Clinical trials: Cholestasis, Intrahepatic
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
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.
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.
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
Intrahepatic Cholestasis of Pregnancy
A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)
Part 1 is an open-label randomized study of volixibat in patients with Intrahepatic Cholestasis of Pregnancy (ICP) and elevated serum bile acid concentrations (sBA) to evaluate safety and tolerability of two doses of volixibat.
- Trial name
- OHANA (NCT04718961)
- Testing
- Volixibat
- Where
- 20 sites in United States, New Zealand and 1 more
- Volunteers
- 4 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Bile Salt Export Pump (BSEP) Transporter, Polysorbate 80
Impact of Metformin and Polysorbate 80 on Drug Absorption and Disposition
The study employs two-sub-studies that share a common placebo arm. The objective of one sub-study is to assess the impact of metformin on pravastatin and chenodeoxycholic acid pharmacokinetics.
- Trial name
- NCT04640571
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- University of Maryland, Baltimore
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.