Clinical trials: Progressive Familial Intrahepatic Cholestasis, active
Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
The objective of this 5-year, prospective, observational cohort study is to evaluate the long-term safety and clinical outcomes of patients with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC)…
- Trial name
- LEAP (NCT06193928)
- Testing
- Livmarli
- Where
- 8 sites in United States
- Volunteers
- 70 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Progressive Familial Intrahepatic Cholestasis
A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea
This study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) as they use odevixibat in their daily lives.
- Trial name
- NCT07185919
- Where
- 5 sites in South Korea
- Volunteers
- 10 people
- Sponsor
- Ipsen
Cholestatic Liver Disease, Intrahepatic Cholestasis
Genotype-phenotype Relationship Between Cryptogenic Cholestasis and Familial Intrahepatic Cholestasis
Genotype-phenotype relationship between adult cryptogenic cholestasis and mutations in genes responsible for progressive familial intrahepatic cholestasis
- Conditions
- Cholestatic Liver Disease, Intrahepatic Cholestasis, Progressive Familial Intrahepatic Cholestasis, Hepatobiliary Cancer
- Trial name
- NCT06781242
- Where
- 2 sites in Italy
- Volunteers
- 300 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- 2 sites in United States, Australia
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC)
A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)
This study is a database study in Japan for maralixibat (TAK-625) used to treat participants with Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC).
- Trial name
- NCT07293897
- Testing
- Maralixibat
- Where
- Japan
- Volunteers
- 50 people
- Sponsor
- Takeda
Progressive Familial Intrahepatic Cholestasis
Prospective Analysis of the Treatment of Progressive Familial Intrahepatic Cholestasis (TreatFIC)
The project has the following general aims: 1. Natural course and prognosis: To prospectively follow the natural course and prognosis of the different types of PFIC, to broaden the understanding of the different very rare…
- Trial name
- TreatFIC (NCT06778174)
- Testing
- observational study
- Where
- Netherlands
- Volunteers
- 200 people
- Sponsor
- University Medical Center Groningen
Cholestatic Liver Disease, Progressive Familial Intrahepatic Cholestasis
DEFINING THE GENETIC DRIVERS OF ADULT-ONSET CHOLESTATIC LIVER DISEASE
Cholestatic disease in adults comprises a heterogeneous group of conditions characterized by intra- or extrahepatic alterations of bile flow that can lead to fibrosis or hepatic decompensation.
- Trial name
- FIRST (NCT07317193)
- Testing
- Advanced Whole Genome Sequencing to Identify Rare Pathogenic Variants in Unexplained Cholestatic Liver Disease
- Where
- Italy
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Progressive Familial Intrahepatic Cholestasis
NAtural Course and Prognosis of PFIC and Effect of Biliary Diversion
The natural course of PFIC syndromes and the effect of diversion techniques, have so far not been characterized in a rigorous manner within a larger population of patients.
- Trial name
- NAPPED (NCT03930810)
- Testing
- Surgical biliary diversion
- Where
- Netherlands
- Volunteers
- 1,500 people
- Sponsor
- University Medical Center Groningen
Dental Diseases
Dental Dyschromia and Quality of Life in Early Prolonged Hyperbilirubinemia
The main objective of this study is to assess the prevalence of dental discoloration (dental dyschromia) in children who experienced high levels of bilirubin in their blood (hyperbilirubinemia) during their early years.
- Trial name
- SMILE (NCT06506734)
- Testing
- Questionnaires
- Where
- 9 sites in France
- Volunteers
- 120 people
- Sponsor
- University Hospital, Toulouse
PFIC - Progressive Familial Intrahepatic Cholestasis
Odevixibat Outcomes in Patients With PFIC Versus an External Control Cohort (OvEC-PFIC)
Progressive familial intrahepatic cholestasis (PFIC) is a rare inherited liver disease that causes a build-up of bile acids in the liver.
- Trial name
- OvEC-PFIC (NCT07497724)
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Ipsen
Hepatobiliary Cancers, Progressive Familial Intrahepatic Cholestasis (PFIC)
Familial Intrahepatic Cholestasis-related Genes Associated with Disease Susceptibility in Hepato-biliary Cancers
This is a cross-sectional, multicenter tissue study with an exploratory aim to estimate the prevalence of genetic mutations that predispose individuals to diseases in the context of cholestatic disorders and hepatobiliary…
- Conditions
- Hepatobiliary Cancers, Progressive Familial Intrahepatic Cholestasis (PFIC), Cholestatic Liver Disease
- Trial name
- NCT06777914
- Where
- 5 sites in Italy
- Volunteers
- 600 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
PFIC - Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome (ALGS)
Pediatric Evaluation and Registry for Liver Cholestasis in Canada
The purpose of this study is to create a national, multi-centre registry for children with Alagille syndrome (ALGS) and Genetic Intrahepatic Cholestasis (GIC) that follows participants long-term, ensuring standardized…
- Conditions
- PFIC - Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome (ALGS), Cholestasis, Intrahepatic
- Trial name
- PEARL (NCT07411716)
- Where
- 13 sites in Canada
- Volunteers
- 220 people
- Sponsor
- Children's Hospital of Eastern Ontario
Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)
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.
- Trial name
- NCT07290257
- Testing
- Livmarli Oral Product
- Where
- 15 sites in Belgium, France and 6 more
- Volunteers
- 230 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Progressive Familial Intrahepatic Cholestasis
A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China
This registry-based study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) who take odevixibat (Bylvay) as part of routine clinical care in China.
- Trial name
- NCT07588880
- Where
- 6 sites in China
- Volunteers
- 20 people
- Sponsor
- Ipsen