Clinical trials: Hereditary Eye Disease
Leber's Hereditary Optic Neuropathy (LHON)
Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)
The purpose of this survey is to collect visual acuity data from patients with LHON in order to establish the clinical course (natural history) and visual acuity outcomes in patients with a genetically confirmed diagnosis of…
- Trial name
- NCT02796274
- Where
- 21 sites in Belgium, France and 5 more
- Volunteers
- 219 people
- Sponsor
- Santhera Pharmaceuticals
Bronchopulmonary Dysplasia, Chronic Lung Disease of Prematurity
A Clinical Efficacy and Safety Study of OHB-607 in Preventing Bronchopulmonary Dysplasia in Extremely Premature Infants
The purpose of this study is to determine if an investigational drug can prevent Bronchopulmonary Dysplasia, reducing the burden of chronic lung disease in extremely premature infants, as compared to extremely premature infants…
- Conditions
- Bronchopulmonary Dysplasia, Chronic Lung Disease of Prematurity, Intraventricular Hemorrhage, Retinopathy of Prematurity (ROP)
- Trial name
- NCT03253263
- Testing
- OHB-607
- Where
- 62 sites in United States, Australia and 11 more
- Volunteers
- 295 people
- Sponsor
- OHB Neonatology Ltd.
Leber Congenital Amaurosis 10, Blindness
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A>G…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders
- Trial name
- HYPERION (NCT06891443)
- Testing
- sepofarsen
- Where
- 18 sites in United States, Australia and 8 more
- Volunteers
- 32 people
- Sponsor
- Laboratoires Thea
Neurologic Disorders, Nervous System Diseases
Neurologic Stem Cell Treatment Study
This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3 of the nasal passages in order to determine if such a treatment will…
- Conditions
- Neurologic Disorders, Nervous System Diseases, Neurodegenerative Diseases, Neurological Disorders
- Trial name
- NEST (NCT02795052)
- Testing
- Intravenous and Intranasal BMSC
- Where
- 3 sites in United States, United Arab Emirates
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
Leber Hereditary Optic Neuropathy (LHON)
Leber Hereditary Optic Neuropathy (LHON) Historical Case Record Survey
The objective of this survey is to establish the clinical course of vision loss and recovery in patients with a genetically confirmed diagnosis of Leber Hereditary Optic Neuropathy (LHON).
- Trial name
- NCT01892943
- Where
- 8 sites in Belgium, Denmark and 3 more
- Volunteers
- 306 people
- Sponsor
- Santhera Pharmaceuticals
Retinitis Pigmentosa
Clinical Trial of Intravitreal Injection of Autologous Bone Marrow Stem Cells in Patients With Retinitis Pigmentosa
The purpose of this study is to evaluate the safety of intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa by a prospective, single-center, randomized, parallel, double-blind, phase I…
- Trial name
- TC/RP (NCT02280135)
- Testing
- Intravitreal injection of Autologous bone marrow Stem Cell, Subconjunctival injection of saline
- Where
- Spain
- Volunteers
- 8 people
- Sponsor
- Red de Terapia Celular
Retinopathy of Prematurity (ROP)
Aflibercept for Retinopathy of Prematurity - Intravitreal Injection Versus Laser Therapy
The purpose of this study is to demonstrate how well aflibercept works in babies with ROP, comparing it with laser therapy. The study also has the objective to demonstrate how safe aflibercept is when used in babies, and describe…
- Trial name
- FIREFLEYE (NCT04004208)
- Testing
- Eylea (Aflibercept, BAY86-5321), Laser photocoagulation
- Where
- 90 sites in Argentina, Austria and 25 more
- Volunteers
- 113 people
- Sponsor
- Bayer
Retinal Dystrophies, Color Vision Defects
Natural History Study of Inherited Retinal Diseases
This prospective, observational investigation seeks to delineate the interplay between chromatic vision deficits and both functional visual outcomes and anatomical retinal biomarkers in individuals affected by Inherited Retinal…
- Conditions
- Retinal Dystrophies, Color Vision Defects, Vision Disorders, Macular Degeneration
- Trial name
- MojiLVCDT (NCT07085533)
- Where
- China
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Augelux Therapeutics
Renal Fanconi Syndrome, Photophobia
Biomarker for Cystinosis Disease: BioCystinosis (BioCystinosis)
Development of a new mass spectrometry-based biomarker for the early and sensitive diagnosis of Cystinosis disease from the blood
- Conditions
- Renal Fanconi Syndrome, Photophobia, Hypophosphatemia
- Trial name
- BioCystinosis (NCT02837523)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Retinitis Pigmentosa (RP)
Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
The purpose of this study is: To evaluate whether 7-day treatment with oral QLT091001 can improve visual function in RP subjects with an autosomal dominant mutation in RPE65.
- Trial name
- NCT01543906
- Testing
- QLT091001
- Where
- 2 sites in Canada, Ireland
- Volunteers
- 5 people
- Sponsor
- QLT Inc.
Retinitis Pigmentosa, Retinitis
Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study
This study will be conducted following Good Clinical Practice (GCP) and International Conference on Harmonization (ICH) guidelines.
- Conditions
- Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases
- Trial name
- REMAIN (NCT06162585)
- Testing
- Gene Therapy product-MCO-010
- Where
- 6 sites in United States, Puerto Rico
- Volunteers
- 18 people
- Sponsor
- Nanoscope Therapeutics Inc.
ROSAH
Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients
The purpose of this study is to evaluate the safety and tolerability of DF-003 in retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and migraine headache (ROSAH) syndrome patients.
- Trial name
- NCT06395285
- Testing
- DF-003
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 12 people
- Sponsor
- Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)
Retinitis Pigmentosa
A Prospective, Observational Study in Adults With Retinitis Pigmentosa (RP)
The natural history in individuals with severe retinitis pigmentosa (RP) is variable and there remains an unmet need to better understand disease progression in this population.
- Trial name
- NCT06517940
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Sumitomo Pharma America, Inc.
Glaucoma, Age Related Macular Degeneration
Assessment of Visual Function With a Portable Brain-computer Interface
1. The purpose of this study is to evaluate the nGoggle's accuracy and repeatability in detecting visual function loss. In addition, the ability to stage glaucomatous damage and investigate the relationship between nGoggle…
- Conditions
- Glaucoma, Age Related Macular Degeneration, Optic Neuropathy, Retinal Degeneration
- Trial name
- NCT03760055
- Testing
- nGoggle
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- NGoggle
Thyroid Eye Disease (TED), Graves' Disease
Evaluation of ETHY-001 for Safety, Tolerability, and Clinical Activity in Thyroid Eye Disease
The primary objective of study is to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ETHY-001 in healthy volunteers and participants with Thyroid Eye Disease after single subcutaneous doses.
- Conditions
- Thyroid Eye Disease (TED), Graves' Disease, Eye Diseases, Hereditary, Eye Diseases
- Trial name
- NCT07885605
- Testing
- ETHY-001
- Where
- 3 sites in United States
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Ethyreal Bio, Inc.
ARB, BVMD
Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)
The goal of this clinical trial is to learn if drug OPGx-BEST1 works to treat BVMD and ARB Bestrophinopathy. It will also learn about the safety of drug OPGx-BEST1.
- Conditions
- ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy
- Trial name
- BIRD-1 (NCT07185256)
- Testing
- OPGx-BEST1
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Opus Genetics, Inc
Macular Degeneration, Choroidal Neovascularization
Safety Study for Treatment of Wet Macular Degeneration Using the TheraSight(TM) Ocular Brachytherapy System
The study will investigate the safety and feasibility of using the TheraSight(TM) Brachytherapy System for treatment of wet age-related macular degeneration (AMD).
- Trial name
- NCT00100087
- Testing
- Ocular Brachytherapy
- Where
- 6 sites in United States
- Volunteers
- 30 people
- Sponsor
- Theragenics Corporation
Behcet Syndrome
A Study to Evaluate the Efficacy and Safety of Apremilast (CC-10004) in the Treatment of Behçet Disease
The purpose of this study is to assess whether Apremilast is safe and effective in the treatment of patients with Behcet Disease.
- Trial name
- NCT00866359
- Testing
- Apremilast (CC-10004)
- Where
- 7 sites in United States, Turkey (Türkiye)
- Volunteers
- 111 people
- Sponsor
- Amgen
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
The objective of this study is to collect long-term safety information (i.e., for 5 years after treatment) associated with voretigene neparvovec-rzyl (vector and/or transgene), its subretinal injection procedure, the concomitant…
- Trial name
- NCT03597399
- Testing
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Where
- 10 sites in United States
- Volunteers
- 87 people
- Sponsor
- Spark Therapeutics, Inc.
Fuchs Endothelial Corneal Dystrophy
A Study to Evaluate the Safety and Efficacy of K-321 Eye Drops After Descemetorhexis in Participants With Fuchs Endothelial Corneal Dystrophy (FECD)
A study to assess the safety and efficacy of K-321 in participants with FECD after descemetorhexis.
- Trial name
- NCT05795699
- Testing
- Ripasudil
- Where
- 50 sites in United States, Canada and 4 more
- Volunteers
- 107 people
- Sponsor
- Kowa Research Institute, Inc.