Clinical trials: Alagille Syndrome (ALGS)
Alagille Syndrome
Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome
The study is a randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 versus placebo on the biochemical markers and…
- Trial name
- IMAGO (NCT01903460)
- Testing
- LUM001
- Where
- 3 sites in United Kingdom
- Volunteers
- 20 people
- Sponsor
- Mirum Pharmaceuticals, 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.
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
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 (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 (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
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
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.
Alagille Syndrome
A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
An open-label, single-arm, multi-centre program providing treatment access to ALGS patients with cholestatic pruritus in the US.
- Trial name
- NCT04530994
- Testing
- maralixibat
- Where
- 14 sites in United States
- Sponsor
- Mirum Pharmaceuticals, Inc.
Alagille Syndrome
Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome.
- Trial name
- ASSERT (NCT04674761)
- Testing
- Odevixibat
- Where
- 32 sites in United States, Belgium and 11 more
- Volunteers
- 52 people
- Sponsor
- Albireo
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
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
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.
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
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.
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