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.
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, 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
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
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
Stargardt Disease, Stargardt Macular Dystrophy
A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions
Macular dystrophies are a group of inherited eye conditions that affect the macula. The macula is in the center of the retina, the light sensitive part at the back of the eye.
- Conditions
- Stargardt Disease, Stargardt Macular Dystrophy, Stargardt-like Macular Dystrophy
- Trial name
- DEVOTE (NCT07734064)
- Testing
- ASP2020
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Astellas Institute for Regenerative Medicine
Retinal Disease, Macular Degeneration
Stem Cell Ophthalmology Treatment Study
This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease. http://mdstemcells.com/scots-ii/
- Conditions
- Retinal Disease, Macular Degeneration, Hereditary Retinal Dystrophy, Optic Nerve Disease
- Trial name
- SCOTS (NCT01920867)
- Testing
- RB (Retrobulbar), ST (Subtenon), IV (Intravenous)
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- MD Stem Cells
Retinitis Pigmentosa, Leber Congenital Amaurosis
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 in patients with retinitis pigmentosa associated with NR2E3 and RHO mutations and in patients with LCA due to mutation(s) in CEP290 gene (OCU400-101).
- Trial name
- NCT05203939
- Testing
- OCU400 Low Dose, OCU400 Med Dose, OCU400 High Dose
- Where
- 7 sites in United States
- Volunteers
- 22 people
- Sponsor
- Ocugen
Usher Syndrome, Type 1B
Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa
The purpose of the 081-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-081 in USH1B patients with retinitis pigmentosa due to a mutation in the MYO7A gene.
- Trial name
- NCT06591793
- Testing
- AAVB-081
- Where
- 3 sites in Italy, United Kingdom
- Volunteers
- 15 people
- Sponsor
- AAVantgarde Bio Srl
Stargardt Disease
Study of ALK-001 on the Progression of Stargardt Disease
This study evaluates the efficacy and safety of investigational study drug ALK-001 in participants 8 to 45 years of age, inclusive, with symptoms and signs of autosomal recessive Stargardt disease (STGD)
- Trial name
- NORTHSTAR (NCT07419334)
- Testing
- ALK-001
- Where
- 14 sites in United States
- Volunteers
- 230 people
- Sponsor
- Alkeus Pharmaceuticals, Inc.
Retinitis Pigmentosa
A Study to Investigate the Safety of DSP-3077 After a Unilateral Eye Injection in Male and Female Participants 18 Years of Age or Older With Retinitis Pigmentosa
The Goal of this study is to evaluate the safety, tolerability, and clinical responses following single dose of DSP-3077. Study enrolls both male and female patients in 3 cohorts with each cohort defined by visual acuity (VA)…
- Trial name
- NCT06891885
- Testing
- DSP-3077 Retinal Sheet Cohort 1, DSP-3077 Retinal Sheet Cohort 2, DSP-3077 Retinal Sheet Cohort 3
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sumitomo Pharma America, Inc.
Inherited Retinal Dystrophy Associated With RPE65 Mutations
Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
The purpose of the study is to evaluate the efficacy and safety of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy.
- Trial name
- NCT07054632
- Testing
- LX101
- Where
- 4 sites in China
- Volunteers
- 30 people
- Sponsor
- Innostellar Biotherapeutics Co.,Ltd
Retinitis Pigmentosa
Argus® II Retinal Stimulation System Feasibility Protocol
Investigational Phase of the Study: The objective of this feasibility study is to evaluate the safety and utility of the Argus II Retinal Stimulation System in providing visual function to blind subjects with retinitis…
- Trial name
- NCT00407602
- Testing
- Implant of Argus II Retinal Prosthesis
- Where
- 12 sites in United States, France and 3 more
- Volunteers
- 30 people
- Sponsor
- Second Sight Medical Products
Retinitis Pigmentosa
Transcorneal Electrostimulation for Therapy of Retinitis Pigmentosa
The purpose of this study is to determine the long-term effects of transcorneal electrostimulation (TcES) in patients with retinitis pigmentosa (RP).
- Trial name
- NCT01837901
- Testing
- Transcorneal electrostimulation
- Where
- Germany
- Volunteers
- 88 people
- Sponsor
- Okuvision GmbH
Retinitis Pigmentosa
Long-term Safety of UC-MSC Transplantation in Patients With Retinitis Pigmentosa
The study will perform to follow-up UC-MSCs and CM transplantation. 18 patients will be called back to be examined after 5 years of UC-MSC and/or CM transplantation.
- Trial name
- NCT05786287
- Testing
- Conditioned Medium (CM), Umbilical Cord Mesenchymal Stem Cell (UC-MSC)
- Where
- 2 sites in Indonesia
- Volunteers
- 18 people
- Sponsor
- PT. Prodia Stem Cell Indonesia
Retinitis Pigmentosa
Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa.
- Trial name
- NCT06291935
- Testing
- VG901
- Where
- Germany
- Volunteers
- 6 people
- Sponsor
- VeonGen Therapeutics GmbH
Rod Cone Dystrophy
Natural History Study of Advanced RCD
ECSTASI is a Natural History Study, which will be monitoring how standard of care (SoC) affect advanced rod-cone dystrophy (RCD), a type of eye disease also known as retinitis pigmentosa.
- Trial name
- ECSTASI (NCT07854899)
- Testing
- Eye examinations, Questionnaire
- Where
- 3 sites in Belgium, France and 1 more
- Volunteers
- 100 people
- Sponsor
- SparingVision