Clinical trials: Alagille Syndrome (ALGS)
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)
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
Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome
A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].
- Conditions
- Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome, Cholestatic Liver Disease
- Trial name
- RISE (NCT04729751)
- Testing
- Maralixibat
- Where
- 14 sites in United States, Belgium and 5 more
- Volunteers
- 27 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Alagille Syndrome (ALGS)
A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)
The main aim of the study is to check if TAK-625 improves symptoms of Alagille Syndrome (ALGS), side effect from the study treatment or TAK-625, and how much TAK-625 stays in their blood over time.
- Trial name
- NCT05543174
- Testing
- TAK-625
- Where
- 8 sites in Japan
- Volunteers
- 7 people
- Sponsor
- Takeda
Alagille Syndrome
Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)
This is a long-term, open-label study with a double-blind, placebo-controlled, randomized drug withdrawal period in children with Alagille Syndrome (ALGS) designed to evaluate the safety and efficacy of LUM001 (Also known as…
- Trial name
- ICONIC (NCT02160782)
- Testing
- LUM001 (Maralixibat)
- Where
- 9 sites in Australia, Belgium and 4 more
- Volunteers
- 31 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Liver Diseases, Alagille Syndrome
Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)
Cholestasis is a condition in which bile is not properly transported from the liver to the small intestine. Cholestasis can be caused by an array of childhood diseases, including the genetic diseases Alagille syndrome (ALGS)…
- Conditions
- Liver Diseases, Alagille Syndrome, Alpha 1-Antitrypsin Deficiency
- Trial name
- LOGIC (NCT00571272)
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,675 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 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.
Alagille Syndrome, Atorvastatin
Atorvastatin Therapy on Xanthoma in Alagille Syndrome
To observe the efficacy and safety of atorvastatin on xanthoma in Alagille syndrome through a prospective study.
- Conditions
- Alagille Syndrome, Atorvastatin, Xanthoma
- Trial name
- NCT05488067
- Testing
- atorvastatin
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Children's Hospital of Fudan University
Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease
The purpose of the study is to validate the ItchRO instrument (a clinical outcome assessment measure of itching) prior to the analysis of longitudinal treatment effect data being generated in ongoing clinical trials.
- Trial name
- NCT02131623
- Where
- 5 sites in United States, Canada
- Volunteers
- 23 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Orphan Cholestatic Liver Diseases, Progressive Familial Intrahepatic Cholestasis
An Open Label, Single-dose, Single Period ADME Study of A4250 in Healthy Subjects
The primary objectives of the study are to assess the mass balance recovery after a single dose of carbon-14 [14C]-A4250 as a capsule and to provide plasma, urine and faecal samples for metabolite profiling and structural…
- Conditions
- Orphan Cholestatic Liver Diseases, Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome, Primary Biliary Cirrhosis
- Trial name
- NCT03082937
- Testing
- 3 mg [14C]-A4250 capsule
- Where
- United Kingdom
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Albireo
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