Clinical trials: Alagille Syndrome
Orphan Cholestatic Liver Diseases, Primary Biliary Cirrhosis
A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384
The primary objectives of the study are to evaluate the safety, tolerability and pharmacokinetics of A4250 after single or multiple oral doses in healthy subjects.
- Conditions
- Orphan Cholestatic Liver Diseases, Primary Biliary Cirrhosis, Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome
- Trial name
- NCT02963077
- Testing
- A4250, CRC (A3384), Questran
- Volunteers
- 94 people, healthy volunteers welcome
- Sponsor
- Albireo
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
Alagille Syndrome
Positional Cloning of the Gene(s) Responsible for Alagille Syndrome
The goal of the project is to identify and clone the gene(s) responsible for the Alagille Syndrome (AGS) by a positional cloning approach.
- Trial name
- NCT00001642
- Where
- United States
- Volunteers
- 225 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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, Cholestasis
Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease
OBJECTIVES: I. Determine the role of magnesium deficiency in the pathogenesis of decreased serum vitamin D and reduced bone density in children with chronic cholestatic liver disease.
- Conditions
- Alagille Syndrome, Cholestasis, Biliary Atresia
- Trial name
- NCT00007033
- Testing
- magnesium gluconate, magnesium sulfate
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Center for Research Resources (NCRR)
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.
Alagille Syndrome
Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome
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…
- Trial name
- ITCH (NCT02057692)
- Testing
- LUM001
- Where
- 13 sites in United States, Canada
- Volunteers
- 37 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Alagille Syndrome
An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol.
- Trial name
- IMAGINE-II (NCT02117713)
- Testing
- LUM001 (Maralixibat)
- Where
- 11 sites in United States, Canada
- Volunteers
- 34 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Hepatitis Autoimmune Disease, Primary Biliary Cholangitis (PBC)
Spanish Registry of Autoimmune and Cholestatic Liver Diseases (ColHai)
The purpose of the registry is to know the status of primary biliary colgantis, autoimmune hepatitis, primary sclerosing cholagitis and genetic cholestatic diseases in Spain.
- Conditions
- Hepatitis Autoimmune Disease, Primary Biliary Cholangitis (PBC), Primary Sclerosing Cholangitis (PSC), Cholestatic Genetic Diseases
- Trial name
- ColHai (NCT06767605)
- Where
- Spain
- Volunteers
- 5,000 people
- Sponsor
- Asociación Española para el Estudio del Hígado
Biliary Atresia, Alagille Syndrome
FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)
Noninvasive monitoring of liver fibrosis is an unmet need within the clinical management of pediatric chronic liver disease. While liver biopsy is often used in the initial diagnostic evaluation, subsequent biopsies are rarely…
- Conditions
- Biliary Atresia, Alagille Syndrome, Alpha1 Anti-Trypsin Deficiency, Portal Hypertension
- Trial name
- FORCE (NCT02922751)
- Testing
- Liver Stiffness Measurement (LSM)
- Where
- 13 sites in United States, Canada
- Volunteers
- 552 people
- Sponsor
- Arbor Research Collaborative for Health
Alagille Syndrome
An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS)
The purpose of this extension study is to determine the long-term safety and tolerability of an investigational treatment (LUM001 also known as Maralixibat) in children with ALGS who have completed participation in a core LUM001…
- Trial name
- IMAGINE (NCT02047318)
- Testing
- LUM001 (Maralixibat)
- Where
- 3 sites in United Kingdom
- Volunteers
- 19 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Alagille Syndrome
A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives.
- Trial name
- NCT06850038
- Where
- 10 sites in United States
- Volunteers
- 30 people
- Sponsor
- Ipsen