Clinical trials: Liver Metabolism
- Condition Liver Metabolism
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Blood Clots, Infection and Inflammatory Reaction
Cobas® Lumira Collection of Venous and Capillary Blood Samples for the Research, Optimization and Calibration of New Diagnostic Devices
This study is a blood sample collection study, collecting venous and capillary blood samples from adult patients in the UK, with a range of health conditions.
- Conditions
- Blood Clots, Infection and Inflammatory Reaction, Heart Disease, Kidney Disease
- Trial name
- EVOLVE (NCT07378514)
- Testing
- Blood samples are obtained and pseudonymized, prior to use for the development, optimization and calibration of diagnostic tests
- Volunteers
- 30,000 people
- Sponsor
- LumiraDx UK Limited
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Wilson's Disease
Efficacy and Safety, Long-term Study of Zinc Acetate to Treat Wilson's Disease in Japan.
The purpose of this long-term study is to determine whether Zinc Acetate is effective and safe in the treatment of Wilson's disease among Japanese.
- Trial name
- NCT00212355
- Testing
- NPC-02
- Volunteers
- 37 people
- Sponsor
- Nobelpharma
Urea Cycle Disorders
Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders
Treatment with liver cell infusion for children with urea cycle disorders (UCD).
- Trial name
- NCT01195753
- Testing
- HHLivC
- Where
- 5 sites in United States, Canada
- Volunteers
- 10 people
- Sponsor
- Cytonet GmbH & Co. KG
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP
To investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with EPP or XLP.
- Trial name
- INSPIRE (NCT06144840)
- Testing
- Dersimelagon
- Where
- 35 sites in United States, Australia and 9 more
- Volunteers
- 165 people
- Sponsor
- Tanabe Pharma America, Inc.
Head Injury
Effect of Intrathoracic Pressure Regulation on Traumatic Brain Injury
The purpose of this study is to demonstrate proof of clinical concept that application of the Intrathoracic Pressure Regulator (ITPR) will result in an increase in Cerebral Perfusion Pressure (CPP) and a decrease in Intracranial…
- Trial name
- NCT01824589
- Testing
- -7 cm H2O ITPR, -12cm H2O ITPR
- Volunteers
- 1 people
- Sponsor
- Advanced Circulatory Systems
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
Urea Cycle Disorders, Carbamoylphosphate Synthetase I Deficiency
Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders
Urea cycle disorders are rare inherited diseases that generally have a poor outcome. In this study, neonates and infants with UCD will be included within the first 3 months of life and will be treated by repetitive application of…
- Conditions
- Urea Cycle Disorders, Carbamoylphosphate Synthetase I Deficiency, Ornithine Transcarbamylase Deficiency, Citrullinemia
- Trial name
- NCT00718627
- Testing
- Human Heterologous Liver Cells
- Where
- 2 sites in Germany
- Volunteers
- 12 people
- Sponsor
- Cytonet GmbH & Co. KG
Wilson's Disease
A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson's Disease Patients
To evaluate the effects of a single dose of trientine in children ≥6 years and adult patients with Wilson's disease.
- Trial name
- NCT01874028
- Testing
- trientine dihydrochloride
- Where
- Germany
- Volunteers
- 20 people
- Sponsor
- Univar BV
Wilson Disease
Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months.
- Trial name
- NCT02426905
- Testing
- trientine dihydrochloride
- Where
- 4 sites in Germany, Greece and 2 more
- Volunteers
- 90 people
- Sponsor
- Univar BV
Erythropoietic Protoporphyria
HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.
- Trial name
- NCT05883748
- Testing
- DISC-1459
- Where
- 26 sites in United States, Australia and 9 more
- Volunteers
- 230 people
- Sponsor
- Disc Medicine, Inc
Wilson Disease
Early Access Program for ALXN1840 in Patients With Wilson Disease
This is an open-label, single-arm, multi-center EAP, designed to provide early access to ALXN1840 for eligible patients with WD.
- Trial name
- NCT05686564
- Testing
- ALXN1840
- Sponsor
- Alexion Pharmaceuticals, Inc.
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Expanded Access Program of Bitopertin For Participants With EPP or XLP
The goal of this expanded access program is to provide bitopertin to patients with EPP and XLP who have no satisfactory treatment options available in the US and to learn if bitopertin is safe to treat EPP and XLP.
- Trial name
- NCT07603401
- Testing
- DISC-1459
- Where
- 8 sites in United States
- Sponsor
- Disc Medicine, Inc
Parenteral Nutrition Associated Liver Disease, Essential Fatty Acid Deficiency
Safety of Omegaven in Pediatric Patients With Parenteral Nutrition-Associated Cholestasis (PNAC)
This study will demonstrate Short-, mid-, and long-term 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…
- Conditions
- Parenteral Nutrition Associated Liver Disease, Essential Fatty Acid Deficiency, Neurocognitive Deficit, Malnutrition
- Trial name
- NCT04807478
- Testing
- Omegaven® (fish oil triglycerides) Injectable Emulsion
- Sponsor
- Fresenius Kabi
Acute Hepatic Porphyria
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).
- Trial name
- NCT04056481
- Testing
- Givosiran
- Sponsor
- Alnylam Pharmaceuticals
Bile Acid Synthesis Disorders
The REPLACE Registry for Cholbam® (Cholic Acid)
This is a prospective, observational, non-interventional patient registry study designed to document product safety and effectiveness outcomes for 10 years in patients treated with Cholbam, including those who have been using…
- Trial name
- NCT03115086
- Testing
- Cholbam
- Where
- 24 sites in United States
- Volunteers
- 55 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also…
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
- Trial name
- NCT06805695
- Testing
- No Intervention
- Where
- 4 sites in United States, France and 1 more
- Volunteers
- 13 people
- Sponsor
- iECURE, Inc.
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
PATHWAY is a Phase 2b/3 clinical study with an Open-Label Extension (OLE). It tests PORT-77 in patients with EPP, EPP2, and XLP.
- Conditions
- Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP), EPP2
- Trial name
- PATHWAY (NCT07820085)
- Testing
- PORT-77
- Where
- 2 sites in United States
- Volunteers
- 225 people
- Sponsor
- Portal Therapeutics, Inc.
Obesity, Type 2 Diabetes (T2D)
A Retrospective Real-World Cohort Study
The primary objectives are to determine the prevalence and overlap of major cardiometabolic diseases, identify multimorbidity clusters, and characterize demographic, clinical, laboratory, and treatment patterns across the cohort…
- Conditions
- Obesity, Type 2 Diabetes (T2D), Prediabetes, Cardiovascular Disease (CVD)
- Trial name
- SCALA-TK-CM (NCT07781371)
- Testing
- No treatment given
- Where
- Denmark
- Volunteers
- 100,000 people
- Sponsor
- Novo Nordisk A/S
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.