Clinical trials: Genetic Liver Disorder
Diabetes Mellitus, Hypertension
Evidence-based Laboratory Test Order Sets in Primary Care
Cluster randomised controlled trial to evaluate what the effect is of evidence-based order sets aimed at five indications on the appropriateness of laboratory test ordering in primary care.
- Conditions
- Diabetes Mellitus, Hypertension, Cardiovascular Diseases, Fatigue
- Trial name
- NCT02950142
- Testing
- CPOE with order sets for lab testing, CPOE without order sets for lab testing
- Where
- Belgium
- Volunteers
- 288 people, healthy volunteers welcome
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Advanced Solid Tumor, Head and Neck Squamous Cell Carcinoma
A Study of [225Ac]-FPI-1966 in Participants With Advanced Solid Tumours
This first-in-human study evaluates safety, tolerability and distribution of [225Ac] FPI-1966, [111In]-FPI-1967, and vofatamab in patients with FGFR3-expressing solid tumors.
- Conditions
- Advanced Solid Tumor, Head and Neck Squamous Cell Carcinoma, Bladder Carcinoma, Susceptible FGFR3 Genetic Alterations
- Trial name
- NCT05363605
- Testing
- [225Ac]-FPI-1966, [111In]-FPI-1967, vofatamab
- Where
- 6 sites in United States, Australia
- Volunteers
- 6 people
- Sponsor
- Fusion Pharmaceuticals Inc.
Thalassemia, Iron Overload
Long-term Clinical Study of CN128 in Thalassemia With Sever Liver Iron Overloaded Patients
The safety and efficacy of CN128 is studied in thalassaemia with sever liver iron overloaded patients.
- Trial name
- NCT05355766
- Testing
- CN128 Tablets
- Where
- China
- Volunteers
- 16 people
- Sponsor
- Hangzhou Zede Pharma-Tech Co., Ltd.
Alpha 1-Antitrypsin Deficiency
Study of ARO-AAT in Normal Adult Volunteers
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-AAT in healthy adult volunteers.
- Trial name
- NCT03362242
- Testing
- ARO-AAT Injection, Sterile Normal Saline (0.9% NaCl)
- Where
- New Zealand
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Arrowhead Pharmaceuticals
Acute Intermittent Porphyria (AIP)
Identification of Acute Intermittent Porphyria Modifying Genes
This study proposes to identify the predisposing/protective modifying genes that underlie the acute attacks in symptomatic patients with Acute Intermittent Porphyria (AIP), an autosomal dominant inborn error of heme biosynthesis.
- Trial name
- NCT05502133
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Rheumatoid Arthritis, Ankylosing Spondylitis
Induction of Regulatory t Cells by Low Dose il2 in Autoimmune and Inflammatory Diseases
TRANSREG will assess the safety and biological efficacy of low-dose IL2 as a Treg inducer in a set of 14 autoimmune and auto-inflammatory diseases, with the aim to select diseases in which further therapeutic development will be…
- Conditions
- Rheumatoid Arthritis, Ankylosing Spondylitis, Systemic Lupus Erythematosus, Psoriasis
- Trial name
- TRANSREG (NCT01988506)
- Testing
- Interleukin 2
- Where
- 11 sites in France
- Volunteers
- 81 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Alpha1-Antitrypsin Deficiency
Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE)
This is a multi-center, open-label study to evaluate the long-term safety of weekly intravenous (IV) infusions of 60 mg/kg alpha1-PI (human), modified process (Alpha-1 MP) in adult participants with Alpha1 Antitrypsin Deficiency…
- Trial name
- NCT02870348
- Testing
- Alpha-1 MP
- Where
- 3 sites in Japan
- Volunteers
- 4 people
- Sponsor
- Grifols Therapeutics LLC
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.
Wilson Disease
Spanish Wilson Disease Registry
The main objective and purpose of the Registry is to know the current status of Wilson Disease in Spain. As secondary objectives, the prevalence and incidence of the disease will be analysed.
- Trial name
- NCT06466291
- Where
- Spain
- Volunteers
- 600 people
- Sponsor
- Asociación Española para el Estudio del Hígado
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men
A Study to Learn How Different Levels of Decreased Liver Function Influence Blood Levels of Elinzanetant Compared to Normal Liver Function in Male and Female Participants
Researchers are looking for a better way to treat people who have symptoms caused by hormonal changes, like those that happen in women during menopause. These symptoms can include vasomotor symptoms.
- Trial name
- NCT04903821
- Testing
- BAY3427080
- Where
- 2 sites in Germany
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Bayer
Acute Hepatic Porphyria
EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.
- Trial name
- NCT02240784
- Where
- 30 sites in United States, Australia and 18 more
- Volunteers
- 136 people
- Sponsor
- Alnylam Pharmaceuticals
Fatty Liver, Obesity
Genetic Variant in Apolipoprotein C3 Gene and Fatty Liver in Obese Children
In the past decades, obesity in children is much more prevalent in the world. Given the increasing prevalence of pediatric obesity worldwide, fatty liver incidence is on the rise.
- Trial name
- NCT01682655
- Where
- Taiwan
- Volunteers
- 500 people
- Sponsor
- Far Eastern Memorial Hospital
Wilson Disease
A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease
Wilson's disease (WD), also known as Wilson's disease, is a rare autosomal recessive metabolic disorder caused by a mutation of the copper transport ATPase β (ATP7B) gene located on the long arm of chromosome 13 (13q14.3).
- Trial name
- WD (NCT06650319)
- Testing
- LY-M003
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Chaohui Yu
Chronic Hepatitis C - Genotype 3
Chronic Hepatitis C (CHC) - Genotype 3 Infection in Canada
Hepatitis C is a small RNA virus spread by blood to blood contamination. There are to date 6 known genotypes and within each there are several subtypes.
- Trial name
- NCT01100749
- Where
- 2 sites in Canada
- Volunteers
- 25 people
- Sponsor
- University Health Network, Toronto
Chronic Liver Disease, Hepatocellular Carcinoma
Genetic Polymorphisms of TNF-α Promoter and HBV Genotype on Outcome of HBV-Related Chronic Liver Disease
The prognosis of Hepatitis B virus (HBV)infection varies from minimal progressive liver disease to cirrhosis or hepatocellular carcinoma (HCC).Both viral and host genetic factors contribute to disease severity.
- Trial name
- NCT00629668
- Testing
- TNF-alpha and HBV genotype, TNF-alpha and HBV genotypes
- Where
- Taiwan
- Volunteers
- healthy volunteers welcome
- Sponsor
- Kaohsiung Medical University Chung-Ho Memorial Hospital
Porphyria Cutanea Tarda, Hepatitis C
Harvoni Treatment Porphyria Cutanea Tarda
In the medical literature there case reports that Harvoni improves symptoms in patients with PCT. However, this has never been systematically tested.
- Trial name
- NCT03118674
- Testing
- Harvoni
- Where
- 3 sites in United States
- Volunteers
- 23 people
- Sponsor
- Wake Forest University Health Sciences
Wilson Disease
Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
The main purpose of the study was to evaluate the efficacy of ALXN1840 (formerly WTX101) for 24 weeks on non-ceruloplasmin-bound copper (NCC) concentrations adjusted for molybdenum plasma concentration in participants newly…
- Trial name
- NCT02273596
- Testing
- ALXN1840
- Where
- 9 sites in United States, Austria and 3 more
- Volunteers
- 29 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
MASLD
Structured Physical Activity Intervention in Patients With MASLD Receiving a Mediterranean Diet
Lifestyle interventions based on diet and physical activity are the cornerstone of management for metabolic dysfunction-associated steatotic liver disease (MASLD), yet individual responses remain highly variable.
- Trial name
- EHMet-DIA (NCT07478588)
- Testing
- Exercise plus Mediterranean diet, At-home exercise plus Mediterranean diet
- Where
- 2 sites in Spain
- Volunteers
- 96 people
- Sponsor
- Fundación Pública Andaluza para la gestión de la Investigación en Sevilla
Healthy, Pulmonary Fibrosis
Identification of Genes Associated With Lung Disease in Patients With Rheumatoid Arthritis
Pulmonary fibrosis (PF) is a condition in which the lungs of a patient become scarred and fibrous. It has been known to occur in as many as 40% of patients diagnosed with rheumatoid arthritis (RA).
- Conditions
- Healthy, Pulmonary Fibrosis, Rheumatoid Arthritis
- Trial name
- NCT00001885
- Where
- United States
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Wilson Disease
Role for Biochemical Assays and Kayser-Fleischer Rings in Diagnosis of Wilson Disease
The investigators aimed to identify factors associated with symptoms and features of Wilson disease from a large cohort during long-term follow-up
- Trial name
- NCT04965545
- Testing
- low copper diet
- Where
- China
- Volunteers
- 1,000 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine