Clinical Trials on Rare Genetic Disorders in Austria

Total 10718 results

Medical University of Graz

Completed

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

Bipolar Affective Disorder

Austria
Medical University of Vienna

Completed

Effects of Creatine Supplementation in Rett Syndrome

Rett Syndrome

Austria
Medical University of Vienna

Completed

Recombinant Factor VIIa (NovoSeven) on Restoring Coagulation Activation

Hemophilia A

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
Hospital Rudolfstiftung
Oesterreichische Muskelforschung

Completed

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

Carrier of Duchenne Muscular Dystrophy

Austria
Medical University of Vienna

Completed

BT200 in Hereditary Bleeding Disorders

Hemophilia A | Von Willebrand Diseases

Austria
Tirol Kiniken GmbH
LFB BIOMEDICAMENTS

Unknown

Von Willebrand Factor Concentrate During ECMO Support

Acquired Von Willebrand Disease

Austria
RHEACELL GmbH & Co. KG

Recruiting

Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III, Cross-over)

Epidermolysis Bullosa

Austria
Holostem Terapie Avanzate s.r.l.
Paracelsus Medical University

Terminated

Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With JEB (HOLOGENE17) (HOLOGENE17)

Junctional Epidermolysis Bullosa

Austria
Stefan Woehrl
Medical University of Vienna

Completed

Skin Testing With Recombinant Bet v 1 and Hypoallergenic Recombinant Bet v 1 Fragments

Atopic Dermatitis

Austria
Medical University of Vienna

Completed

FACE-Q in Blepharoplasty

Dermatochalasis of Upper Eyelid

Austria
Medical University of Vienna

Recruiting

Ketamine and Stress in OCD (KET-OCD)

Obsessive-Compulsive Disorder | Psychiatric Disorder

Austria
Daniel Doberer

Completed

Effects of Heme Arginate in Healthy Male Subjects (HEMAHS)

Healthy Subjects | Genetic Polymorphism | Heme Oxygenase

Austria
CSL Behring

Completed

A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B

Hemophilia B

Germany, Australia, Italy, Israel, Czech Republic, Spain, France, Canada, Austria, Russian Federation
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
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
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
Medical University of Vienna

Recruiting

The Effect of Ketamine on Aesthetics and Role for Antidepressant Effects

Major Depressive Disorder

Austria
Holostem Terapie Avanzate s.r.l.
Paracelsus Medical University

Terminated

Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB. (HOLOGENE7)

Recessive Dystrophic Epidermolysis Bullosa

Austria
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
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
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
Prof. Johann Bauer

Recruiting

Rigosertib for RDEB-SCC

Epidermolysis Bullosa Dystrophica | Squamous Cell Carcinoma

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
Medical University of Graz
University of Graz

Recruiting

CBT Training for Cognitive Reappraisal as an Intervention for Patients With Functional Tremor - an EEG Study

Functional Movement Disorder

Austria
Murdoch Childrens Research Institute
University of Sydney; Sydney Children's Hospitals Network; Women's and Children... and other collaborators

Active, not recruiting

Mackenzie's Mission: The Australian Reproductive Carrier Screening Project

Autosomal Recessive Disorder | X-Linked Genetic Diseases

Australia
Medical University of Graz

Completed

Use of an SPF30 Sunscreen and an After-sun-lotion in Skin Cancer Risk Patients

Skin Cancer | Basal Cell Nevus Syndrome | Xeroderma Pigmentosum

Austria
Medical University of Vienna

Completed

Comparison of UVA1 Phototherapy Versus Photochemotherapy for Patients With Severe Generalized Atopic Dermatitis

Atopic Dermatitis

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
Spark Therapeutics
Children's Hospital of Philadelphia; University of Pittsburgh; St. James's Hospital... and other collaborators

Terminated

Hemophilia B Gene Therapy With AAV8 Vector

Hemophilia B

United States, Australia
Spark Therapeutics

Completed

Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants

Hematologic Diseases | Hemophilia A | Blood Coagulation Disorders, Inherited | Coagulation Protein Disorders | Hemorrhagic Disorders | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Blood Coagulation Disorder | Factor VIII Deficiency

United States, Australia, Thailand, Canada
LEO Pharma

Completed

A Multi-omics Disease Signature Trial in Adult Patients With Moderate to Severe AD

Atopic Dermatitis

Austria
Medical University of Vienna

Completed

Does Complement Factor H Gene Polymorphism Play a Role in the Regulation of Vascular Tone in the Choroid?

Macular Degeneration | Ocular Physiology | Regional Blood Flow | Genetic Polymorphism

Austria
Bayer

Completed

International PMS Study - KOGENATE Bayer

Hemophilia A

Switzerland, France, Spain, Sweden, Austria, Italy, Belgium, Denmark, Greece, Netherlands
Oslo University Hospital
St John of God Hospital, Vienna; Nepean Blue Mountains Local Health District

Completed

Bowel ENDOmetriosis; Evaluation of Diagnostics and Quality of Life (ENDO)

Quality of Life | Pain, Chronic | Deep Endometriosis | Bowel Dysfunction | Endometriosis Related Pain | Endometriosis Rectum | Deep Infiltrating Endometriosis

Norway, Australia, Austria
Pfizer

Completed

Registry For Patients Treated With BeneFix In Usual Care Setting In Germany

Hemophilia B

Germany, Austria
Michael Frass

Completed

Homeopathic Treatment of Children Suffering From PFAPA (HOMPFAPA)

Periodic Fever Syndrome

Austria
Kiora Pharmaceuticals, Inc.

Recruiting

A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS) (ABACUS)

Retinitis Pigmentosa | Choroideremia

Australia
NORCE Norwegian Research Centre AS
The Research Council of Norway; Helse Stavanger HF; Helse Vest; Helse Forde; University... and other collaborators

Completed

Resource-oriented Music Therapy for Psychiatric Patients With Low Therapy Motivation

Personality Disorders | Mental Disorders | Mood Disorders | Schizophrenia and Disorders With Psychotic Features

Norway, Australia, Austria
Shire

Completed

Short Term Observational Study in DEB Patients

Dystrophic Epidermolysis Bullosa

United States, Czechia, Italy, Austria, Australia, France, Germany
Medical University Innsbruck

Unknown

Characterization of Two Novel Mutations in the Apob Gene

Familial Hypobetalipoproteinaemia - Heterozygous Form | Low-LDL-syndrome

Austria
CSL Behring

Terminated

Study of Biostate for Treatment of Children With Hemophilia A Complicated by Antibody Development

Hemophilia A

Russian Federation, Germany, Italy, Austria, Greece
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
Novo Nordisk A/S

Completed

Safety of NNC 0172-0000-2021 in Healthy Male Subjects and Subjects With Haemophilia A or B (Explorer 1)

Healthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Austria, Sweden, Spain, Switzerland, United Kingdom, Germany, Malaysia, Denmark, South Africa, Thailand, Italy
Medical University of Vienna

Unknown

Soy Food Intervention Trial (SOYFIT)

Familial Hypercholesterolemia

Austria
Sciotec Diagnostic Technologies GmbH

Not yet recruiting

Observational Study on the Safety and Efficacy of the Medical Product Fructosin.

Fructose Intolerance

Austria
Medical University of Vienna

Unknown

Patient Stratification and Treatment Response Prediction in Neuropharmacotherapy Using Hybrid Positron Emmission Tomography/Magnetic Resconance Imaging (PET/MR)

Major Depressive Disorder

Austria
AOP Orphan Pharmaceuticals AG

Completed

Lancovutide (Moli1901) Inhalation Solution Study in Adolescents and Adults With Cystic Fibrosis

Cystic Fibrosis

Austria

Clinical Trials on Rare Genetic Disorders in Austria

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Belgium

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations