Clinical trials: Genetic Liver Disorder
Bleeding ,Arrhythmias, Heart Failure, Acute Renal Failure, Acute Liver Failure, Dyspnoea, Gout
Extended Drug Utilization Study Among Patients Exposed to Ticagrelor, Clopidogrel and Prasugrel
The purpose of the study is to describe patient characteristics and drug usage among patients that are prescribed ticagrelor for the first time and to compare them with patients who are prescribed clopidogrel and prasugrel for…
- Trial name
- NCT01276275
- Testing
- Risk of selected safety events
- Where
- Sweden
- Volunteers
- 7,200 people
- Sponsor
- AstraZeneca
Lysosomal Acid Lipase Deficiency
A Study to Identify and Characterize LAL-D Patients in High-risk Populations
The objective of this study is to determine the frequency of Lysosomal Acid Lipase Deficiency (LAL D) by lysosomal acid lipase (LAL) enzyme activity assay in patients who are considered to be at risk.
- Trial name
- NCT02345421
- Where
- 15 sites in United States
- Volunteers
- 640 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hemophilia, AIDS
Host Genetic Factors Influencing HIV1 and HCV Viral Loads and AIDS Clinical Progression in a Hemophilia Cohort (HGDS-3)
Background: Over 80% of the hemophiliac population who became infected with HIV prior to 1985 are also co-infected with HCV. Thus, hemophiliacs represent an important population for studies of the natural history of these chronic…
- Trial name
- NCT00340548
- Where
- 15 sites in United States
- Volunteers
- 333 people
- Sponsor
- National Cancer Institute (NCI)
Rare Diseases, Porphyrias
Colombia National Porphyria Registry
porphyria is classified as a rare disease and is produced by defects in the enzymatic activity in the biosynthesis of the heme group that leads to the over-accumulation and excretion of porphyrin precursors in hepatocytes or…
- Trial name
- NCT05496933
- Where
- Colombia
- Volunteers
- 100 people
- Sponsor
- Fundación Grupo de Investigación en Cuidados Intensivos y Obstetricia
Acute Hepatic Porphyria, Acute Intermittent Porphyria
ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
The purpose of this study is to evaluate the effect of subcutaneous givosiran (ALN-AS1), compared to placebo, on the rate of porphyria attacks in patients with Acute Hepatic Porphyrias (AHP).
- Conditions
- Acute Hepatic Porphyria, Acute Intermittent Porphyria, Porphyria, Acute Intermittent, Acute Porphyria
- Trial name
- NCT03338816
- Testing
- Givosiran
- Where
- 36 sites in United States, Australia and 16 more
- Volunteers
- 94 people
- Sponsor
- Alnylam Pharmaceuticals
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Amifostine to Protect From Side Effects of PSCT in Treating Patients With Solid Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of…
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Chordoma, Kidney Cancer
- Trial name
- NCT00003926
- Testing
- amifostine trihydrate, busulfan, filgrastim
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Hepatocellular Carcinoma
A Study of LY2157299 in Participants With Advanced Hepatocellular Carcinoma
The main purpose of this study is to evaluate the safety and effectiveness of the study drug known as LY2157299 in participants with hepatocellular carcinoma.
- Trial name
- NCT02178358
- Testing
- LY2157299, Sorafenib
- Where
- 16 sites in China, Hong Kong and 3 more
- Volunteers
- 132 people
- Sponsor
- Eli Lilly and Company
Hepatitis C, Chronic
Biliverdin Reductase A in Chronic Hepatitis C Virus Infection
In this project, the investigators aim to study the role of biliverdin reductase A (BLVRA) in HCV infected patients prior and during/after standard antiviral therapy in association with viral clearance, disease progression and…
- Trial name
- NCT01112033
- Where
- Czechia
- Volunteers
- 113 people, healthy volunteers welcome
- Sponsor
- Charles University, Czech Republic
MRI Scans
Quantitative Susceptibility Mapping (QSM) to Guide Iron Chelating Therapy
The overall goal of this research is to help develop a new magnetic resonance (MR) method, Quantitative Susceptibility Mapping (QSM), to improve the measurement of liver iron concentrations without the need for a liver biopsy.
- Trial name
- NCT04171635
- Testing
- Quantitative Susceptibility Mapping (QSM) Magnetic Resonance Imaging (MRI), R2* Magnetic Resonance Imaging (MRI)
- Where
- 2 sites in United States
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University
Hepatocellular Carcinoma
Genetic Profiling of Liver Cancer in Patients Undergoing Liver Transplantation
The purpose of this study is to determine whether genetic markers unique to liver cancer are present and to assess usefulness as a diagnostic tool.
- Trial name
- NCT02412579
- Testing
- None - Standard of Care
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Baylor Research Institute
Chronic Obstructive Pulmonary Disease, Acute Exacerbation Copd
COPD Exacerbation Blood and Urine Biomarkers Study
This will be a prospective study examining serum levels of MMP-13 and alpha-1 antitrypsin as well as other biomarkers as well as urine biomarkers of smoking status and collagen degradation in the COPD patient population.
- Conditions
- Chronic Obstructive Pulmonary Disease, Acute Exacerbation Copd, Alpha-1 Anti-trypsin Deficiency
- Trial name
- NCT03823443
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Columbia University
Lipid Metabolism, Inborn Errors, Hyperlipidemias
Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration
This study will assess what, if any, effect that ISIS 301012 (mipomersen) has on liver triglyceride content in multiple groups of subjects with varying degrees of risk for hepatic steatosis.
- Conditions
- Lipid Metabolism, Inborn Errors, Hyperlipidemias, Metabolic Diseases, Hypolipoproteinemia
- Trial name
- NCT00362180
- Testing
- mipomersen
- Where
- Netherlands
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Kastle Therapeutics, LLC
Wilson Disease
Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.
This was a Phase 1, randomized, 2-period, 2-sequence, cross-over study designed to determine the effect of ALXN1840 on the metabolism of celecoxib, a sensitive cytochrome P450 2C9 (CYP2C9) substrate, in healthy male and female…
- Trial name
- NCT04526197
- Testing
- ALXN1840, Celecoxib
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Liver Cirrhosis
A Phase 2 Study of Foscenvivint in Liver Cirrhosis Patients Caused by HIV/HCV Co-infection With Hemophilia
This is a phase 2 study to evaluate the efficacy and safety of foscenvivint in patients with liver cirrhosis resulting from HIV/HCV co-infection in the setting of hemophilia.
- Trial name
- OP-724-H202 (NCT07681089)
- Testing
- Foscenvivint
- Where
- 4 sites in Japan
- Volunteers
- 7 people
- Sponsor
- Kiminori Kimura, MD
Alpha 1 Antitrypsin Deficiency, AATD
Genomic Research in Alpha-1 Antitrypsin Deficiency
This project is designed to examine the interaction between the microflora in the lower airway and the concentration of a serum protein called alpha-1 antitrypsin.
- Trial name
- GRADS Alpha-1 (NCT01832220)
- Where
- 9 sites in United States
- Volunteers
- 140 people
- Sponsor
- University of Pittsburgh
Wilson Disease
Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
This study is being conducted to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease (WD).
- Trial name
- NCT05047523
- Testing
- ALXN1840, Standard of Care
- Where
- 20 sites in Australia, France and 5 more
- Volunteers
- 40 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
The Recipients Undergoing Liver Transplantation
Identification of Genetic Polymorphism Related to Acute Kidney Injury After Liver Transplantation Through Genome-wide Association Study (GWAS) in Korean Population
Some of the liver transplantation recipients experience postoperative acute kidney injury due to various causes including genetic factors.
- Trial name
- NCT03344380
- Where
- South Korea
- Volunteers
- 330 people
- Sponsor
- Yonsei University
Liver Cirrhosis
A Phase 2 Trial of Foscenvivint in Liver Cirrhosis Patients Caused by HIV/HCV Co-infection With Hemophilia (OP-724-H201)
This is a phase 2 trial of foscenvivint in liver cirrhosis patients caused by HIV/HCV co-infection with hemophilia to evaluate the efficacy, safety and pharmacokinetics.
- Trial name
- NCT06144086
- Testing
- Foscenvivint
- Where
- 3 sites in Japan
- Volunteers
- 5 people
- Sponsor
- Kiminori Kimura, MD