Clinical trials: Genetic Liver Disorder
Alpha 1-Antitrypsin Deficiency, Emphysema
A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo.
The purpose of this study is to investigate the effect of alvelestat (an oral neutrophil elastase inhibitor) on blood and sputum biomarkers in patients with PiZZ, null or rare variant phenotype/genotype alpha-1 anti-trypsin…
- Conditions
- Alpha 1-Antitrypsin Deficiency, Emphysema, COPD
- Trial name
- ASTRAEUS (NCT03636347)
- Testing
- Alvelestat oral tablet - dose 1, Alvelestat oral tablet - dose 2
- Where
- 26 sites in United States, Belgium and 6 more
- Volunteers
- 99 people
- Sponsor
- Mereo BioPharma
Liver Iron Overload
Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study
The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.
- Trial name
- NCT01033747
- Testing
- Deferasirox
- Where
- 4 sites in Italy
- Volunteers
- 70 people
- Sponsor
- Novartis Pharmaceuticals
Alpha 1-Antitrypsin Deficiency
Pharmacokinetic Study of ARALAST (Human Alpha1- PI)
The primary purpose of this study is to characterize the pharmacokinetic profile of intravenous Aralast Fraction (Fr.) IV-1, a sterile, stable, lyophilized preparation of functionally intact human Alpha1- Proteinase Inhibitor…
- Trial name
- NCT00242385
- Testing
- Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor, Dose of 60 mg/kg alpha1-proteinase inhibitor
- Where
- 7 sites in Australia, New Zealand
- Volunteers
- 25 people
- Sponsor
- Baxalta now part of Shire
Alpha-1 Antitrypsin Deficiency
Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation"
To evaluate different doses of "Kamada-AAT for Inhalation" on the levels of alpha 1-proteinase inhibitor and other analytes in epithelial lining fluid (ELF) and serum and to assess the safety of the treatment in subjects with AAT…
- Trial name
- NCT02001688
- Testing
- Kamada-AAT for Inhalation, 80mg, Kamada-AAT for Inhalation, 160mg
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Kamada, Ltd.
Wilson Disease
Pharmacokinetic Study of Oral ALXN1840 in Japanese and Non-Japanese Adult Healthy Participants
The main purpose of the study is to confirm how long ALXN1840 stays in the body of Japanese and non-Japanese healthy participants (that is, pharmacokinetic [PK] profile).
- Trial name
- NCT05641311
- Testing
- ALXN1840
- Where
- United Kingdom
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Alpha 1-Antitrypsin Deficiency
Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)
The goal of this trial was to explore the utility of evaluating emphysema progression through CT scans measuring lung density during a 2 year period of weekly infusions of either placebo or human alpha-1-antitrypsin (AAT…
- Trial name
- NCT00263887
- Testing
- Alpha1-Proteinase Inhibitor (Human), Albumin (Human) 20%, United States Pharmacopeia (USP)
- Where
- 3 sites in Denmark, Sweden and 1 more
- Volunteers
- 77 people
- Sponsor
- Grifols Therapeutics LLC
Hemophilia A
Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"
In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor VIII gene into the human body by use of a viral vector to treat the…
- Trial name
- NCT03588299
- Testing
- BAY2599023 (DTX201)
- Where
- 13 sites in United States, Bulgaria and 4 more
- Volunteers
- 11 people
- Sponsor
- Bayer
Orphan Cholestatic Liver Diseases, Primary Biliary Cirrhosis
A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384
The primary objectives of the study are to evaluate the safety, tolerability and pharmacokinetics of A4250 after single or multiple oral doses in healthy subjects.
- Conditions
- Orphan Cholestatic Liver Diseases, Primary Biliary Cirrhosis, Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome
- Trial name
- NCT02963077
- Testing
- A4250, CRC (A3384), Questran
- Volunteers
- 94 people, healthy volunteers welcome
- Sponsor
- Albireo
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.
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
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
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.
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.