Clinical trials: Inherited Retinal Dystrophies
Retinitis Pigmentosa, Stargardt Macular Dystrophy
Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD)
The Vision Research and Assessment Institute (VRAI) was established with the purpose of serving as a testing facility for efficacy endpoints for patients with Low Vision.
- Conditions
- Retinitis Pigmentosa, Stargardt Macular Dystrophy, Stargardt Disease, Geographic Atrophy From Age-related Macular Degeneration
- Trial name
- DEFINE-IRD (NCT07502664)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Ray Therapeutics, Inc.
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Retinitis Pigmentosa.
Safety Study in Retinal Transplantation for Retinitis Pigmentosa.
The long-term goal is to show that retinal transplantation can help to prevent blindness and to restore eyesight in patients with the inherited disease retinitis pigmentosa.
- Trial name
- NCT00345917
- Testing
- Retinal transplantation instrument, Fetal tissue.
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Radtke, Norman D., M.D.
Retinal Diseases, Stargardt's Disease
Microcurrent Stimulation to Treat Macular Degeneration
Microcurrent stimulation thereapy improves retinal efficiency and may restore and/or improve retinal function.
- Conditions
- Retinal Diseases, Stargardt's Disease, Retinitis Pigmentosa
- Trial name
- NCT01790958
- Where
- 4 sites in United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Retina Institute of Hawaii
Stargardt's Macular Dystrophy
A Follow up Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
The purpose of this study is to evaluate the long term safety and tolerability of hESC-RPE cellular therapy in patients with advanced SMD from 1 to 5 years following the surgical procedure to implant the hESC-RPE cells.
- Trial name
- NCT02941991
- Testing
- hESC-RPE
- Where
- 2 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Astellas Institute for Regenerative Medicine
Retinitis Pigmentosa, Visual Impairment
Environmental Localization Mapping and Guidance for Visual Prosthesis Users
This study is driven by the hypothesis that independent navigation by blind users of visual prosthetic devices can be greatly aided by use of an autonomous navigational aid that provides information about the environment and…
- Conditions
- Retinitis Pigmentosa, Visual Impairment, Visual Prosthesis
- Trial name
- SLAM (NCT04359108)
- Testing
- Navigation system mode: Argus Vision, Navigation system mode: Depth Vision, Navigation system mode: Depth Vision with Haptic / Audio
- Where
- 2 sites in United States
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Macular Degeneration
Effect of DHA Supplements on Macular Function in Patients With Stargardt Macular Dystrophy and Stargardt-like Macular Dystrophy
This study will evaluate whether docosahexaenoic acid (DHA) dietary supplementation can improve macular function in patients with Stargardt macular dystrophy and Stargardt-like macular dystrophy.
- Trial name
- NCT00060749
- Testing
- Docosahexaenoic Acid (DHA) Dietary Supplement
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- National Eye Institute (NEI)
Retinitis Pigmentosa, Usher Syndromes
Prospective Open Clinical and Genetic Study of Patients With Retinitis Pigmentosa
This study is aimed to characterize Russian population of Retinitis Pigmentosa
- Trial name
- RU-RP (NCT03901391)
- Testing
- Whole Exome Sequencing
- Where
- 2 sites in Russia
- Volunteers
- 130 people
- Sponsor
- Sensor Technology for Deafblind
Diabetic Retinopathy, Retinal Vein Occlusion
Computer Aided Diagnosis of Multiple Eye Fundus Diseases From Color Fundus Photograph
Blindness can be caused by many ocular diseases, such as diabetic retinopathy, retinal vein occlusion, age-related macular degeneration, pathologic myopia and glaucoma.
- Conditions
- Diabetic Retinopathy, Retinal Vein Occlusion, Retinal Artery Occlusion, Central Serous Chorioretinopathy
- Trial name
- NCT04723160
- Testing
- Software assisted imaging diagnosis
- Where
- 5 sites in China
- Volunteers
- 748 people, healthy volunteers welcome
- Sponsor
- Visionary Intelligence Ltd.
Inherited Retinal Dystrophy Due to RPE65 Mutations
Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
Multi-site, non-randomized, observational study, for up to 15 years after subretinal AAV2-hRPE65v2 administration for each subject.
- Trial name
- NCT03602820
- Testing
- AAV2-hRPE65v2
- Volunteers
- 41 people
- Sponsor
- Genentech, Inc.
Inherited Retinal Dystrophy Associated With RPE65 Mutations
Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
The purpose of the study is to evaluate the safety, tolerability and efficacy of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy.
- Trial name
- NCT06196827
- Testing
- LX101
- Where
- 2 sites in China
- Volunteers
- 9 people
- Sponsor
- Innostellar Biotherapeutics Co.,Ltd
Retinitis Pigmentosa, Cone Dystrophy
Phenotype Correlates Genotype of Inherited Retina Dystrophies, Retinitis Pigmentosa, Con>Rod Dystrophies.
Patients with retina dystrophies (retinitis pigmentosa, cone>rods dystrophies, Usher and syndromic) will be correlated with genotype and validate inheritance mode by segregation analysis.
- Conditions
- Retinitis Pigmentosa, Cone Dystrophy, Usher Syndromes, Retina; Dystrophy
- Trial name
- NCT03990727
- Testing
- Retina Analysis-mosaic, Autofluorescence, OCT- 1 micra
- Where
- Mexico
- Volunteers
- 17,000 people, healthy volunteers welcome
- Sponsor
- MejoraVisionMD
Macular Dystrophy, Corneal
Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular Dystrophy
The purpose of this study is to investigate whether taking 4-methylpyrazole (4-MP, fomepizole, Antizol™) inhibits dark adaptation of the eye.
- Trial name
- NCT00346853
- Testing
- 4-Methylpyrazole, saline
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of Utah
Cystoid Macular Edema, Fuchs Dystrophy
Descemet Membrane Endothelial Keratoplasty vs. Descemet's Stripping With Endothelial Keratoplasty vs. Descemet Stripping Only
This study aims to evaluate and compare incidence of post-operative cystoid macular edema (CME) after Descemet Membrane Endothelial Keratoplasty (DMEK), Descemet's Stripping Automated endothelial Keratoplasty (DSEK) and Descemet…
- Trial name
- NCT04527523
- Testing
- Optical Coherence Tomography
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Wake Forest University Health Sciences
Retinitis Pigmentosa (RP)
Subthreshold Micropulse Laser Therapy (SML) in Retinitis Pigmentosa
Introduction Retinitis pigmentosa (RP) is the most common form of genetic retinal degenerative disease. The disease is progressive and leads to blindness and permanent disability within a few years of diagnosis.
- Trial name
- NCT07088705
- Testing
- SML 577 nm, SML 810 nm
- Where
- Poland
- Volunteers
- 60 people
- Sponsor
- Marta P. Wiącek
Retinitis Pigmentosa
Clinical Trials of Continuous Oxygen Therapy Combined With Blue Light Deprivation in the Treatment of Retinitis Pigmentosa
The purpose of this study is to investigate the role of continuous oxygen therapy combined with blue light deprivation in prevention and control of retinitis pigmentosa, in order to find a new strategy of treatment for retinitis…
- Trial name
- NCT02465749
- Testing
- Continuous oxygen, blue light-absorbing sunglasses, Compound thrombosis capsule sig: 1.5g/tid
- Where
- China
- Volunteers
- 404 people
- Sponsor
- Sun Yat-sen University
Choroideremia
Gene Therapy for Blindness Caused by Choroideremia
Primary objective: To assess the safety and tolerability of the AAV.REP1 vector, administered at two different doses to the retina in 12 patients with a diagnosis of choroideremia.
- Trial name
- NCT01461213
- Testing
- rAAV2.REP1
- Where
- 4 sites in United Kingdom
- Volunteers
- 14 people
- Sponsor
- University of Oxford
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl).
- Trial name
- NCT00999609
- Testing
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Spark Therapeutics, Inc.
Retinitis Pigmentosa Syndrome
Evaluation of the Visual Field on the Ground
Retinopathy pigmentosa (which affects about 40,000 patients in France) leads to destruction of the peripheral retina. This condition, often familial, frequently affects young patients.
- Trial name
- ECVS (NCT03989622)
- Testing
- evaluation of the visual field on the ground
- Sponsor
- University Hospital, Limoges
Retinitis Pigmentosa
A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
The purpose of this first-in-human study is to explore the maximum tolerated dose (MTD) of CPK850 as determined by the single ascending dose ranging portion of the study.
- Trial name
- NCT03374657
- Testing
- CPK850
- Where
- Sweden
- Volunteers
- 12 people
- Sponsor
- Novartis Pharmaceuticals