Search clinical trials for: Inherited Retinal Disorders

Total 147852 results

Xinhua Hospital, Shanghai Jiao Tong University...
HuidaGene Therapeutics Co., Ltd.

Recruiting

Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)

Leber Congenital Amaurosis

China
HuidaGene Therapeutics Co., Ltd.
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Recruiting

Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial (LIGHT)

Leber Congenital Amaurosis

China
HuidaGene Therapeutics Co., Ltd.
Cholgene Therapeutics, Inc.

Recruiting

Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR) (STAR)

Leber Congenital Amaurosis | Inherited Retinal Diseases Caused by RPE65 Mutations

United States, China
Centre Hospitalier Universitaire de Liege
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators

Recruiting

Baby Detect : Genomic Newborn Screening

Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions

Belgium
The Hospital for Sick Children

Recruiting

Adaptive Optics Retinal Imaging in Inherited and Acquired Retinal Disorders

Genetic Disease | Inherited Disease

Canada
MejoraVisionMD
Maisonneuve-Rosemont Hospital; Retina and Genomics Institute

Recruiting

Phenotype Correlates Genotype of Inherited Retina Dystrophies, Retinitis Pigmentosa, Con>Rod Dystrophies.

Retinitis Pigmentosa | Usher Syndromes | Cone Dystrophy | Retina; Dystrophy

Mexico
Foundation Fighting Blindness

Recruiting

Inherited Retinal Degenerative Disease Registry (MRTR)

Retinitis Pigmentosa | Choroideremia | Usher Syndrome | Batten Disease | Leber Congenital Amaurosis | Goldmann-Favre Syndrome | Kearns-Sayre Syndrome | Retinal Disease | Bardet-Biedl Syndrome | Stargardt Disease | Cone Dystrophy | Retinoschisis | Achromatopsia | Gyrate Atrophy | Eye Diseases Hereditary | Bassen-Kornzweig... and other conditions

United States
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
Spark Therapeutics, Inc.
Children's Hospital of Philadelphia; University of Iowa

Active, not recruiting

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Leber Congenital Amaurosis | Inherited Retinal Dystrophy Due to RPE65 Mutations

United States
RTI International
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

Early Check: Expanded Screening in Newborns

Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditions

United States
GenSight Biologics

No longer available

EAP_GS010_single Patient

Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
University of Campania "Luigi Vanvitelli"
Retina Italia Onlus

Completed

Natural History of Patients With Inherited Retinal Diseases Due to Mutations in RPE65 Gene (RPE65-NHS)

Leber Congenital Amaurosis 2 | Retinitis Pigmentosa 20

Italy
Daniel Benjamin
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Completed

Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care (PTN_POPS)

Heart Failure | Epilepsy | Pain | Pneumonia | Sepsis | Inflammation | Hypertension | Chronic Kidney Diseases | Hemophilia | Schizophrenia | Central Nervous System Infections | Nosocomial Pneumonia | Insomnia | Anxiety | Bipolar Disorder | Neutropenia | Staphylococcal Infections | Bradycardia | Seizures | Urinary Tract Infections | Pulmonary... and other conditions

United States, Canada, United Kingdom, Israel, Singapore
ProQR Therapeutics

Terminated

Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, United Kingdom
Editas Medicine, Inc.

Active, not recruiting

Single Ascending Dose Study in Participants With LCA10

Retinal Degeneration | Eye Diseases, Hereditary | Vision Disorders | Leber Congenital Amaurosis 10 | Inherited Retinal Dystrophies | Retinal Disease | Eye Disorders Congenital

United States
ProQR Therapeutics

Active, not recruiting

Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, Netherlands, United Kingdom, Germany
GenSight Biologics

Active, not recruiting

Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year (REFLECT)

Leber Hereditary Optic Neuropathy

Belgium, Spain, United States, France, United Kingdom, Taiwan, Italy
Applied Genetic Technologies Corp
National Eye Institute (NEI)

Active, not recruiting

Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)

Achromatopsia

United States
SparingVision

Active, not recruiting

Retrospective Natural History Study of Retinitis Pigmentosa (PHENOROD1)

Retinitis Pigmentosa (RP)

France
GenSight Biologics

Completed

REALITY LHON Registry (REALITY)

Leber Hereditary Optic Neuropathy

United States, France, Italy, Spain, United Kingdom
University of Oxford

Completed

Visual and Functional Assessment in Low Vision Patients

Age-related Macular Degeneration | Retinitis Pigmentosa | Inherited Retinal Dystrophies | Optic Nerve Pathology

United Kingdom
Applied Genetic Technologies Corp
National Eye Institute (NEI)

Completed

Clinical and Genetic Characterization of Individuals With Achromatopsia

Achromatopsia

United States
National Eye Institute (NEI)

Completed

Potential Research Participants for Future Studies of Inherited Eye Diseases

Glaucoma | Macular Degeneration | Retinitis Pigmentosa | Cataract | Strabismus

United States
QLT Inc.

Completed

Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT

Leber Congenital Amaurosis (LCA) | Retinitis Pigmentosa (RP)

United States, Canada, Denmark, Germany, Netherlands, Switzerland, United Kingdom
QLT Inc.

Completed

Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)

LCA (Leber Congenital Amaurosis) | RP (Retinitis Pigmentosa)

Canada, United States, Germany, Netherlands, United Kingdom
QLT Inc.

Completed

Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations

LCA (Leber Congenital Amaurosis) | RP (Retinitis Pigmentosa)

United States, Canada, Germany, Netherlands, United Kingdom
NeuroVision Imaging

Recruiting

Effect of LEQEMBI on Cerebral and Retinal Amyloid in Mild Cognitive Impairment Due to Alzheimer's Disease

Alzheimer Disease | Mild Cognitive Impairment

United States
Regeneron Pharmaceuticals

Not yet recruiting

An Observational Study to Investigate the Use of Aflibercept 8 mg to Treat Adult Patients With Neovascular Age-Related Macular Degeneration (nAMD) and Diabetic Macular Edema (DME) in a Real-World Setting (SPECTRUM-US)

Visual Impairment | Diabetic Macular Edema (DME) | Neovascular Age-related Macular Degeneration (nAMD) | Wet Age-related Macular Degeneration (AMD)
CSL Behring

Not yet recruiting

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy

Hemophilia B
Washington University School of Medicine
Alzheimer's Association; Eisai Inc.

Not yet recruiting

DIAN-TU Amyloid Removal Trial (ART) in Dominantly Inherited Alzheimer's Disease (DIAN-TU)

Dementia | Alzheimer's Disease | Alzheimer's Disease, Familial

United States, Australia, United Kingdom
University of Wisconsin, Madison

Not yet recruiting

COmparison of Clarus and Optos Ultrawide Field Imaging Systems for Inherited Retinal Disease (COCO-IRD)

Retinal Disease

United States
Regeneron Pharmaceuticals
Intellia Therapeutics

Not yet recruiting

A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Pediatric, Adolescent and Adult Patients With Hemophilia B (BEYOND-9)

Hemophilia B
Maastricht University Medical Center
Diabetes Fonds

Recruiting

The Effect of Pyridoxamine Supplementation on Microvascular Function in Type 2 Diabetes (PYRAMID)

Type 2 Diabetes | Neuropathy, Diabetic | Nephropathy, Diabetic | Microvascular Function | Retinopathy, Diabetic

Netherlands
Enzyre B.V.
Indiana Hemophilia & Thrombosis Center

Not yet recruiting

Case and Use Scenario Study to Gain Knowledge on the User Needs for a Medical Device for Hemophilia A Monitoring

Hemophilia A

United States
University Hospital, Bonn
Swedish Orphan Biovitrum

Recruiting

Prevalence of Synovitis in Patients With Haemophilia A (SynoPrev)

Haemophilia A | Synovitis | Hemophilia Arthropathy | Sonography

Germany
Equilibra Bioscience LLC

Recruiting

A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B)

Hemophilia A | Hemophilia B | Healthy Participants

United States
Centre Hospitalier Universitaire Dijon

Not yet recruiting

Study of Vitreoretinal Molecular Changes During Rhegmatogenous Retinal Detachment (LIPIDRET)

Rhegmatogenous Retinal Detachments

France
Washington University School of Medicine
National Cancer Institute (NCI); The Foundation for Barnes-Jewish Hospital

Recruiting

Washington University Participant Engagement and Cancer Genomic Sequencing Center (WU-PE-CGS)

Multiple Myeloma | Rectal Cancer | Cholangiocarcinoma | Colon Cancer

United States
IRCCS Multimedica
AbbVie

Recruiting

OCT and OCT-Angiography Biomarkers of Treatment Response to Dexamethasone Implant in Macular Edema Due to Retinal Vascular Diseases - DME and RVO

Diabetic Macular Edema | Retinal Vein Occlusion | Macular Edema Due to Diabetes Mellitus

Italy
Dr. Sascha Drewlo

Not yet recruiting

Optimization of Cervical Collections in Pregnancy (OCCP)

Fetal Growth Retardation | Preeclampsia | Cervix; Pregnancy | Inherited Genetic Conditions (Diagnosis)
University of Rochester

Not yet recruiting

The Effects of Disulfiram (Antabuse®) on Visual Acuity in Patients With Retinal Degeneration

Age-Related Macular Degeneration | Retinitis Pigmentosa | Retinal Dystrophies | Alcohol Use Disorder | Stargardt Disease

United States
Kathelijn Fischer
Radboud University Medical Center; University Medical Center Groningen; Maastricht... and other collaborators

Recruiting

Pharmacokinetic-guided Dosing of Emicizumab (DosEmi)

Adolescent | Child | Hemophilia A With Inhibitor | Adult | Hemophilia A Without Inhibitor | Hemophilia A, Severe

Netherlands
Suzhou Alphamab Co., Ltd.

Recruiting

Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors

Hemophilia A With Inhibitor | Hemophilia B With Inhibitor

China
Chia Tai Tianqing Pharmaceutical Group Nanjing...

Not yet recruiting

A Clinical Study of Recombinant Human Coagulation Factor VIII for Injection in Patients With Severe Hemophilia A

Hemophilia A

China
Spark Therapeutics, Inc.

Recruiting

Study of a Gene Therapy Treatment for Hemophilia A (KEYSTONE 1)

Hemophilia A

United States
Hopital Foch

Not yet recruiting

Ursodeoxycholic Acid (UDCA) as a Neuroprotective Adjuvant Treatment to Rhegmatogenous Retinal Detachment Surgery (UDCA)

Retinal Detachment

France
Central Hospital, Nancy, France

Not yet recruiting

Clinical Characteristics and Surgical Outcomes of the Fellow Eye in Bilateral Retinal Detachments

Retinal Detachment

France
Staidson (Beijing) Biopharmaceuticals Co., Ltd

Recruiting

Safety and Efficacy of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor

Hemophilia

China
Investigación en Hemofilia y Fisioterapia

Not yet recruiting

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

Hemophilia

Spain
Radboud University Medical Center
Enzyre B.V.

Not yet recruiting

Version Testing of EnzySystem Version A for Hemophilia A

Hemophilia A

Netherlands

Search clinical trials for: Inherited Retinal Disorders

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