Clinical trials: Progressive Familial Intrahepatic Cholestasis
- Condition Progressive Familial Intrahepatic Cholestasis
- Funder type Industry (pharma, biotech, devices)
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
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
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
Progressive Familial Intrahepatic Cholestasis
Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC
Open-label extension study to evaluate long-term safety and persistence of effect of A4250 in children with progressive familial intrahepatic cholestasis (PFIC).
- Trial name
- PEDFIC 2 (NCT03659916)
- Testing
- A4250 (odevixibat)
- Where
- 41 sites in United States, Australia and 13 more
- Volunteers
- 116 people
- Sponsor
- Albireo, an Ipsen Company
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
A Study to Assess the Genetic Variations in Bile Flow Disorders: Linking Progressive Familial Intrahepatic Cholestasis (PFIC)-Related Genes to Symptoms in Adults With Recurrent Cholestasis in Spain
Progressive Familial Intrahepatic Cholestasis (PFIC) is a group of inherited conditions that affect how bile moves in the liver, which can lead to serious liver problems.
- Trial name
- REGENIC (NCT07191704)
- Where
- 10 sites in Spain
- Volunteers
- 150 people
- Sponsor
- Ipsen
Progressive Familial Intrahepatic Cholestasis
Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis
To provide treatment access to patients with PFIC in the US who have pruritus and elevated serum bile acids and who are not able to enroll in A4250-008 (PEDFIC2) for the following reasons: 1) Do not meet eligibility criteria for…
- Trial name
- NCT04483531
- Testing
- Odevixibat
- Where
- United States
- Sponsor
- Albireo
Progressive Familial Intrahepatic Cholestasis (PFIC)
An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
- Trial name
- NCT04185363
- Testing
- Maralixibat
- Where
- 28 sites in United States, Argentina and 14 more
- Volunteers
- 84 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
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, 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), 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 (PFIC)
A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)
The main aim of the study is to check if TAK-625 improves symptoms of Progressive Familial Intrahepatic Cholestasis (PFIC), side effect from the study treatment or TAK-625, and how much TAK-625 stays in their blood over time.
- Trial name
- NCT05543187
- Testing
- TAK-625
- Where
- 8 sites in Japan
- Volunteers
- 5 people
- Sponsor
- Takeda
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
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
Progressive Familial Intrahepatic Cholestasis (PFIC)
A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).
- Trial name
- MARCH-PFIC (NCT03905330)
- Testing
- Maralixibat
- Where
- 31 sites in United States, Argentina and 15 more
- Volunteers
- 93 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Progressive Familial Intrahepatic Cholestasis (PFIC)
Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis
This is an open label study in children with Progressive Familial Intrahepatic Cholestasis (PFIC) designed to evaluate the safety and efficacy of LUM001, also known as Maralixibat (MRX).
- Trial name
- INDIGO (NCT02057718)
- Testing
- LUM001 (Maralixibat)
- Where
- 10 sites in United States, France and 2 more
- Volunteers
- 33 people
- Sponsor
- Mirum Pharmaceuticals, 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.
Progressive Familial Intrahepatic Cholestasis (PFIC)
A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)
The purpose of this study is to determine if the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).
- Trial name
- NCT03353454
- Testing
- Maralixibat
- Sponsor
- Mirum Pharmaceuticals, Inc.