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In patients with Alagille syndrome (ALGS), the key objectives are to evaluate the tolerability, long-term safety (including possible liver toxicity) and long-term efficacy of Livmarli treatment.
In patients with Progressive Familial Intrahepatic Cholestasis (PFIC), the key objectives are to evaluate tolerability, long-term safety (including possible liver toxicity and potential impact of chronic exposure to propylene glycol (PG)), long-term efficacy, and growth and development.
This is a multicenter, open-label, low-intervention clinical study in participants diagnosed with either ALGS with cholestatic pruritus or PFIC who are treated with Livmarli.
Participants will be treated according to standard of care for the duration of the study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Livmarli | Experimental | ALGS: Primary cohort with a minimum of 45 previously untreated (treatment naïve). Supplemental cohort of participants who have previously received Livmarli. PFIC: Patients with Progressive Familial Intrahepatic Cholestasis treated with Livmarli. The study will enroll a minimum of 130 patients with PFIC |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Livmarli Oral Product | Drug | Participants will be treated according to standard of care for the duration of the study and Schedule of Assessments. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Adverse Events (AEs) | Number and proportion of participants with AEs. | Up to 7 days after the last dose of Livmarli. |
| Liver function tests (LFTs) - measuring enzyme activity or concentration | Change in LFTs from Baseline: Total and direct bilirubin | Once every 3-4 months, for 5 years. |
| Liver function tests (LFTs) - measuring enzyme activity or concentration | Change in LFTs from Baseline: Aminotransferases and GGT. | Once every 3-4 months, for 5 years. |
| Fat-Soluble Vitamins (FSV) Concentration | Change in FSV levels (vitamins A, D, and E) from Baseline. | Once every 3-4 months, for 5 years. |
| International Normalized Ratio (INR) | Change in INR level from Baseline. | Once every 3-4 months, for 5 years. |
| Long-Term Clinical Outcomes | Number of participants with surgical biliary diversion, liver transplantation, liver transplant waitlist status change, clinically evident portal hypertension, complications of liver cirrhosis, liver carcinoma, liver decompensation, and death. | Once every 3-4 months, for 5 years. |
| Laboratory Test for Monitoring of Propylene Glycol Toxicity in Participants with PFIC | Change in PG level from Baseline. | Once every 3-4 months, for 5 years. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trials Mirum | Contact | +16506674085 | clinicaltrials@mirumpharma.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cliniques Universitaires Saint Luc (UCLouvain) | Recruiting | Brussels | 1200 | Belgium |
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This is a single-group, open-label study enrolling participants with ALGS and PFIC treated with Livmarli.
For ALGS, the primary cohort will include at least 45 treatment-naïve participants. A supplemental ALGS cohort will include participants previously treated with Livmarli, with no enrollment limit.
For PFIC, the study will enroll at least 130 participants across PFIC subtypes, and all participants enrolled for at least 1 year will be included in the interim analysis.
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| University Hospital Gent (UZ Gent) | Recruiting | Ghent | 9000 | Belgium |
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| Hospices Civils de Lyon - Hopital Femme Mère Enfant | Recruiting | Bron | Auvergne-Rhône-Alpes | 69500 | France |
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| CHU de Toulouse - Hôpital des Enfants | Recruiting | Toulouse | Occitanie | 31049 | France |
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| Bicetre University Hospital | Recruiting | Le Kremlin-Bicêtre | Île-de-France Region | 94270 | France |
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| Charite Berlin | Recruiting | Berlin | 13353 | Germany |
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| Children's University Hospital Essen | Recruiting | Essen | 45149 | Germany |
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| University Hospital Hamburg-Eppendorf | Recruiting | Hamburg | 20246 | Germany |
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| 2nd Paediatric Clinic P &A Kyriakou Children's Hospital | Not yet recruiting | Athens | 11527 | Greece |
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| AO Ospedale PAPA GIOVANNI XXIII | Recruiting | Bergamo | Lombardy | 24126 | Italy |
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| Istituto mediterraneo trapianti - ISMETT | Recruiting | Palermo | Sicily | 90127 | Italy |
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| University Medical Center Groningen (UMCG) | Recruiting | Groningen | 9713GZ | Netherlands |
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| Unidade Local de Saude de Coimbra Paediatric Department Avenida Afonso Romăo | Not yet recruiting | Coimbra | 3000-602 | Portugal |
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| Hospital Universitairo Vall D'Hebron | Recruiting | Barcelona | Catalonia | 08035 | Spain |
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| Hospital Universitario La Paz | Recruiting | Madrid | 28046 | Spain |
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| ID | Term |
|---|---|
| D016738 | Alagille Syndrome |
| C535933 | Cholestasis, progressive familial intrahepatic 1 |
| D008107 | Liver Diseases |
| D002779 | Cholestasis |
| D011537 | Pruritus |
| ID | Term |
|---|---|
| D002780 | Cholestasis, Intrahepatic |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System 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 |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| C000722912 | maralixibat |
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