Clinical trials: Cholestasis
Parenteral Nutrition Associated Liver Disease (PNALD), Essential Fatty Acid Deficiency
Safety Study to Evaluate the Occurrence of EFAD in Pediatric Patients With PNAC Who Require More Than Eight Weeks of Omegaven Treatment
This study will demonstrate safety in pediatric patients with Parenteral Nutrition-Associated Cholestasis treated with Omegaven®, which is indicated as a source of calories and fatty acids in this patient population
- Conditions
- Parenteral Nutrition Associated Liver Disease (PNALD), Essential Fatty Acid Deficiency, Malnutrition, Pediatric ALL
- Trial name
- NCT06274788
- Testing
- Omegaven® (fish oil triglycerides) Injectable Emulsion
- Where
- 10 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fresenius Kabi
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.
PFIC1, PFIC2
This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2
Double blind, randomized, placebo controlled, Phase 3 study to investigate the efficacy and safety of low doses and high doses of A4250 compared to placebo in children with progressive familial intrahepatic cholestasis (PFIC)…
- Trial name
- PEDFIC 1 (NCT03566238)
- Testing
- A4250 (odevixibat)
- Where
- 45 sites in United States, Australia and 13 more
- Volunteers
- 62 people
- Sponsor
- Albireo
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
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
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 (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
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
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
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
Infantile Refsum's Disease, Zellweger Syndrome
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
OBJECTIVES: I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism II.
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders
- Trial name
- NCT00007020
- Testing
- Cholic Acids
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Malnutrition, Child, Malnutrition
Safety Study of SMOFlipid to Evaluate the Risk of Developing EFAD and/or PNAC in Pediatric and Adult Patients
Evaluate the risk of developing EFAD and/or PNAC in adult and pediatric patients 1 month of age and older, who are anticipated to need 8 weeks or longer of parenteral nutrition treatment with SMOFlipid.
- Conditions
- Malnutrition, Child, Malnutrition, Essential Fatty Acid Deficiency (EFAD), Parenteral Nutrition Associated Cholestasis
- Trial name
- NCT06049680
- Testing
- SMOFlipid® (lipid injectable emulsion)
- Where
- 6 sites in United States
- Volunteers
- 100 people
- Sponsor
- Fresenius Kabi
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 (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
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
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
Pediatric Cholestasis
A4250, an IBAT Inhibitor in Pediatric Cholestasis
This study will evaluate A4250 (IBATinhibitor) as a treatment option in pediatric patients with chronic cholestasis with main emphasis on safety evaluation and on effects on pruritus
- Trial name
- NCT02630875
- Testing
- A4250
- Where
- 7 sites in Denmark, France and 3 more
- Volunteers
- 24 people
- Sponsor
- Albireo
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.
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, 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.