Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
The study will be conducted at multiple sites in North America, Europe, Asia, and South America.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Maralixibat | Experimental | All subjects will receive Maralixibat oral solution |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Maralixibat | Drug | All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) twice daily |
|
| Measure | Description | Time Frame |
|---|---|---|
| Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Severity Score | The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch) | From Baseline to Weeks 75-78 |
| Measure | Description | Time Frame |
|---|---|---|
| Maintenance of ItchRO(Obs) Response (Weeks 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. | Week 15 - 26 |
Not provided
Key Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States | ||
| Medstar Georgetown University Hospital |
Not provided
| Label | URL |
|---|---|
| Genetics Home Reference - PFIC | View source |
| US FDA Resources | View source |
| Mirum Pharmaceuticals homepage |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
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)
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | 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:
|
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 18, 2021 | Feb 12, 2026 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Proportion of ItchRO(Obs) Responders Over Time | 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. | Baseline to EOT |
| Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Frequency Score | The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch) | From Baseline to Weeks 75-78 |
| Mean Change From Baseline Over Time in Total Serum Bile Acid (sBA) Levels | Mean change from baseline over time in total serum bile acid (sBA) levels | Baseline to week 70 |
| Proportion of Subjects Who Experience an sBA Control Over Time From Week 18 to Week 26 | Proportion of subjects who experience an sBA control over time from Week 18 to Week 26 | From Week 18 to Week 26 |
| Change From Baseline in Height Z-score | 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. | From baseline to Week 70 |
| Change From Baseline in Weight Z-score | 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. | From Baseline to Week 70 |
| Washington D.C. |
| District of Columbia |
| 20007 |
| United States |
| Advent Health | Orlando | Florida | 32803 | United States |
| Children's Hospital at Montefiore | The Bronx | New York | 10461 | United States |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229 | United States |
| Children Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| University of Texas, Health Science Center San Antonio | San Antonio | Texas | 78229 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Hospital Italiano de Buenos Aires | Buenos Aires | Argentina |
| Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde | Vienna | Austria |
| Cliniques Universitaires Saint-Luc | Brussels | Belgium |
| Sociedade Beneficente de Senhoras Hospital Sírio-Libanês | São Paulo | Brazil |
| University of Alberta - Women and Children's Health Research Institute | Edmonton | Alberta | Canada |
| Fundacion Cardioinfantil | Bogotá | Colombia |
| Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon | Lyon | France |
| CHU de Toulouse - Hôpital des Enfants | Toulouse | 31059 | France |
| Medizinische Hochschule | Hanover | Germany |
| Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria | Bergamo | 24127 | Italy |
| Ospedale Pediatrico bambino Gesu' | Roma | Italy |
| Hotel Dieu de France, Alfred Naccache | Beirut | Lebanon |
| Consultario de Joshue David Covarrubias Esquer | Zapopan | Mexico |
| Instytut Pomnik Centrum, Zdrowia Dziecka | Warsaw | Poland |
| KK Women's and Children's Hospital | Singapore | Singapore |
| Koc University Hospital | Istanbul | 34010 | Turkey (Türkiye) |
| Birmingham Children's Hospital | Birmingham | United Kingdom |
| King's College Hospital NHS Foundation Trust | London | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Maralixibat | This open-label, extension study comprised one continuous treatment period, including:
Subjects were analyzed in two cohorts:
|
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||||||||
| Region of Enrollment | Number | 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 | Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Severity Score | The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch) | 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. | Posted | Mean | Standard Deviation | score on a scale | From Baseline to Weeks 75-78 |
|
|
| ||||||||||||||||||||||||||||
| Secondary | Maintenance of ItchRO(Obs) Response (Weeks 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. | Posted | Number | percentage of participants | Week 15 - 26 |
|
| |||||||||||||||||||||||||||||||
| Secondary | Proportion of ItchRO(Obs) Responders Over Time | 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. | Posted | Number | percentage of participants | Baseline to EOT |
|
| |||||||||||||||||||||||||||||||
| Secondary | Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Frequency Score | The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch) | 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. | Posted | Mean | Standard Deviation | score on a scale | From Baseline to Weeks 75-78 |
|
| |||||||||||||||||||||||||||||
| Secondary | Mean Change From Baseline Over Time in Total Serum Bile Acid (sBA) Levels | Mean change from baseline over time in total serum bile acid (sBA) levels | 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. | Posted | Mean | Standard Deviation | umol/L | Baseline to week 70 |
|
| |||||||||||||||||||||||||||||
| Secondary | Proportion of Subjects Who Experience an sBA Control Over Time From Week 18 to Week 26 | Proportion of subjects who experience an sBA control over time from Week 18 to Week 26 | Posted | Number | percentage of participants | From Week 18 to Week 26 |
|
| |||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Height Z-score | 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. | 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. | Posted | Mean | Standard Deviation | Z-Score | From baseline to Week 70 |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Weight Z-score | 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. | 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. | Posted | Mean | Standard Deviation | Z-Score | From Baseline to Week 70 |
|
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.
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 | 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 | 2 | 84 | 25 | 84 | 84 | 84 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Progressive familial intrahepatic cholestasis | Congenital, familial and genetic disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Oesophageal varices haemorrhage | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Disease progression | General disorders and administration site conditions | MedDRA 22.1 | Systematic Assessment |
| |
| Oedema peripheral | General disorders and administration site conditions | MedDRA 22.1 | Systematic Assessment |
| |
| Biliary colic | Hepatobiliary disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Cholangitis | Hepatobiliary disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Hepatic function abnormal | Hepatobiliary disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Jaundice | Hepatobiliary disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Adenovirus infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Bacterial translocation | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Corona virus infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Enterovirus infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Escherichia urinary tract infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Gastroenteritis rotavirus | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Hepatitis viral | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Accidental exposure to product | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Accidental overdose | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Postoperative renal failure | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Stoma site haemorrhage | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Nasal obstruction | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Biliary tract operation | Surgical and medical procedures | MedDRA 22.1 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA 22.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Coagulopathy | Blood and lymphatic system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Influenza like illness | General disorders and administration site conditions | MedDRA 22.1 | Systematic Assessment |
| |
| Pyrexia | General disorders and administration site conditions | MedDRA 22.1 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Jaundice | Hepatobiliary disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Corona virus infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Gastrointestinal viral infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Prothrombin time prolonged | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Vitamin A increased | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Vitamin D decreased | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Vitamin E decreased | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Vitamin A deficiency | Metabolism and nutrition disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Vitamin E deficiency | Metabolism and nutrition disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Mirum Clinical Trials | Mirum Pharmaceuticals | 650-667-4085 | medinfo@mirumpharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 12, 2022 | Feb 12, 2026 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| C535933 | Cholestasis, progressive familial intrahepatic 1 |
| D002779 | Cholestasis |
| D001649 | Bile Duct Diseases |
| D008107 | Liver Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System Diseases |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| C000722912 | maralixibat |
Not provided
Not provided
Not provided
| Title | Measurements |
|---|---|
|
| Unknown or Not Reported |
|
| Singapore |
|
| United States |
|
| United Kingdom |
|
| Lebanon |
|
| Canada |
|
| Austria |
|
| Turkey |
|
| Belgium |
|
| Poland |
|
| Brazil |
|
| Mexico |
|
| Italy |
|
| France |
|
| Germany |
|
|
|
|
|
|
|
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.
|
|
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.
|
|