Clinical Trials on Urea Cycle Disorders, Inborn in Austria

Total 503 results

Ultragenyx Pharmaceutical Inc

Completed

A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects

Ornithine Transcarbamylase Deficiency

United States
Aeglea Biotherapeutics

Completed

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

Arginase I Deficiency | Hyperargininemia

United States, Italy, Canada, France, United Kingdom, Austria, Germany
Ultragenyx Pharmaceutical Inc

Recruiting

Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency

OTC Deficiency

United States, Portugal, Australia, Italy, Spain, United Kingdom, Japan, Canada, Germany, France, Argentina, Netherlands, Brazil
Medical University of Graz

Completed

Evaluation of an App for Smartphones for People With a Bipolar Affective Disorder

Bipolar Affective Disorder

Austria
Austrian Academic Institute for Clinical Nutrition
Medical University of Vienna

Completed

Effects of Soy Protein on Cholesterol Levels in Children Affected With Familial Hypercholesterolemia (SOYFIT)

Heterozygous Familial Hypercholesterolemia

Austria
Medical University of Vienna

Unknown

Effect of Rapeseed Oil and Sunflower Oil

Familial Hypercholesterolemia

Austria
Medical University of Vienna

Unknown

Soy Food Intervention Trial (SOYFIT)

Familial Hypercholesterolemia

Austria
Orthopedic Hospital Vienna Speising

Completed

Foot Disorders in X-linked Hypophosphatemia

X-linked Hypophosphatemia

Austria
Klinikum Wels-Grieskirchen
Genzyme, a Sanofi Company

Completed

Anderson-Fabry Disease in Chronic Kidney Disease Patients Not on Renal Replacement Therapy

Focus of Study: Prevalence of Fabry Disease in CKD Population

Austria
Medical University of Vienna
Medical University of Graz

Unknown

Viennese Prevalence Study of Anderson-Fabry Disease (VIEPAF)

Fabry Disease | Left Ventricular Hypertrophy

Austria
Medical University Innsbruck

Unknown

Characterization of Two Novel Mutations in the Apob Gene

Familial Hypobetalipoproteinaemia - Heterozygous Form | Low-LDL-syndrome

Austria
Medical University of Vienna

Completed

Recombinant Factor VIIa (NovoSeven) on Restoring Coagulation Activation

Hemophilia A

Austria
Medical University of Vienna

Completed

BT200 in Hereditary Bleeding Disorders

Hemophilia A | Von Willebrand Diseases

Austria
FH Campus Wien, University of Applied Sciences
Medical University of Vienna; Shire

Completed

Gait Examination in Patients With Hemophilia in Austria (JOSEPHA)

Hemophilia

Austria
Medical University Innsbruck

Unknown

Human Solute Carrier Family 5 Member 2 (SLC5A2) Deficiency and the Glucagon-Incretin Axis

Cardiovascular Diseases | Diabetes Mellitus | Familial Renal Glucosuria

Austria
The University of Western Australia
National Health and Medical Research Council, Australia

Unknown

Postprandial Lipid Metabolism in Familial Hypercholesterolaemia:Effects of Fish Oils (FIFH)

Familial Hypercholesterolaemia | Hypertriglyceridaemia

Australia
Tirol Kiniken GmbH
LFB BIOMEDICAMENTS

Unknown

Von Willebrand Factor Concentrate During ECMO Support

Acquired Von Willebrand Disease

Austria
Regeneron Pharmaceuticals

Completed

Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia

United States, Australia, Austria, Netherlands, Taiwan, Ukraine
Bond Biosciences

Terminated

A Study to Evaluate BBI-001 in Hereditary Haemochromatosis (HH) Patients and Iron Deficient Volunteers

Hereditary Hemochromatosis

Australia
Shire

Completed

Home Therapy With VPRIV in Gaucher's Disease

Gaucher Disease

Austria, Germany
Medical University of Vienna

Completed

Effects of Creatine Supplementation in Rett Syndrome

Rett Syndrome

Austria
Glycomine, Inc.

Recruiting

24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG

Pmm2-CDG

Spain, Australia, United States
Murdoch Childrens Research Institute
Melbourne Health; Austin Health; Royal Brisbane and Women's Hospital; The University... and other collaborators

Completed

Mi-iron - Moderately Increased Iron - is Reducing Iron Overload Necessary? (Mi-iron)

Hereditary Haemochromatosis

Australia
Arrowhead Pharmaceuticals

Completed

Study of ARO-ANG3 in Healthy Volunteers and in Dyslipidemic Patients

Dyslipidemias | Hypertriglyceridemia | Familial Hypercholesterolemia

Australia, New Zealand
Amicus Therapeutics

Completed

Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease

Fabry Disease

France, Austria, United States, Belgium, Japan, United Kingdom, Denmark, Brazil, Australia, Italy
Academisch Medisch Centrum - Universiteit van Amsterdam...

Unknown

The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B (DYNAMO)

Hemophilia

Netherlands, Austria, Australia, Belgium, Canada, Finland, Germany, Italy, Spain, United Kingdom
ISU Abxis Co., Ltd.

Completed

A Phase I Study to Compare Abcertin and EU-sourced Cerezyme® in Healthy Volunteers

Gaucher Disease

Australia
Novo Nordisk A/S

Terminated

A Multi-centre, Open Labelled, Multiple Dosing Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of NNC 0172-2021 Administered Subcutaneously to Healthy Male Subjects and Haemophilia Subjects (explorer™2)

Healthy | Congenital Bleeding Disorder | Haemophilia A

Austria, Spain, United Kingdom, France, Germany
Alexion

Completed

Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.

Wilson Disease

United States
Alexion

Completed

Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants

Wilson Disease

United States
Regeneron Pharmaceuticals

Completed

Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia

United States, Netherlands, Australia, Austria, Canada, France, Greece, Italy, Japan, South Africa, Ukraine
Bayer

Terminated

Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors

Hemophilia A and B

Australia, United Kingdom, Taiwan, Japan, Korea, Republic of, Austria, Bulgaria, Hungary, Italy, France, New Zealand
Genzyme, a Sanofi Company

Terminated

Study of the Effects of Fabrazyme Treatment on Lactation and Infants

Fabry Disease | Alpha Galactosidase A Deficiency

United States, Austria, United Kingdom
Hospital Rudolfstiftung
Oesterreichische Muskelforschung

Completed

Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-study

Carrier of Duchenne Muscular Dystrophy

Austria
Archemix Corp.

Completed

ARC1779 Injection in Patients With Von Willebrand Factor-Related Platelet Function Disorders

Purpura, Thrombotic Thrombocytopenic | Von Willebrand Disease Type-2b

Austria
Bayer

Active, not recruiting

Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding (HA-SAFE)

Hemophilia A

Spain, Germany, Austria, Greece, Italy, Slovenia
Amicus Therapeutics

Completed

A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects

Fabry Disease

Australia
Arrowhead Pharmaceuticals

Completed

Study of ARO-APOC3 in Healthy Volunteers, Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome (FCS)

Hypertriglyceridemia | Familial Chylomicronemia

Australia, Canada, New Zealand
4D Molecular Therapeutics

Active, not recruiting

4D-310 in Adults With Fabry Disease and Cardiac Involvement

Fabry Disease

Taiwan, Australia
Amicus Therapeutics

Completed

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

Fabry Disease

Australia, Brazil
Bayer

Completed

Non-interventional Observation of Practical Implementation, Efficacy and Safety of Continuous Infusion With KOGENATE Bayer in Surgery (NO PEACKS)

Hemophilia A

Germany, Spain, Austria, Italy, Slovenia, Greece, Netherlands
Amicus Therapeutics

Completed

Migalastat Food Effect Study

Fabry Disease

United States
Kyowa Kirin Pharmaceutical Development Ltd

Completed

Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age

X-linked Hypophosphatemia (XLH)

Sweden, Spain, United Kingdom, Italy, Austria, France
Arrowhead Pharmaceuticals

Active, not recruiting

Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH) (Gateway)

Homozygous Familial Hypercholesterolemia

Australia, Canada, United States, South Africa
Melbourne Health
Sanofi

Withdrawn

Complement Activation in the Lysosomal Storage Disorders (CATALYST)

Lysosomal Storage Diseases | Fabry Disease | Niemann-Pick Disease, Type C | Gaucher Disease

Australia
University College, London
University of Sydney; University Hospital Birmingham

Unknown

Native T1 Mapping by Cardiovascular Magnetic Resonance Imaging in Rare Diseases (FABRY400)

Fabry Disease

United Kingdom, Australia
The University of Western Australia
Sir Charles Gairdner Hospital; Perron Institute for Neurological and Translational...

Withdrawn

Optimising Anterior Pallidal Deep Brain Stimulation for Tourette's Syndrome

Tourette's Syndrome

Australia
Bayer

Completed

International PMS Study - KOGENATE Bayer

Hemophilia A

Switzerland, France, Spain, Sweden, Austria, Italy, Belgium, Denmark, Greece, Netherlands
Alexion Pharmaceuticals

Completed

Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients

Wilson Disease

United States, Germany, Austria, Poland, United Kingdom
Alexion

Completed

The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment

Wilson Disease

United States, Germany, Austria, Poland, United Kingdom

Clinical Trials on Urea Cycle Disorders, Inborn in Austria

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