Clinical Trials on Congenital Abnormalities in Austria

Total 1416 results

Shire

Completed

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment (AIM-IT)

Hunter Syndrome

Spain, United States, Canada, United Kingdom, France, Australia, Mexico
CHDI Foundation, Inc.
Monash University

Completed

Cognitive Assessment Battery (CAB) Beta Study (CAB)

Huntington's Disease

United States, Australia, United Kingdom, Canada
Amgen

Active, not recruiting

A Study Assessing Rocatinlimab in Combination With Topical Corticosteroid and/or Topical Calcineurin Inhibitors in Adult Participants With Moderate-to-severe Atopic Dermatitis (AD) (ROCKET-SHUTTLE)

Atopic Dermatitis

Belgium, Spain, China, Germany, United States, Austria, France, Hungary, Switzerland, Canada, United Kingdom, Bulgaria, Australia, Netherlands, Malaysia, Singapore, Slovenia, Italy, Japan, Argentina, Greece, Turkey, Poland, Puerto Rico, Romani... and more
Regeneron Pharmaceuticals
Sanofi

Completed

Safety, Pharmacokinetics and Efficacy of Dupilumab in Patients ≥6 Months to <6 Years With Moderate-to-Severe Atopic Dermatitis (Liberty AD PRESCHOOL) (Liberty AD)

Dermatitis, Atopic

United States, Germany, Poland, United Kingdom
Pfizer

Completed

Analgesic Efficacy And Safety of Valdecoxib For Treatment Of Post-Surgical Pain From Bunionectomy Surgery

Pain | Hallux Valgus

United States
Regeneron Pharmaceuticals
Sanofi

Completed

Study of Dupilumab and Immune Responses in Adults With Atopic Dermatitis (AD)

Atopic Dermatitis

United States
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
Bayer
Regeneron Pharmaceuticals

Completed

Aflibercept for Retinopathy of Prematurity - Intravitreal Injection Versus Laser Therapy (FIREFLEYE)

Retinopathy of Prematurity (ROP)

Spain, Singapore, Hong Kong, Korea, Republic of, Malaysia, Japan, Taiwan, Sweden, Portugal, Belgium, Argentina, Bulgaria, Italy, Austria, Brazil, Czechia, Greece, Hungary, Israel, Netherlands, Poland, Romania, Russian Federation, Slov... and more
Sanofi
Regeneron Pharmaceuticals

Completed

SAR231893-LPS15497- "Dupilumab Effect on Sleep in AD Patients"

Atopic Dermatitis

France, United States, Australia, Germany, Israel, Italy, Spain, Switzerland, United Arab Emirates, United Kingdom
Seelos Therapeutics, Inc.

Terminated

STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia (STRIDES)

Spinocerebellar Ataxia Type 3

United States, Spain, Korea, Republic of, Australia, United Kingdom, Brazil, Germany, Portugal
Jazz Pharmaceuticals
GW Pharmaceuticals

Terminated

A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome

Rett Syndrome | RTT

United States, Canada, Italy, Spain, United Kingdom, Australia
Wave Life Sciences Ltd.

Recruiting

Study of WVE-003 in Patients With Huntington's Disease

Huntington Disease

Canada, Spain, Australia, Denmark, Netherlands, Poland, United Kingdom, France, Germany, Italy
Hoffmann-La Roche

Completed

An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington's Disease Participants Who Participated in Prior Roche and Genentech Sponsored Studies

Huntington Disease

United States, Canada, Netherlands, Germany, Spain, United Kingdom, Austria, Italy
University of Minnesota
University of Colorado, Denver; United States Department of Defense

Completed

Sigh Ventilation to Increase Ventilator-Free Days in Victims of Trauma at Risk for Acute Respiratory Distress Syndrome (SiVent)

Acute Respiratory Distress Syndrome

United States
Mitsubishi Tanabe Pharma America Inc.

Completed

Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria

EPP | XLP

Spain, United States, Germany, Norway, Italy, Australia, Canada, Japan, Sweden, United Kingdom
Shionogi
Integrium

Completed

A Study to Evaluate Daily Pravastatin, Fenofibrate or Pravafen in the Treatment of Combined Hyperlipidemia

Combined Hyperlipidemia

United States
Jiangsu Atom Bioscience and Pharmaceutical Co.,...

Recruiting

Phase 2b/3 Study to Assess ABP-671 a Novel URAT1 Inhibitor in Participants With Gout

Gout

United States, Georgia, Australia, Guatemala, Taiwan
AbbVie

Enrolling by invitation

A Study to Evaluate Upadacitinib in Combination With Topical Corticosteroids in Adolescent and Adult Participants With Moderate to Severe Atopic Dermatitis (AD Up)

Atopic Dermatitis

United States, Australia, Austria, Belgium, Canada, China, Czechia, France, Germany, Greece, Hong Kong, Hungary, Ireland, Israel, Italy, Japan, Netherlands, New Zealand, Norway, Puerto Rico, Slovakia, Spain, Sweden, United Kingdom
LEO Pharma

Completed

Tralokinumab Monotherapy for Moderate to Severe Atopic Dermatitis - ECZTRA 2 (ECZema TRAlokinumab Trial no. 2) (ECZTRA 2)

Atopic Dermatitis

United States, Denmark, United Kingdom, Korea, Republic of, Canada, Italy, Australia, Poland, Russian Federation
Dyne Therapeutics

Recruiting

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER)

Duchenne Muscular Dystrophy (DMD)

United States, Belgium, Canada, United Kingdom, Australia, Spain, Italy
Ultragenyx Pharmaceutical Inc
Mereo BioPharma

Active, not recruiting

Study to Assess Dose, Efficacy and Safety of Setrusumab in Participants With Osteogenesis Imperfecta (Orbit)

Osteogenesis Imperfecta

United States, Turkey, Germany, United Kingdom, Italy, Portugal, Canada, Australia, Argentina, France, Netherlands, Poland
Christoph Hornik
National Heart, Lung, and Blood Institute (NHLBI); University of North Carolina...

Recruiting

Safety of Sildenafil in Premature Infants With Severe Bronchopulmonary Dysplasia (SILDI-SAFE)

Bronchopulmonary Dysplasia of Newborn

United States
Sanofi

Active, not recruiting

Evaluation of Biomarkers of Atopic Dermatitis in Pediatric Patients (PEDISTAD BIOMARKER STUDY)

Dermatitis Atopic

Australia, United States, Argentina, Brazil, Canada, Colombia, France, Mexico, Netherlands
Sanofi
Regeneron Pharmaceuticals

Completed

Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy (ODYSSEY FH I)

Hypercholesterolemia

United States, Austria, Canada, Czech Republic, Denmark, France, Israel, Netherlands, Norway, Russian Federation, South Africa, Spain, Sweden, United Kingdom
Ardea Biosciences, Inc.

Completed

Allopurinol Outcome Study (LASSO)

Gout

United States, South Africa, Canada, Belgium, New Zealand, Australia, Germany
LEO Pharma

Completed

Vaccine Responses in Tralokinumab-Treated Atopic Dermatitis - ECZTRA 5 (ECZema TRAlokinumab Trial No. 5) (ECZTRA 5)

Atopic Dermatitis

United States, Canada
Massachusetts General Hospital
National Center for Complementary and Integrative Health (NCCIH); University...

Terminated

Creatine Safety, Tolerability, & Efficacy in Huntington's Disease (CREST-E) (CREST-E)

Huntington's Disease

United States, Canada, New Zealand, Australia
HP Therapeutics Foundation
Huntington Study Group

Unknown

Cooperative Huntington's Observational Research Trial

Huntington Disease

United States, Canada, Australia
Swedish Orphan Biovitrum

Completed

Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care (OPAL)

Hereditary Tyrosinemia, Type I

Belgium, Denmark, France, Germany, Austria, Czechia, Finland, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom
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
Novartis Pharmaceuticals

Recruiting

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials (SPECTRUM)

Spinal Muscular Atrophy (SMA)

Spain, Canada, Taiwan, Belgium, Malaysia, China, Japan, Italy, Singapore, United Kingdom, France, Australia, Thailand, Denmark, United States
Santhera Pharmaceuticals

Terminated

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids (SIDEROS)

Duchenne Muscular Dystrophy (DMD)

United States, Spain, Netherlands, Sweden, Germany, France, Belgium, United Kingdom, Italy, Ireland, Switzerland, Austria, Bulgaria, Hungary, Israel
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
KalVista Pharmaceuticals, Ltd.

Recruiting

An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, Bulgaria, France, Greece, Hungary, Israel, Italy, Netherlands, New Zealand, North Macedonia, Poland, Spain, United Kingdom, Australia, Germany, South Africa, Austria, Portugal, Slovakia, Romania, Canada, Saudi Arabia, Japa...
Protalix
Chiesi Farmaceutici S.p.A.

Completed

Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

Fabry Disease

United States, Serbia, Spain, United Kingdom, Australia, Paraguay
Seattle Children's Hospital
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Cystic...

Completed

Baby Observational and Nutritional Study (BONUS)

Cystic Fibrosis | Growth Failure | Exocrine Pancreatic Insufficiency

United States
Parion Sciences
Vertex Pharmaceuticals Incorporated

Completed

Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF) (CLEAN-CF)

Cystic Fibrosis

United States
Janssen Research & Development, LLC

Completed

A Study to Characterize the Clinical Course of Pregnant Women and Children at High Risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn

Erythroblastosis, Fetal | Early Onset Severe Hemolytic Disease of the Fetus and Newborn (EOS-HDFN)

United States, Canada, Netherlands, Belgium, Australia, Germany, Spain, Sweden, United Kingdom
Alexion Pharmaceuticals, Inc.

Terminated

Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease

Wilson Disease

Spain, Korea, Republic of, France, Germany, Japan, Australia, Poland, United Kingdom
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.

Completed

Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

Duchenne Muscular Dystrophy

Korea, Republic of, Chile, Taiwan, China, United States, United Kingdom, Netherlands, Spain, Australia, Norway, Russian Federation, Greece, Italy, Turkey, Mexico, Canada, Hong Kong, New Zealand, Ukraine
Pfizer

Terminated

A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

United States, Australia, Canada, United Kingdom, Japan, Bulgaria, Italy, Poland
BioMarin Pharmaceutical

No longer available

Extension Study of Drisapersen in DMD Subjects

Duchenne Muscular Dystrophy

United States, Germany, United Kingdom, Japan, Netherlands, France, Spain, Korea, Republic of, Italy, Belgium, Taiwan, Argentina, Australia, Bulgaria, Czechia, Israel, Norway, Poland, Russian Federation, Turkey
Actelion

Completed

Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease

Gaucher Disease Type 1

United States, Taiwan, Canada, Spain, Australia, France, Brazil, Hungary, Czechia, Germany, Italy, Netherlands, United Kingdom
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
Selecta Biosciences, Inc.

Completed

A Study to Compare the Efficacy of SEL-212 to KRYSTEXXA® in Gout Participants Refractory to Conventional Therapy (COMPARE)

Chronic Gout

United States
Regeneron Pharmaceuticals
Sanofi

Completed

Open-label Study of Dupilumab in Patients With Atopic Dermatitis

Atopic Dermatitis

Hungary, Netherlands, Spain, United Kingdom, United States, Australia, Austria, Belgium, Bulgaria, Canada, China, Czechia, Denmark, Estonia, Finland, France, Germany, Ireland, Italy, Japan, Korea, Republic of, Lithuania, New Zealand, ... and more
Genzyme, a Sanofi Company

Completed

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease (COMET)

Glycogen Storage Disease Type II;Pompe's Disease

Czechia, Taiwan, Belgium, Germany, Korea, Republic of, United States, Argentina, Australia, Austria, Brazil, Canada, Denmark, France, Hungary, Italy, Japan, Mexico, Netherlands, Poland, Portugal, Russian Federation, Spain, Switzerland, Turk... and more
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
RAPT Therapeutics, Inc.

Terminated

An Efficacy and Safety Study of RPT193 in Adults With Atopic Dermatitis

Atopic Dermatitis

United States, Canada

Clinical Trials on Congenital Abnormalities in Austria

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