Search clinical trials for: Leber congenital amaurosis

Total 23839 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
Ocugen

Recruiting

Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis (OCU400)

Retinitis Pigmentosa | Leber Congenital Amaurosis

United States
ProQR Therapeutics

Recruiting

An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. (BRIGHTEN)

Eye Diseases | Neurologic Manifestations | Retinal Degeneration | Retinal Dystrophies | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

Belgium, Brazil, Canada, Germany, Italy, Netherlands, United Kingdom
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
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
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
Spark Therapeutics

Active, not recruiting

Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)

Inherited Retinal Dystrophy Due to RPE65 Mutations
Atsena Therapeutics Inc.

Active, not recruiting

Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

Leber Congenital Amaurosis | LCA | LCA1

United States
Spark Therapeutics

Active, not recruiting

A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy

United States
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
Spark Therapeutics

Active, not recruiting

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

Leber Congenital Amaurosis

United States
MeiraGTx UK II Ltd

Completed

Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65

Leber Congenital Amaurosis

United Kingdom, United States
University of Pennsylvania
National Eye Institute (NEI)

Active, not recruiting

Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations (LCA)

Retinal Diseases | Amaurosis of Leber

United States
ProQR Therapeutics

Terminated

Extension Study to Study PQ-110-001 (NCT03140969) (INSIGHT)

Eye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

United States, Belgium
ProQR Therapeutics

Completed

Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Leber's Congenital Amaurosis

United States, Belgium
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
Editas Medicine, Inc.

Completed

Natural History Study of CEP290-Related Retinal Degeneration

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

United States, Netherlands, France, Germany
ProQR Therapeutics

Active, not recruiting

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) (ILLUMINATE)

Eye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

United States, Belgium, Brazil, Canada, France, Germany, Italy, Netherlands, United Kingdom
MeiraGTx UK II Ltd

Completed

Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) (OPTIRPE65)

Leber Congenital Amaurosis

United Kingdom, United States
Spark Therapeutics

Completed

Safety Study in Subjects With Leber Congenital Amaurosis

Leber Congenital Amaurosis

United States
Eyecure Therapeutics Inc.
Beijing Tongren Hospital

Unknown

Treatment of RP and LCA by Primary RPE Transplantation

Leber Congenital Amaurosis, Retinitis Pigmentosa

China
Hadassah Medical Organization

Completed

Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

Leber Congenital Amaurosis

Israel
Applied Genetic Technologies Corp
Oregon Health and Science University; University of Massachusetts, Worcester

Completed

Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis

Leber Congenital Amaurosis

United States
Assistance Publique - Hôpitaux de Paris

Completed

Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families. (GENPHENACL)

Leber Congenital Amaurosis

France
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
University College, London
Moorfields Eye Hospital NHS Foundation Trust; Targeted Genetics Corporation

Completed

Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

Retinal Degeneration

United Kingdom
Nantes University Hospital

Completed

Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 (RPE65)

Leber Congenital Amaurosis

France
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
Nantes University Hospital

Completed

Genetic Study of Patients Suffering From Congenital Amaurosis of Leber or From an Early Severe Retinal Dystrophy

Retinal Diseases | Amaurosis

France
Children's Mercy Hospital Kansas City
Indiana Clinical and Translational Sciences Institute

Recruiting

IMProving DRug Dosing and Outcomes for Single VEntricle Patients With Fontan Associated Liver Disease (IMPROVE-FALD)

Fontan Circulation

United States
Mirum Pharmaceuticals, Inc.

Recruiting

Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP) (LEAP)

Alagille Syndrome

United States
McGill University Health Centre/Research Institute...
Canadian Institutes of Health Research (CIHR)

Recruiting

Longitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum Disorder (ZSDvision)

Zellweger Spectrum Disorder

Canada
Lifetech Scientific (Shenzhen) Co., Ltd.

Not yet recruiting

Cera™ Vascular Plug System Post-Market Clinical Follow-Up

Aneurysm | Portal Hypertension | Arteriovenous Fistula | Endoleak | Pulmonary Arteriovenous Malformation | Splenic Laceration
National Human Genome Research Institute (NHGRI)

Recruiting

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

Metabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditions

United States
King's College London

Recruiting

The Living With a Long-Term Condition Study (LTC)

Liver Diseases | Heart Diseases | Heart Failure | Stroke | Cerebrovascular Disorders | Epilepsy | Pain | Multiple Sclerosis | Hypertension | Obesity | Diabetes | Cancer | Arthritis | Osteoarthritis | Rheumatoid Arthritis | Parkinson Disease | Migraine | Hepatitis | Atrial Fibrillation | Asthma | Endometriosis | Dementia | Fibromyalgia | Celiac... and other conditions

United Kingdom
Shanghai General Hospital, Shanghai Jiao Tong University...

Not yet recruiting

An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA

To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)
National Taiwan University Hospital
Insight Medical Solutions

Recruiting

Wired Magnetically Assisted Capsule Endoscopy and Esophageal Varices

Esophageal Varices

Taiwan
Tao Zhang

Recruiting

Analgesic Effects of Caudal S-ketamine for Supplementation of Ropivacaine Caudal Analgesia in Children With Hypospadias

Hypospadias | Caudal Block | Pediatrics | Postoperative Analgesia | S-ketamine

China
Weibing Tang

Recruiting

Early Predictors for the Short Term Native Liver Survival in Patients With Biliary Atresia After Kasai Procedure

Biliary Atresia

China
Tianjin First Central Hospital

Not yet recruiting

Effect of Desflurane on Pediatric Acute Respiratory Distress Syndrome After Living Donor Liver Transplant Recipients (EDPARDSLDLTR)

Desflurane | Biliary Atresia | Pediatric Acute Respiratory Distress Syndrome

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
Sanguine Biosciences

Recruiting

Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

Heart Diseases | Stroke | Lymphoma | Multiple Sclerosis | Kidney Diseases | Diabetes | Leukemia | Age-Related Macular Degeneration | Cancer | Arthritis | Schizophrenia | Thyroid Diseases | Parkinson Disease | Hepatitis | Vasculitis | Psoriasis | Asthma | Amyloidosis | Fibromyalgia | Graves Disease | Alzheimer Disease | Celiac Disease | Cirr... and other conditions

United States
Radboud University Medical Center

Recruiting

Assessment of Treatment With Laparoscopic Fenestration or Aspiration Sclerotherapy for Large Symptomatic Hepatic Cysts (ATLAS)

Autosomal Dominant Polycystic Kidney | Polycystic Liver Disease | Liver Cyst | Autosomal Dominant Polycystic Liver Disease

Netherlands
Children's Hospital of Fudan University

Recruiting

Atorvastatin Therapy on Xanthoma in Alagille Syndrome

Alagille Syndrome | Atorvastatin | Xanthoma

China
University Medical Center Groningen
Radboud University Medical Center

Recruiting

A GnRH Agonist IN Pre-menopausal Women STudy to Treat Severe Polycystic Liver Disease (AGAINST-PLD)

Autosomal Dominant Polycystic Kidney | Polycystic Liver Disease

Netherlands

Search clinical trials for: Leber congenital amaurosis

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