-503 includes subjects with non-truncated PFIC2 (nt-PFIC2) mutations previously enrolled in MRX-502.	PFIC Cohort n=60 Participants The PFIC (All PFIC) Cohort in MRX-503 combines the nt-PFIC2 primary cohort with other PFIC types to evaluate safety and efficacy across the wider PFIC population.
Proportion of Subjects Who Experience an sBA Control Over Time From Week 18 to Week 26 Week 18	42.9 percentage of participants	40 percentage of participants
Proportion of Subjects Who Experience an sBA Control Over Time From Week 18 to Week 26 Week 22	42.9 percentage of participants	38.3 percentage of participants
Proportion of Subjects Who Experience an sBA Control Over Time From Week 18 to Week 26 Week 26	46.4 percentage of participants	43.3 percentage of participants
Proportion of Subjects Who Experience an sBA Control Over Time From Week 18 to Week 26 Average of Weeks 18, 22, 26	42.9 percentage of participants	40 percentage of participants

SECONDARY outcome

Timeframe: From baseline to Week 70

Height-for-age Z-score measures a participant's height relative to a reference population of children of the same age and sex. Z-scores were derived using World Health Organization growth charts for participants less than 24 months of age and Centers for Disease Control and Prevention growth charts for participants 24 months of age or older. A Z-score of 0 represents the population mean for age and sex. Positive Z-scores indicate height above the reference population mean, and negative Z-scores indicate height below the reference population mean. Higher Z-scores generally indicate greater linear growth relative to the reference population. Change from baseline was calculated as post-baseline height-for-age Z-score minus baseline height-for-age Z-score.

Outcome measures

Outcome measures
Measure	The Primary Cohort n=22 Participants The Primary Cohort in MRX-503 includes subjects with non-truncated PFIC2 (nt-PFIC2) mutations previously enrolled in MRX-502.	PFIC Cohort n=47 Participants The PFIC (All PFIC) Cohort in MRX-503 combines the nt-PFIC2 primary cohort with other PFIC types to evaluate safety and efficacy across the wider PFIC population.
Change From Baseline in Height Z-score	0.486 Z-Score Standard Deviation 0.6665	0.335 Z-Score Standard Deviation 0.6229

SECONDARY outcome

Timeframe: From Baseline to Week 70

Weight-for-age Z-score measures a participant's weight relative to a reference population of children of the same age and sex. Z-scores were derived using World Health Organization growth charts for participants less than 24 months of age and Centers for Disease Control and Prevention growth charts for participants 24 months of age or older. A Z-score of 0 represents the population mean for age and sex. Positive Z-scores indicate weight above the reference population mean, and negative Z-scores indicate weight below the reference population mean. Higher Z-scores generally indicate greater body weight relative to the reference population. Change from baseline was calculated as post-baseline weight-for-age Z-score minus baseline weight-for-age Z-score.

Outcome measures

Outcome measures
Measure	The Primary Cohort n=21 Participants The Primary Cohort in MRX-503 includes subjects with non-truncated PFIC2 (nt-PFIC2) mutations previously enrolled in MRX-502.	PFIC Cohort n=46 Participants The PFIC (All PFIC) Cohort in MRX-503 combines the nt-PFIC2 primary cohort with other PFIC types to evaluate safety and efficacy across the wider PFIC population.
Change From Baseline in Weight Z-score	0.518 Z-Score Standard Deviation 0.9349	0.330 Z-Score Standard Deviation 0.7421

Adverse Events

Maralixibat

Serious events: 25 serious events

Other events: 84 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Maralixibat n=84 participants at risk All subjects will receive Maralixibat oral solution Maralixibat: All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram \[mcg/kg\]) twice daily
Gastrointestinal disorders Oesophageal varices haemorrhage	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Gastroenteritis	4.8% 4/84 • Number of events 5 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Gastroenteritis rotavirus	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Congenital, familial and genetic disorders Progressive familial intrahepatic cholestasis	2.4% 2/84 • Number of events 2 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Gastrointestinal disorders Diarrhoea	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Gastrointestinal disorders Haematochezia	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
General disorders and administration site conditions Disease progression	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
General disorders and administration site conditions Oedema peripheral	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Hepatobiliary disorders Biliary colic	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Hepatobiliary disorders Cholangitis	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Hepatobiliary disorders Cholecystitis acute	2.4% 2/84 • Number of events 2 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Hepatobiliary disorders Cholelithiasis	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Hepatobiliary disorders Hepatic function abnormal	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Hepatobiliary disorders Jaundice	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Adenovirus infection	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Bacterial translocation	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Corona virus infection	2.4% 2/84 • Number of events 2 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Enterovirus infection	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Escherichia urinary tract infection	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Gastroenteritis viral	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Hepatitis viral	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Nasopharyngitis	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Rhinovirus infection	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Septic shock	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Tonsillitis	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Urinary tract infection	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Viral infection	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Injury, poisoning and procedural complications Accidental exposure to product	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Injury, poisoning and procedural complications Accidental overdose	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Injury, poisoning and procedural complications Femur fracture	1.2% 1/84 • Number of events 2 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Injury, poisoning and procedural complications Head injury	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Injury, poisoning and procedural complications Postoperative renal failure	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Injury, poisoning and procedural complications Stoma site haemorrhage	1.2% 1/84 • Number of events 3 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Metabolism and nutrition disorders Dehydration	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Respiratory, thoracic and mediastinal disorders Epistaxis	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Respiratory, thoracic and mediastinal disorders Nasal obstruction	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Respiratory, thoracic and mediastinal disorders Respiratory distress	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Skin and subcutaneous tissue disorders Pruritus	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Surgical and medical procedures Biliary tract operation	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Vascular disorders Haematoma	1.2% 1/84 • Number of events 1 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.

Other adverse events

Other adverse events
Measure	Maralixibat n=84 participants at risk All subjects will receive Maralixibat oral solution Maralixibat: All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram \[mcg/kg\]) twice daily
Blood and lymphatic system disorders Anaemia	8.3% 7/84 • Number of events 9 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Blood and lymphatic system disorders Coagulopathy	4.8% 4/84 • Number of events 10 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Gastrointestinal disorders Abdominal pain	27.4% 23/84 • Number of events 37 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Gastrointestinal disorders Abdominal pain upper	6.0% 5/84 • Number of events 6 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Gastrointestinal disorders Constipation	9.5% 8/84 • Number of events 9 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Gastrointestinal disorders Dental caries	4.8% 4/84 • Number of events 5 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Gastrointestinal disorders Diarrhoea	46.4% 39/84 • Number of events 82 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Gastrointestinal disorders Vomiting	15.5% 13/84 • Number of events 20 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
General disorders and administration site conditions Influenza like illness	4.8% 4/84 • Number of events 4 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
General disorders and administration site conditions Pyrexia	47.6% 40/84 • Number of events 77 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Hepatobiliary disorders Cholelithiasis	6.0% 5/84 • Number of events 6 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Hepatobiliary disorders Jaundice	7.1% 6/84 • Number of events 8 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Conjunctivitis	7.1% 6/84 • Number of events 7 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Corona virus infection	14.3% 12/84 • Number of events 12 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Ear infection	13.1% 11/84 • Number of events 12 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Gastroenteritis	13.1% 11/84 • Number of events 17 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Gastrointestinal viral infection	4.8% 4/84 • Number of events 4 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Influenza	19.0% 16/84 • Number of events 23 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Nasopharyngitis	25.0% 21/84 • Number of events 45 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Otitis media	10.7% 9/84 • Number of events 10 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Otitis media acute	7.1% 6/84 • Number of events 9 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Pharyngitis	9.5% 8/84 • Number of events 11 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Respiratory tract infection	6.0% 5/84 • Number of events 6 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Rhinitis	10.7% 9/84 • Number of events 18 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Tonsillitis	7.1% 6/84 • Number of events 8 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Upper respiratory tract infection	19.0% 16/84 • Number of events 53 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Viral infection	8.3% 7/84 • Number of events 9 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Infections and infestations Viral upper respiratory tract infection	4.8% 4/84 • Number of events 4 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Injury, poisoning and procedural complications Skin abrasion	6.0% 5/84 • Number of events 5 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Investigations Activated partial thromboplastin time prolonged	6.0% 5/84 • Number of events 9 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Investigations Alanine aminotransferase increased	16.7% 14/84 • Number of events 27 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Investigations Aspartate aminotransferase increased	7.1% 6/84 • Number of events 8 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Investigations Blood bilirubin increased	19.0% 16/84 • Number of events 23 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Investigations International normalised ratio increased	13.1% 11/84 • Number of events 14 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Investigations Prothrombin time prolonged	4.8% 4/84 • Number of events 5 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Investigations Vitamin A increased	6.0% 5/84 • Number of events 5 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Investigations Vitamin D decreased	16.7% 14/84 • Number of events 28 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Investigations Vitamin E decreased	10.7% 9/84 • Number of events 12 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Metabolism and nutrition disorders Vitamin A deficiency	7.1% 6/84 • Number of events 6 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Metabolism and nutrition disorders Vitamin D deficiency	11.9% 10/84 • Number of events 10 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Metabolism and nutrition disorders Vitamin E deficiency	4.8% 4/84 • Number of events 5 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Nervous system disorders Headache	7.1% 6/84 • Number of events 9 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Respiratory, thoracic and mediastinal disorders Cough	33.3% 28/84 • Number of events 61 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Respiratory, thoracic and mediastinal disorders Epistaxis	9.5% 8/84 • Number of events 31 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Respiratory, thoracic and mediastinal disorders Nasal congestion	6.0% 5/84 • Number of events 7 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	7.1% 6/84 • Number of events 8 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	14.3% 12/84 • Number of events 16 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Skin and subcutaneous tissue disorders Pruritus	25.0% 21/84 • Number of events 35 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Skin and subcutaneous tissue disorders Rash	4.8% 4/84 • Number of events 4 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.
Skin and subcutaneous tissue disorders Urticaria	6.0% 5/84 • Number of events 6 • From the first dose through 14 days after the last dose of maralixibat, up to approximately 260 weeks. Adverse events were systematically collected from all participants at each scheduled visit and through continuous monitoring from first dose to 14 days after last dose. Investigators were required to actively elicit and record all new or worsening events, with assessment of severity, relationship, and outcome.

Additional Information

Mirum Clinical Trials

Mirum Pharmaceuticals

Phone: 650-667-4085

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60