Clinical Trials on Optic, Atrophy, Hereditary, Leber in United Kingdom

Total 109 results

GenSight Biologics

Completed

RESCUE and REVERSE Long-term Follow-up (RESTORE)

Leber Hereditary Optic Neuropathy

United States, France, Italy, United Kingdom, Germany
GenSight Biologics

Completed

REALITY LHON Registry (REALITY)

Leber Hereditary Optic Neuropathy

United States, France, Italy, Spain, United Kingdom
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
Neurophth Therapeutics Inc

Recruiting

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations

Leber Hereditary Optic Neuropathy (LHON)

China, United States
Neurophth Therapeutics Inc

Active, not recruiting

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations

Leber Hereditary Optic Neuropathy (LHON)

United States
Santhera Pharmaceuticals

Completed

RHODOS Follow-up Single-visit Study (RHODOS-OFU)

Leber's Hereditary Optic Neuropathy

Canada, Germany, United Kingdom
Santhera Pharmaceuticals

Completed

Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)

Leber's Hereditary Optic Neuropathy (LHON)

Belgium, Netherlands, France, Germany, Norway, United Kingdom, Italy
Santhera Pharmaceuticals

Completed

Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy (RHODOS)

Leber's Hereditary Optic Neuropathy

Canada, Germany, United Kingdom
Santhera Pharmaceuticals

Completed

Study to Assess the Efficacy and Safety of Raxone in LHON Patients (LEROS)

Leber's Hereditary Optic Neuropathy (LHON)

United States, United Kingdom, Spain, Poland, Austria, Belgium, Bulgaria, Germany, Italy, Portugal
Medical College of Wisconsin

Terminated

Near-infrared Light-emitting Diode (NIR-LED) Therapy for Leber's Hereditary Optic Neuropathy (LHON) (LHON)

Leber's Hereditary Optic Neuropathy (LHON)

United States
Byron Lam
National Eye Institute (NEI)

Active, not recruiting

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON)

Leber's Hereditary Optic Neuropathy

United States
Stealth BioTherapeutics Inc.

Completed

A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy

Leber's Hereditary Optic Neuropathy

United States
Edison Pharmaceuticals Inc

No longer available

Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

Leber's Hereditary Optic Neuropathy

United States
GenSight Biologics

Completed

Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE) (REVERSE)

Optic, Atrophy, Hereditary, Leber

United Kingdom, United States, Germany, Italy, France
GenSight Biologics

Completed

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation (RESCUE)

Optic, Atrophy, Hereditary, Leber

United Kingdom, United States, France, Germany, Italy
Randy Kardon

Recruiting

New Non-invasive Modalities for Assessing Retinal Structure and Function

Leber Hereditary Optic Neuropathy | Ischemic Optic Neuropathy | Hemianopia | Branch Retinal Artery Occlusion | Acute Zonal Occult Outer Retinopathy

United States
MD Stem Cells

Recruiting

Stem Cell Ophthalmology Treatment Study II (SCOTS2)

Glaucoma | Age-Related Macular Degeneration | Macular Degeneration | Retinitis Pigmentosa | Leber Hereditary Optic Neuropathy | Optic Atrophy | Blindness | Optic Neuropathy | Vision, Low | Retinal Disease | Stargardt Disease | Maculopathy | Retinopathy | Optic Nerve Disease | Nonarteritic Ischemic Optic Neuropathy | Vision... and other conditions

United States, United Arab Emirates
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
State University of New York at Buffalo
Hadley Jo Foundation

Completed

Natural History Study of SLC25A46 Mutation-related Mitochondriopathy

Mitochondrial Diseases | Optic Atrophy | Neurodegenerative Disease, Hereditary

United States
State University of New York at Buffalo
The Callum McKeefery and Nikki Albano McKeefery Pediatric Division of Genetics...

Completed

Natural History Study of FDXR Mutation-related Mitochondriopathy

Mitochondrial Diseases | Optic Atrophy | Neurodegenerative Disease, Hereditary

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
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
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
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
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
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
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
Quark Pharmaceuticals

Completed

Safety Study of a Single IVT Injection of QPI-1007 in Chronic Optic Nerve Atrophy and Recent Onset NAION Patients

Optic Atrophy | Non-arteritic Anterior Ischemic Optic Neuropathy

United States, Israel
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
Washington University School of Medicine
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Recruiting

Wolfram Syndrome and WFS1-related Disorders International Registry and Clinical Study

Diabetes Mellitus | Deafness | Ataxia | Diabetes Insipidus | Wolfram Syndrome | Optic Nerve Atrophy

United States
University College London Hospitals

Completed

Muscle Structure, Function and Gait in dHMN (GAIT-dHMN)

Distal Hereditary Motor Neuropathy, Type II | Distal Hereditary Motor Neuropathy, Type V | Distal Hereditary Motor Neuronopathy Type I | Distal Hereditary Motor Neuronopathy Type VI

United Kingdom
University of Birmingham

Active, not recruiting

Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome

Wolfram Syndrome

France, United Kingdom, Poland, Spain
Washington University School of Medicine
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National...

Completed

A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome

Diabetes Mellitus | Ataxia | Wolfram Syndrome | Optic Nerve Atrophy

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
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
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
Nova Southeastern University

Recruiting

A More Engaging Visual Field Test to Increase Use and Reliability in Pediatrics

Glaucoma, Suspect | Optic Nerve Hypoplasia and Abnormalities of the Central Nervous System | Congenital Coloboma of the Optic Nerve | Optic Nerve Head Pits, Bilateral Congenital

United States
Great Ormond Street Hospital for Children NHS Foundation...
AstraZeneca

Completed

Intermittent Dosing Of Selumetinib In Childhood NF1 Associated Tumours (INSPECT)

Neurofibromatosis Type 1 | Optic Nerve Glioma | Plexiform Neurofibroma

United Kingdom
Jaeb Center for Health Research
Department of Health and Human Services; Food and Drug Administration (FDA); Conquering...

Recruiting

Gyrate Atrophy Ocular and Systemic Study (GYROS)

Gyrate Atrophy | Gyrata of Choroid and Retina; Atrophy | Ornithine-δ-aminotransferase | OAT | Chorioretinal Degeneration

United States, Canada, Finland, France, United Kingdom, Germany, Brazil
NGoggle
National Eye Institute (NEI); Duke University

Enrolling by invitation

Assessment of Visual Function With a Portable Brain-computer Interface

Glaucoma | Retinal Degeneration | Age Related Macular Degeneration | Optic Neuropathy

United States
Yale University
Children's Hospital Los Angeles

Completed

Biological Clock Dysfunction in Optic Nerve Hypoplasia

Biological Clock Dysfunction | Optic Nerve Hypoplasia

United States
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
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
Gyroscope Therapeutics Limited
Novartis Pharmaceuticals

Active, not recruiting

FOCUS: A Phase I/II First in Human Study to Evaluate the Safety and Efficacy of GT005 Administered in Subjects With Dry AMD

Eye Diseases | Macular Degeneration | Retinal Degeneration | Geographic Atrophy | Dry Age-related Macular Degeneration | Retinal Disease | Macular Atrophy

United States, United Kingdom
Spark Therapeutics

Completed

Safety Study in Subjects With Leber Congenital Amaurosis

Leber Congenital Amaurosis

United States
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
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
Washington University School of Medicine
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Completed

Tracking Neurodegeneration in Early Wolfram Syndrome (TRACK)

Diabetes Mellitus | Type 1 Diabetes | Diabetes Insipidus | Wolfram Syndrome

United States
Complement Therapeutics

Recruiting

Investigating Geographic Atrophy Insights (i-GAIN) Natural History Study (i-GAIN)

Eye Diseases | Macular Degeneration | Retinal Degeneration | Geographic Atrophy | Dry Age-related Macular Degeneration | Retinal Disease | Macular Atrophy

United Kingdom, United States

Clinical Trials on Optic, Atrophy, Hereditary, Leber in United Kingdom

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