Clinical trials: Alagille Syndrome, active
Alagille Syndrome
Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
The purpose of this study is to assess the long-term safety and effectiveness of odevixibat in participants with Alagille syndrome (ALGS).
- Trial name
- ASSERT-EXT (NCT05035030)
- Testing
- Odevixibat
- Where
- 35 sites in United States, Belgium and 8 more
- Volunteers
- 62 people
- Sponsor
- Albireo, an Ipsen Company
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
Alagille Syndrome
A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
This study will collect information from patients with ALGS who are using odevixibat in their daily lives. Odevixibat is a medication that helps patients with ALGS, a rare disease that affects the liver and causes itching.
- Trial name
- NCT07585097
- Where
- 14 sites in France, Italy
- Volunteers
- 30 people
- Sponsor
- Ipsen
Exocrine Pancreatic Insuficiency
Determine the Prevalence of Exocrine Pancreatic Insufficiency (EPI) in Pediatric and Adult Participants With Alagille Syndrome After Liver Transplantation
The goal of this clinical trial is to determine the prevalence of exocrine pancreatic insufficiency (EPI) in a population of pediatric and adult participants with Alagille Syndrome (ALGS) post liver transplant.
- Trial name
- NCT07335523
- Testing
- Fecal Fat
- Where
- 8 sites in United States
- Volunteers
- 30 people
- Sponsor
- Digestive Care, Inc.
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.
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.
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 (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
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
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