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| Name | Class |
|---|---|
| Childhood Liver Disease Research and Education Network | OTHER |
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The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LUM001 | Experimental | LUM001 for oral administration |
|
| Placebo | Placebo Comparator | Placebo administered orally once each day |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LUM001 | Drug | LUM001 administered orally |
| |
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus | Pruritus was assessed using Itch report outcome measure (ItchRO[Obs]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period. | Baseline, Week 13/Early Termination |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level | Fasting sBA level was measured by using a liquid chromatography mass spectrometry method. | Baseline, Week 13/Early Termination |
| Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | An AE was any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life -threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.TEAEs were defined as AEs/SAEs that started or worsened after the study drug treatment. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Mirum | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States | ||
| University of California at San Francisco |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 42002216 | Derived | Hoskins BJ, Mogul DB, Chen C, Karnsakul W. Maralixibat Improves Xanthomas and Hypercholesterolemia in Children with Alagille Syndrome: A Post Hoc Integrated Analysis From Two Clinical Trials. J Pediatr. 2026 Jul;294:115108. doi: 10.1016/j.jpeds.2026.115108. Epub 2026 Apr 17. |
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Overall, 37 participants were enrolled to dose escalation phase started with 14 microgram per kilogram per day (mcg/kg/day) per week on Week 1 up to 280 mcg/kg/day for a maximum up to 5 weeks, until they reached the planned stable dose of 70, 140 and 280 mcg/kg/day and continued up to Week 13.
Study was conducted at 13 study centers in the United States and Canada, between 24 November 2014 (first participant first visit) and 16 November 2016 (last participant last visit).
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| ID | Title | Description |
|---|---|---|
| FG000 | Maralixibat 70 mcg/kg/Day | Participants received low dose maralixibat 70 mcg/kg/day oral solution for 10 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35 and 70 mcg/kg/day dose per each week respectively up to 3 weeks. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
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| Drug |
Placebo administered orally |
|
Liver enzyme levels of alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase and gamma glutamyl transferase were reported here. |
| Baseline, Week 13/Early Termination |
| Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations | Liver enzyme levels of total bilirubin and direct bilirubin were reported here. | Baseline, Week 13/Early Termination |
| From the start of study drug administration up to Week 17 |
| San Francisco |
| California |
| 94143 |
| United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30322 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Riley Hospital for Children | Indianapolis | Indiana | 46202 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| Baylor College of Medicine/Texas Children's Hospital | Houston | Texas | 77030 | United States |
| University of Utah | Salt Lake City | Utah | 84113 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| The Hospital for Sick Children | Toronto | Ontario | M5G 1X8 | Canada |
| Maralixibat 140 mcg/kg/Day |
Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks. |
| FG002 | Maralixibat 280 mcg/kg/Day | Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks. |
| FG003 | Placebo | Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period). |
| COMPLETED |
|
| NOT COMPLETED |
|
|
The Safety Population included all participants who were randomly assigned to study treatment and received at least one dose of the study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Maralixibat 70 mcg/kg/Day | Participants received low dose maralixibat 70 mcg/kg/day oral solution for 10 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35 and 70 mcg/kg/day dose per each week respectively up to 3 weeks. |
| BG001 | Maralixibat 140 mcg/kg/Day | Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks. |
| BG002 | Maralixibat 280 mcg/kg/Day | Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks. |
| BG003 | Placebo | Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period). |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
|
| 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 | Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus | Pruritus was assessed using Itch report outcome measure (ItchRO[Obs]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period. | Modified Intent-to-Treat Population (mITT) population included all participants randomly assigned to study treatment, receiving at least 1 dose of treatment, and having at least 1 post baseline observer itch reported outcome (ItchRO[Obs]) average daily score. | Posted | Least Squares Mean | Standard Error | Score on a scale | Baseline, Week 13/Early Termination |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level | Fasting sBA level was measured by using a liquid chromatography mass spectrometry method. | mITT population included all participants randomly assigned to study treatment, receiving at least 1 dose of treatment, and having at least 1 post baseline observer itch reported outcome (ItchRO[Obs]) average daily score. | Posted | Least Squares Mean | Standard Error | Micro moles per liter (mcmol/L) | Baseline, Week 13/Early Termination |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels | Liver enzyme levels of alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase and gamma glutamyl transferase were reported here. | mITT population included all participants randomly assigned to study treatment, receiving at least 1 dose of treatment, and having at least 1 post baseline observer itch reported outcome (ItchRO[Obs]) average daily score. | Posted | Least Squares Mean | Standard Error | Unit per liter (U/L) | Baseline, Week 13/Early Termination |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations | Liver enzyme levels of total bilirubin and direct bilirubin were reported here. | mITT population included all participants randomly assigned to study treatment, receiving at least 1 dose of treatment, and having at least 1 post baseline observer itch reported outcome (ItchRO[Obs]) average daily score. | Posted | Least Squares Mean | Standard Error | Milligrams per deciliter (mg/dL) | Baseline, Week 13/Early Termination |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | An AE was any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life -threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.TEAEs were defined as AEs/SAEs that started or worsened after the study drug treatment. | The Safety Population included all participants who were randomly assigned to study treatment and received at least one dose of the study drug. | Posted | Count of Participants | Participants | From the start of study drug administration up to Week 17 |
|
From the start of study drug administration up to Week 17
One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Maralixibat 14 mcg/kg/Day | Participants received 14 mcg/kg/day of maralixibat oral solution for 1 week. One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant using this arm. | 0 | 1 | 0 | 1 | 1 | 1 |
| EG001 | Maralixibat 70 mcg/kg/Day | Participants received low dose maralixibat 70 mcg/kg/day oral solution for 10 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35 and 70 mcg/kg/day dose per each week respectively up to 3 weeks. | 0 | 7 | 1 | 7 | 7 | 7 |
| EG002 | Maralixibat 140 mcg/kg/Day | Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks. | 0 | 11 | 0 | 11 | 9 | 11 |
| EG003 | Maralixibat 280 mcg/kg/Day | Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks. | 0 | 6 | 0 | 6 | 5 | 6 |
| EG004 | Placebo | Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period). | 0 | 12 | 0 | 12 | 12 | 12 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vomiting | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Growing pains | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Pallor | Vascular disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Eye pain | Eye disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Eye swelling | Eye disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Haematemesis | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Lip swelling | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Tooth crowding | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Irritability | General disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Left ventricular hypertrophy | Cardiac disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Acarodermatitis | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Herpes simplex | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Pertussis | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Overdose | Injury, poisoning and procedural complications | MedDRA (16.0) | Non-systematic Assessment |
| |
| Acoustic stimulation tests abnormal | Investigations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Blood bicarbonate decreased | Investigations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Blood pressure systolic increased | Investigations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Gamma-Glutamyltransferase increased | Investigations | MedDRA (16.0) | Non-systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA (16.0) | Non-systematic Assessment |
| |
| Enuresis | Renal and urinary disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Vitamin e deficiency | Metabolism and nutrition disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Convulsion | Nervous system disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Peripheral sensory neuropathy | Nervous system disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Mood altered | Psychiatric disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA (16.0) | Non-systematic Assessment |
|
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Physician | Mirum | 1 650-667-4085 | medinfo@mirumpharma.com |
| ID | Term |
|---|---|
| D016738 | Alagille Syndrome |
| ID | Term |
|---|---|
| D002780 | Cholestasis, Intrahepatic |
| D002779 | Cholestasis |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System Diseases |
| D008107 | Liver Diseases |
| D006330 | Heart Defects, Congenital |
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
Not provided
Not provided
| Male |
|
| The primary analysis of change from baseline to endpoint (Week 13/ET) average daily ItchRO(Obs) was evaluated by ANCOVA using a PROC MIXED procedure. LS mean change from baseline to Endpoint (Week 13/ET), along with associated 95% CI for the LS mean, and pair-wise treatment P-values were calculated for each treatment group. | ANCOVA | 0.0145 | LS mean difference | -0.906 | Standard Error of the Mean | 0.3503 | 2-Sided | 95 | -1.620 | -0.192 | Superiority |
| The primary analysis of change from baseline to endpoint (Week 13/ET) average daily ItchRO(Obs) was evaluated by ANCOVA using a PROC MIXED procedure. LS mean change from baseline to Endpoint (Week 13/ET), along with associated 95% confidence interval (CI) for the LS mean, and pair-wise treatment P-values were calculated for each treatment group. | ANCOVA | 0.9298 | LS mean difference | -0.039 | Standard Error of the Mean | 0.4431 | 2-Sided | 95 | -0.942 | 0.863 | Superiority |
Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks.
| OG003 | Placebo | Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period). |
|
|
Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks.
| OG003 | Placebo | Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period). |
|
|
Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks.
| OG003 | Placebo | Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period). |
|
|
| OG002 | Maralixibat 140 mcg/kg/Day | Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks. |
| OG003 | Maralixibat 280 mcg/kg/Day | Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks. |
| OG004 | Placebo | Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period). |
|
|