Clinical Trials on Dominantly Inherited Stargardt's Disease (STGD3) in Austria

Total 166 results

Pfizer

Active, not recruiting

Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

Hemophilia B

United States, Canada, Australia, Turkey
Baxalta now part of Shire

Completed

Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients

Hemophilia A

United States, France, United Kingdom, Spain, Germany, Canada, Puerto Rico, Italy, Sweden, Austria
Krystal Biotech, Inc.

Completed

A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa

Dystrophic Epidermolysis Bullosa | Recessive Dystrophic Epidermolysis Bullosa | Dominant Dystrophic Epidermolysis Bullosa | DEB - Dystrophic Epidermolysis Bullosa

United States
Reata, a wholly owned subsidiary of Biogen

Completed

A Phase 2 Trial of the Safety and Efficacy of Bardoxolone Methyl in Patients With Rare Chronic Kidney Diseases - PHOENIX (PHOENIX)

Focal Segmental Glomerulosclerosis | IgA Nephropathy | Autosomal Dominant Polycystic Kidney | CKD Associated With Type 1 Diabetes

United States
Fondazione Angelo Bianchi Bonomi
Sintesi Research Srl

Completed

Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET)

Hemophilia A

United States, India, Spain, Argentina, Austria, Brazil, Chile, Egypt, Iran, Islamic Republic of, Italy, Mexico, Saudi Arabia, South Africa, Turkey
Novo Nordisk A/S

Completed

A Research Study Investigating Mim8 in People With Haemophilia A (FRONTIER1)

Healthy Volunteers | Haemophilia A With or Without Inhibitors

United States, Germany, Spain, Austria, Bulgaria, Italy, Japan, South Africa, Switzerland, Turkey, United Kingdom, Poland
Genzyme, a Sanofi Company

Completed

A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis (ATLAS-PPX)

Hemophilia

United States, Australia, China, Denmark, France, Ireland, Israel, Italy, Japan, Korea, Republic of, Malaysia, Turkey, Ukraine, United Kingdom, Mexico
Pfizer

Active, not recruiting

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2)

Hemophilia B

Germany, United Kingdom, Canada, Australia, Taiwan, Japan, France, Turkey, Korea, Republic of, United States, Brazil, Greece, Saudi Arabia, Sweden
Genzyme, a Sanofi Company

Active, not recruiting

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX (ATLAS-OLE)

Hemophilia

China, Hungary, Turkey, Canada, Korea, Republic of, United States, Australia, Denmark, France, Germany, India, Ireland, Israel, Italy, Japan, Malaysia, South Africa, Taiwan, Ukraine, United Kingdom
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
Novo Nordisk A/S

Completed

Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia A (alleviate 1)

Congenital Bleeding Disorder | Haemophilia A

United States, Austria, Serbia, Germany, Japan, France
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B

Severe Hemophilia B

Sweden, United States, France, Italy, Russian Federation, United Kingdom, Germany, China, Poland, Japan, Australia, Brazil, Canada, India, South Africa, Hong Kong, Belgium
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A (Kids ALONG)

Hemophilia A

United States, Poland, United Kingdom, Netherlands, South Africa, Hong Kong, Australia, Ireland
Baxalta now part of Shire
Takeda Development Center Americas, Inc.; Baxalta Innovations GmbH, now part...

Active, not recruiting

A Study of BAX 888 in Male Adults With Severe Hemophilia A

Hemophilia A

United States, Spain, Hungary, Italy, Germany, Austria, France
Jaeb Center for Health Research
Foundation Fighting Blindness

Recruiting

Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants (Uni-Rare)

Retinitis Pigmentosa | Inherited Retinal Degeneration

United States, United Kingdom, France, Finland, Israel, Netherlands, Switzerland, Australia, Canada, Brazil, Mexico
Bayer

Completed

Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A

Hemophilia A

Belgium, New Zealand, Netherlands, Greece, United Kingdom, Austria, United States, Italy, Romania, Bulgaria, Canada, Israel, Poland, Lithuania, Norway, Argentina, Spain
Pfizer

Recruiting

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B (BASIS KIDS)

Hemophilia A | Hemophilia B

Denmark, Taiwan, Canada, Turkey, Spain, China, United Kingdom, South Africa, Italy, Saudi Arabia, Israel, Korea, Republic of, Austria, Germany, Japan, Australia, India, Slovakia, France, United States, Brazil, Argentina
Pfizer

Active, not recruiting

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

Hemophilia A | Hemophilia B

United States, Canada, Spain, Australia, Korea, Republic of, Taiwan, Brazil, Italy, Japan, Turkey, Germany, United Kingdom, Saudi Arabia, France, Sweden, Belgium, Greece, Israel
Baxalta now part of Shire
Takeda Development Center Americas, Inc.

Recruiting

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)

Von Willebrand Disease

United States, Belgium, Spain, Italy, Turkey, Germany, Austria, France, Czechia, Russian Federation, Ukraine, United Kingdom, Netherlands
Pfizer

Terminated

Study Evaluating Safety Of Patients Switching To ReFacto AF In Usual Care Settings

Hemophilia A

Netherlands, Spain, Greece, Italy, France, Belgium, Germany, Austria, Sweden, Hungary, Finland, United Kingdom, Denmark, Romania
Hoffmann-La Roche

Completed

A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors (STASEY)

Hemophilia A

Canada, Australia, Belgium, India, Germany, Israel, Italy, Spain, Colombia, United Kingdom, Netherlands, Saudi Arabia, Hungary, Guatemala, Russian Federation, Poland, Switzerland, Finland, Panama, Brazil, Mexico, Portugal, Romania, Sw...
Bioverativ, a Sanofi company

Active, not recruiting

Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A (XTEND-ed)

Hemophilia A

United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, China, France, Germany, Greece, Hungary, Ireland, Italy, Japan, Korea, Republic of, Netherlands, Spain, Sweden, Switzerland, Taiwan, Turkey, United Kingdom
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
PedNet Haemophilia Research Foundation
Lund University Hospital

Recruiting

The European Paediatric Network for Haemophilia Management ( PedNet Registry) (PedNet)

Factor IX Deficiency | Factor VIII Deficiency

Norway, Austria, Belgium, Canada, Czechia, Denmark, Finland, France, Germany, Greece, Ireland, Israel, Italy, Netherlands, Portugal, Spain, Sweden, Switzerland, United Kingdom
Hoffmann-La Roche
Chugai Pharmaceutical

Completed

A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors (HAVEN 1)

Hemophilia A

Korea, Republic of, United States, France, Germany, Italy, South Africa, Spain, Japan, United Kingdom, Poland, Taiwan, Australia, Costa Rica, New Zealand
Wyeth is now a wholly owned subsidiary of Pfizer

Completed

Study to Establish Bioequivalence of ReFacto AF (BDDrFVIII) With Advate (FLrFVIII) in Hemophilia A

Hemophilia A

Poland, Belgium, France, Italy, Spain, United States, Australia, Netherlands, New Zealand, Sweden, Germany, Finland, Hungary
Bioverativ Therapeutics Inc.

Completed

Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B (B-YOND)

Severe Hemophilia B

United States, Italy, Belgium, Australia, Brazil, Hong Kong, India, Ireland, Japan, South Africa, United Kingdom, Netherlands, Sweden, Canada, Poland, China, France, Germany
Novo Nordisk A/S

Active, not recruiting

Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors (explorer7)

Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

Spain, Korea, Republic of, India, Australia, United Kingdom, Russian Federation, Poland, Ukraine, Thailand, Mexico, United States, Turkey, Japan, South Africa, Serbia, France, Croatia, Denmark, Algeria, Bulgaria, Malaysia, Portugal, Slovaki... and more
ApcinteX Ltd
Centessa Pharmaceuticals plc

Recruiting

A Study of SerpinPC in Participants With Hemophilia B (HemB) With Inhibitors (PRESent-3)

Hemophilia B | Hemophilia B With Inhibitor

United States, Spain, Australia, Taiwan, France, United Kingdom, Italy, Turkey, Armenia, India, Germany
Sanford Health
National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators

Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)

Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions

United States, Australia
Bayer

Completed

A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A (PROTECT-VIII)

Hemophilia A

Korea, Republic of, Taiwan, Turkey, Singapore, United States, Netherlands, Austria, Denmark, United Kingdom, Belgium, Italy, France, Japan, Germany, Israel, Poland, Canada, Norway, Romania, Colombia
Bioverativ, a Sanofi company

Completed

Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A (XTEND-Kids)

Hemophilia A

Turkey, Australia, Canada, France, Germany, Hungary, Ireland, Italy, Netherlands, Spain, Sweden, Switzerland, Taiwan, United Kingdom, United States
Pfizer

Active, not recruiting

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults (AFFINE)

Hemophilia A

Spain, Taiwan, Australia, United States, Japan, France, Turkey, Germany, Korea, Republic of, United Kingdom, Saudi Arabia, Canada, Greece, Sweden, Brazil, Italy
Baxalta now part of Shire
Takeda Development Center Americas, Inc.

Active, not recruiting

A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A

Hemophilia A

Spain, United States, Denmark, Italy, Taiwan, Malaysia, Belgium, Thailand, Turkey, Austria, Bulgaria, Canada, Finland, France, Germany, Hong Kong, Hungary, Korea, Republic of, Netherlands, Norway, Singapore, Ukraine, United Kingdom
Novo Nordisk A/S

Completed

Observational Study on Safety of Room Temperature Stable NovoSeven® in Patients With Haemophilia A or B

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Belgium, Germany, France, United Kingdom, Italy, Hungary, Austria, Sweden, Iran, Islamic Republic of, Argentina, Ireland, Poland, Portugal, Slovakia
BioMarin Pharmaceutical

Active, not recruiting

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) (BMN 270-301)

Hemophilia A

United States, Taiwan, United Kingdom, Australia, Israel, Belgium, Brazil, France, Germany, Italy, Korea, Republic of, South Africa, Spain
Bayer

Completed

Trial to Evaluate the Efficacy and Safety of a New Full Length Recombinant Human FVIII for Hemophilia A (Leopold I)

Hemophilia A | Blood Coagulation Disorders

Spain, Taiwan, Austria, United States, Germany, Thailand, United Kingdom, Italy, Turkey, Croatia, Serbia, Poland, Israel, Sweden, Indonesia, Argentina, Norway, South Africa, India, Hong Kong, Denmark, Pakistan
Novo Nordisk A/S

Completed

Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B (paradigm™6)

Congenital Bleeding Disorder | Haemophilia B

Spain, United Kingdom, Australia, Taiwan, France, Malaysia, United States, Austria, Canada, Israel, Japan, Thailand, Algeria, Argentina
Bioverativ, a Sanofi company

Completed

A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A (XTEND-1)

Factor VIII Deficiency

United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, France, Germany, Greece, Hungary, Italy, Japan, Korea, Republic of, Mexico, Netherlands, Spain, Taiwan, United Kingdom
Novo Nordisk A/S

Completed

Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B With Inhibitors (ONE)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

Saudi Arabia, Algeria, Turkey, Germany, Poland, Netherlands, Italy, Belgium, South Africa, United Kingdom, Sweden, Austria, Portugal, France, Czech Republic, Venezuela
Novo Nordisk A/S

Completed

Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A (pathfinder™6)

Congenital Bleeding Disorder | Haemophilia A

United States, Spain, Australia, France, Thailand, Italy, Malaysia, Austria, Greece, Japan, Taiwan, Canada, Israel, Algeria, Argentina, Bulgaria, Germany, Romania
Wyeth is now a wholly owned subsidiary of Pfizer

Completed

Study to Describe the Allergic Reactions to Factor IX in Patients With Hemophilia B

Hemophilia B | Allergic Reactions

France, Italy, United States, Austria, Belgium, Spain, United Kingdom, Canada, Germany
Genzyme, a Sanofi Company

Completed

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors (ATLAS-INH)

Hemophilia A | Hemophilia B

United States, Australia, Canada, China, France, Korea, Republic of, Malaysia, Turkey, Ukraine, United Kingdom, Germany, India, Italy, Japan, South Africa, Spain, Taiwan
Bioverativ, a Sanofi company
Swedish Orphan Biovitrum

Completed

Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B (PUPs B-LONG)

Hemophilia B

United States, Italy, Ireland, New Zealand, United Kingdom, Australia, Netherlands, Denmark, Poland, Sweden, France
Genzyme, a Sanofi Company

Completed

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

Hemophilia A | Hemophilia B

United States, Australia, China, Denmark, France, Israel, Japan, Korea, Republic of, Malaysia, Turkey, Ukraine, United Kingdom, Germany, India, South Africa, Spain, Taiwan, Hungary, Italy
ApcinteX Ltd
Centessa Pharmaceuticals plc

Recruiting

The Efficacy and Safety of SerpinPC in Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B (PRESent-2)

Hemophilia A | Hemophilia B

United States, Italy, Spain, Taiwan, Australia, Belgium, United Kingdom, France, Germany, India, South Africa, Poland, Canada, Armenia, Turkey
Novo Nordisk A/S

Completed

Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725 (mentor™2)

Congenital Bleeding Disorder | Congenital FXIII Deficiency

France, United States, Israel, Spain, Switzerland, Germany, Canada, Japan, United Kingdom, Italy, Finland, Austria
ApcinteX Ltd
Centessa Pharmaceuticals plc

Recruiting

Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)

Hemophilia A | Hemophilia B

Spain, Taiwan, United Kingdom, Italy, Germany, United States, South Africa, Australia, Poland, Armenia, France, India, Canada, Brazil, Turkey, Egypt
Novo Nordisk A/S

Completed

Safety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia A (guardian™ 5)

Congenital Bleeding Disorder | Haemophilia A

Italy, Sweden, Netherlands, Austria, Spain, United States, Switzerland, Germany, France, Hungary, Greece, Slovenia, Czechia, Slovakia, Poland
Novo Nordisk A/S

Completed

A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors (explorer™4)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

United States, Austria, Spain, Japan, Croatia, Italy, United Kingdom, Israel, Sweden, Ukraine, Malaysia, Greece, Denmark, Canada

Clinical Trials on Dominantly Inherited Stargardt's Disease (STGD3) in Austria

Sponsors and Collaborators

Medical Conditions

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CROs by country

CROs in Vietnam

Conditions

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Drug Interventions

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