Search clinical trials for: Inherited Retinal Disorders

Total 149388 results

Takeda

Completed

A Study on the Outcomes of Recombinant Von Willebrand Factor on Demand Treatment and Prevention and Treatment of Bleeding During and After Surgery in Adults With Inherited Von Willebrand Disease in the United Kingdom (UK)

Von Willebrand Disease (VWD)

United Kingdom
Amgen

Completed

Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia) (HAUSER-OLE)

Familial Hypercholesterolemia

Canada, Italy, Norway, Belgium, United States, Turkey, Australia, Austria, South Africa, Poland, Switzerland, Spain, Portugal, Slovenia, Brazil, Russian Federation, Hungary, United Kingdom, Colombia, Czechia, Netherlands, Greece, Ma...
Kodiak Sciences Inc

Completed

A Study to Evaluate the Efficacy and Safety of Intravitreal KSI-301 Compared With Intravitreal Aflibercept in Participants With Neovascular (Wet) Age-related Macular Degeneration (wAMD) (DAYLIGHT)

Wet Age-related Macular Degeneration

United States, Puerto Rico
Investigación en Hemofilia y Fisioterapia

Completed

Changes in Intra-articular Parameters by Doppler Ultrasound in Patients With Hemophilic Ankle Arthropathy

Hemophilia

Spain
Swedish Orphan Biovitrum
Syneos Health

Active, not recruiting

A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa (FREEDOM)

Hemophilia A, Severe

Germany, Spain, France, Czechia, Netherlands, Ireland, Austria, Croatia, Greece, Italy, Norway, Slovenia, Sweden, United Kingdom
Novo Nordisk A/S

Active, not recruiting

A Research Study Investigating Nonacog Beta Pegol (N9-GP) for Treatment and Prevention of Bleedings in Chinese People With Haemophilia B (Paradigm9)

Haemophilia B

China
Washington University School of Medicine
Hoffmann-La Roche; National Institute on Aging (NIA); Alzheimer's Association

Terminated

Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia: A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation (DIAN-TU)

Dementia | Alzheimers Disease | Alzheimers Disease, Familial

United States, France, Spain, Ireland, Canada, Australia, Puerto Rico, United Kingdom
Janssen Research & Development, LLC

Active, not recruiting

Cognitive Health in Ageing Register: Investigational, Observational and Trial Studies in Dementia Research: Prospective Readiness Cohort Study (CHARIOT:PRO)

Alzheimer's Disease | Plaque, Amyloid

United Kingdom
University of Alabama at Birmingham
National Eye Institute (NEI)

Completed

Dyslipidemia and Diabetic Retinopathy (D&D)

Diabetic Retinopathy | Dyslipidemia

United States
CSL Behring

Active, not recruiting

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

Hemophilia B

United States, Belgium, Netherlands, Denmark, Germany, Ireland, Sweden, United Kingdom
Novartis Pharmaceuticals

Active, not recruiting

Observational Study to Evaluate Fluid Resolution and Effectiveness in Patients Receiving Beovu in Neovascular Age-related Macular Degeneration and Visual Impairment Due to Diabetic Macular Edema (BLUE SKY)

Diabetic Macular Edema (DME) | Neovascular Age-related Macular Degeneration (nAMD)

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
CSL Behring

Active, not recruiting

Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B

Hemophilia B

Netherlands, Germany
Medexus Pharma, Inc.

Completed

Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B

Hemophilia B

Turkey, Brazil, Georgia, Moldova, Republic of, South Africa, Ukraine
Hoffmann-La Roche

Active, not recruiting

A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis (Beyond ABR)

Severe Hemophilia A | Moderate Hemophilia A

Brazil, Germany, Italy, Spain, United States, Turkey, United Kingdom, Tunisia, Canada, Hungary, Morocco, Serbia
University of Colorado, Denver
National Institute on Aging (NIA)

Completed

Advancing Understanding of Transportation Options (AUTO)

Stroke | Multiple Sclerosis | Glaucoma | Brain Injuries | Macular Degeneration | Arthritis | Chronic Obstructive Pulmonary Disease | Parkinson Disease | Restless Legs Syndrome | Diabetic Retinopathy | Insomnia | Retinitis Pigmentosa | Obstructive Sleep Apnea | Dementia | End Stage Renal Disease | Sleep Apnea | Syncope | Seizures and other conditions

United States
BioMarin Pharmaceutical

Active, not recruiting

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A (GENEr8-JPN)

Hemophilia A

Japan
Pfizer
Arena is a wholly owned subsidiary of Pfizer

Completed

A Study to Assess the Safety and Efficacy of Oral Etrasimod in Adult Participants With Eosinophilic Esophagitis (VOYAGE)

Eosinophilic Esophagitis

United States, Spain, Switzerland, Australia, Belgium, Netherlands, Canada, Germany
Takeda

Completed

A Proof-of-concept Study to Reduce Treatment Burden in Haemophilia Participants Receiving Factor VIII and Factor IX Infusions

Hemophilia

France
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
Sangamo Therapeutics

Active, not recruiting

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX (LTFU)

Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II

United States
University Hospital, Montpellier
Hospices Civils de Lyon; University Hospital, Bordeaux; University Hospital,... and other collaborators

Completed

Healthy-related Quality of Life and Physical Activity of Children With Cardiac Malformations (QUALIMYORYTHM)

Dilated Cardiomyopathy | Hypertrophic Cardiomyopathy | Brugada Syndrome | Long QT Syndrome | Arrhythmogenic Right Ventricular Dysplasia | Catecholaminergic Polymorphic Ventricular Tachycardia | Restrictive Cardiomyopathy

France
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
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
BioMarin Pharmaceutical

Enrolling by invitation

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) (GENEr8-LTE)

Hemophilia A

United Kingdom, United States
Immunic AG

Active, not recruiting

MRI Trial to exPlore the efficAcy and Safety of IMU-838 in Relapsing Remitting Multiple Sclerosis (EMPhASIS) (EMPhASIS)

Relapsing-Remitting Multiple Sclerosis (RRMS)

Poland, Bulgaria, Romania, Ukraine
BioMarin Pharmaceutical

Active, not recruiting

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 (GENEr8-AAV5+)

Hemophilia A | Gene Therapy | Blood Disorder | Clotting Disorders

Taiwan, Korea, Republic of, United Kingdom, South Africa
BioMarin Pharmaceutical

Completed

A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A (SAAVY)

Hemophilia A

United States
University Hospital Tuebingen

Completed

Stargardt Disease in Childhood

Stargardt Disease

Germany
Andrea Doria
Beijing Children's Hospital, Baxalta US Inc.

Completed

Imaging of Arthropathy in Boys With Hemophilia in China

Hemophilia

China
CSL Behring

Completed

Registry of Patients With Von WilLEbrand Disease Treated With Voncento® (OPALE)

Von Willebrand Disease

France
CSL Behring

Active, not recruiting

Register of Patients With haEmophilia A tReated With Afstyla® (OPERA)

Haemophilia A

France
Hospices Civils de Lyon

Terminated

Bone Microarchitecture in Men With Hemophilia (HEMOS)

Osteoporosis | Severe Hemophilia A

France
University Hospital Tuebingen

Completed

Clinical and Genetic Findings in Patients With PRPF31-associated Retinitis Pigmentosa

Retinitis Pigmentosa

Germany
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
Jaeb Center for Health Research
Foundation Fighting Blindness

Active, not recruiting

Rate of Progression in USH2A-related Retinal Degeneration (RUSH2A)

Usher Syndrome, Type 2A | Retinitis Pigmentosa 39

United States, Canada, France, Germany, Netherlands, United Kingdom
Swedish Orphan Biovitrum

Active, not recruiting

A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health (A-MORE)

Hemophilia A

Spain, Germany, Czechia, Italy, Estonia, Finland, Greece, Netherlands, Saudi Arabia, Slovenia, Sweden, Switzerland, United Kingdom
Kodiak Sciences Inc

Terminated

A Study to Evaluate the Efficacy and Safety of Intravitreal KSI-301 in Participants With Moderately Severe to Severe Non-proliferative Diabetic Retinopathy (NPDR) (GLOW)

Non-proliferative Diabetic Retinopathy

United States, Latvia, Slovakia, Poland, Puerto Rico, Spain, Czechia
MeiraGTx UK II Ltd
Syne Qua Non Limited

Completed

Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)

Eye Diseases | Retinal Diseases | Eye Diseases, Hereditary | Leber Congenital Amaurosis (LCA)

United States, United Kingdom
Enzyre B.V.
Instytut Hematologii i Transfuzjologii, Warschau, Poland

Completed

Real-life Clinical FEIBA Samples Measured Using the Version A of the HemA EnzySystem

Hemophilia A | Hemophilia A With Inhibitor

Poland
Memorial Sloan Kettering Cancer Center

Active, not recruiting

Determining Whether Intra-Arterial Carboplatin Causes Hearing Loss in Children

Hearing Loss | Intraocular Retinoblastoma

United States
Rehabilitation Centre Zivot

Completed

Physiotherapy Approach for Susac Syndrome

Susac Syndrome

Bosnia and Herzegovina
Takeda

Active, not recruiting

A Study of Adynovate in Previously Treated Chinese Teenagers and Adults With Severe Hemophilia A

Hemophilia A

China
Takeda

Active, not recruiting

A Study of TAK-662 for Japanese Patients With Congenital Protein C Deficiency

Congenital Protein C Deficiency

Japan
National Eye Institute (NEI)

Active, not recruiting

Repository for Inherited Eye Diseases

Retinitis Pigmentosa | Inherited Ophthalmic Diseases

United States
Hoffmann-La Roche

Active, not recruiting

Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants (HAVEN 5)

Hemophilia A

China, Thailand, Hong Kong, Malaysia
BioMarin Pharmaceutical

Active, not recruiting

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A (GENEr8-3)

Hemophilia A

United States, Australia, Taiwan, Brazil
Outlook Therapeutics, Inc.

Active, not recruiting

A 3-month Study to Compare the Safety of ONS-5010 in Vials Versus Pre-filled Syringe in Subjects With Visual Impairment Due to Retinal Disorders (NORSE SEVEN)

Age-Related Macular Degeneration | Diabetic Macular Edema | Wet Macular Degeneration | Neovascular Age-related Macular Degeneration | BRVO - Branch Retinal Vein Occlusion

United States
Yuzuncu Yıl University

Completed

Physiotherapy and Reaction Time in Hemophilia

Hemophilia Arthropathy

Turkey
Novo Nordisk A/S

Completed

Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B. (paradigm™5)

Congenital Bleeding Disorder | Haemophilia B

United States, Canada, United Kingdom, France, Malaysia, Brazil, Croatia, Germany, Italy, Japan, Taiwan

Search clinical trials for: Inherited Retinal Disorders

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