Clinical trials: Retinal Dystrophy, open to volunteers
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.
Stargardt Disease, Stargardt Macular Degeneration
A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)
This Phase 1/2 study will evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt's Type 1 (STGD1).
- Conditions
- Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy
- Trial name
- ASTRA (NCT06942572)
- Testing
- SB-007
- Where
- 5 sites in United States
- Volunteers
- 57 people
- Sponsor
- Splice Bio
Retinitis Pigmentosa, Retinal Disease
A Long-Term Follow-Up Study in Subjects Who Received vMCO-I Administered Via Intravitreal Injection
This study "A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection" is an observational…
- Conditions
- Retinitis Pigmentosa, Retinal Disease, Retinal Degeneration
- Trial name
- EXTEND (NCT05921162)
- Testing
- Gene Therapy product:vMCO-I
- Where
- India
- Volunteers
- 11 people
- Sponsor
- Nanoscope Therapeutics Inc.
Stargardt Disease, Retinitis Pigmentosa (RP)
Restoration of Central Vision With PRIMA in Patients With Photoreceptor Degeneration
The objective of this study is to evaluate the efficacy and safety of the PRIMA Products in participants with inherited retinal degeneration affecting the macula (including but not limited to Stargardt disease, and Retinitis…
- Conditions
- Stargardt Disease, Retinitis Pigmentosa (RP), Inherited Retinal Degeneration
- Trial name
- PRIMAlia (NCT07266584)
- Testing
- PRIMA Products
- Where
- Australia
- Volunteers
- 5 people
- Sponsor
- Science Corporation
Retinitis Pigmentosa
A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa
The goal of the study is to investigate the safety, tolerability and efficacy of up to 3 doses of KIO-301 administered by intravitreal (IVT) injection bilaterally every 6 weeks in patients with late-stage retinitis pigmentosa…
- Trial name
- ABACUS-2 (NCT06628947)
- Testing
- 100 μg KIO-301, 50 μg KIO-301
- Where
- 5 sites in Australia
- Volunteers
- 36 people
- Sponsor
- Kiora Pharmaceuticals, Inc.
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
Retinitis Pigmentosa
Role of UC-MSC and CM to Inhibit Vision Loss in Retinitis Pigmentosa Phase I/II
The study will perform UC-MSCs and CM transplantation. There are two groups with different dosages. The first group will be transplanted with 1.5 million cells, meanwhile, the second group is 5 million cells.
- Trial name
- NCT05909488
- Testing
- 1.5 x 10^6 UC-MSC + CM, 5 x 10^6 UC-MSC + CM
- Where
- Indonesia
- Volunteers
- 30 people
- Sponsor
- PT. Prodia Stem Cell Indonesia
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
Stargardt Disease Type 1 (STGD1)
An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT Disease
Stargardt disease type 1 (STGD1) is a rare genetic eye condition that causes progressive vision loss, often beginning in childhood or adolescence.
- Trial name
- START (NCT07063251)
- Testing
- HG005
- Where
- China
- Volunteers
- 6 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Retinitis Pigmentosa, Eye Diseases
Natural History Study of Patients With EYS-Associated RP
This natural history study of patients with EYS mutations from Russia and former CIS (Commonwealth of Independent States) territories will accelerate the development of outcome measures for clinical trials.
- Trial name
- RUS_EYS (NCT07228793)
- Testing
- Whole exome/genome sequencing
- Where
- Russia
- Volunteers
- 45 people
- Sponsor
- Sensor Technology for Deafblind
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
Primary Photoreceptor Disease, Retinitis Pigmentosa (RP)
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in approximately 54 adults with primary photoreceptor…
- Conditions
- Primary Photoreceptor Disease, Retinitis Pigmentosa (RP), Usher Syndrome, Inherited Retinal Disease (IRD)
- Trial name
- CLARICO (NCT06789445)
- Testing
- OpCT-001
- Where
- 5 sites in United States
- Volunteers
- 54 people
- Sponsor
- BlueRock Therapeutics
Retinitis Pigmentosa (RP), Inherited Retinal Diseases
Study to Evaluate the Efficacy of a Single Intravitreal Injection of RTx-015 in Participants With Retinitis Pigmentosa (PERCEPT)
The purpose of this clinical trial is to evaluate the effectiveness and safety of a single injection into the eye of an investigational gene therapy in participants with retinitis pigmentosa (RP).
- Trial name
- PERCEPT (NCT07901751)
- Testing
- RTx-015
- Where
- 14 sites in United States
- Volunteers
- 60 people
- Sponsor
- Ray Therapeutics, Inc.
AMD - Age-Related Macular Degeneration, Retinitis Pigmentosa (RP)
Retinal Investigation Using Optos OCT Device
Retinal investigation using OCT with control and diseased eyes
- Conditions
- AMD - Age-Related Macular Degeneration, Retinitis Pigmentosa (RP), Diabetic Retinopathy, Control Patients
- Trial name
- NCT06846151
- Testing
- retinal functional imaging
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Optos, PLC
Retinitis Pigmentosa (RP), Usher Syndrome
A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
The goal of this clinical trial is to learn if NPI-001 works to prevent progression of retinitis pigmentosa in adults diagnosed with Usher syndrome. It will also provide information about the safety of NPI-001.
- Trial name
- SLORP Preserve (NCT07710196)
- Testing
- NPI-001
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- Nacuity Pharmaceuticals, Inc.
Biallelic RPE65 Mutation-associated Retinal Dystrophy
Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy.
- Trial name
- NCT05858983
- Testing
- FT-001 Low Dose, FT-001 Mid Dose, FT-001 High Dose
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Frontera Therapeutics
Retinitis Pigmentosa, Choroideremia
Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration
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, Choroideremia, Stargardt Macular Dystrophy, Stargardt Disease
- Trial name
- NCT06375239
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- Ray Therapeutics, Inc.
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
Retinal Dystrophy Due to Biallelic ABCA4 Mutations, Stargardt Disease 1
Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt…
- Trial name
- NCT07002398
- Testing
- VG801
- Where
- China
- Volunteers
- 18 people
- Sponsor
- VeonGen Therapeutics GmbH
Retinitis Pigmentosa (RP), Rod Cone Dystrophy
Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy (SPVN20) in Subjects With Rod-Cone Dystrophy
This open-label, multi-center study is to evaluate the safety, tolerability, and efficacy of escalating doses of a gene therapy called SPVN20 administered via a single intravitreal injection in participants with advanced Rod-Cone…
- Trial name
- NYRVANA (NCT07807111)
- Testing
- SPVN20 (high dose), SPVN20 (medium dose), SPVN20 (low dose)
- Where
- 3 sites in Belgium, France and 1 more
- Volunteers
- 27 people
- Sponsor
- SparingVision