Clinical trials: PFIC - Progressive Familial Intrahepatic Cholestasis
- Condition PFIC - Progressive Familial Intrahepatic Cholestasis
- Funder type Industry (pharma, biotech, devices)
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
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 (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 (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
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.
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.