Clinical trials: Retinal Dystrophies
Retinitis Pigmentosa
A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa
This is a Phase 3 study to Assess the Efficacy, Safety and Tolerability of OCU400 in patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical…
- Trial name
- liMeliGhT (NCT06388200)
- Testing
- Sub-Retinal Administration of OCU400-301
- Where
- 17 sites in United States, Canada
- Volunteers
- 140 people
- Sponsor
- Ocugen
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.
Stargardt's Macular Dystrophy
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 safety and tolerability of RPE cellular therapy in patients with SMD . To evaluate potential efficacy endpoints to be used in future studies RPE cellular therapy.
- Trial name
- NCT01469832
- Testing
- MA09-hRPE
- Where
- 3 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Astellas Institute for Regenerative Medicine
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
Autosomal Dominant Retinitis Pigmentosa, Eye Diseases
A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene
This study evaluates the safety, tolerability and efficacy of QR-1123 injection in the eye (intravitreal; IVT) injections (one eye/unilateral) in subjects receiving a single dose or repeat doses.
- Conditions
- Autosomal Dominant Retinitis Pigmentosa, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies
- Trial name
- AURORA (NCT04123626)
- Testing
- QR-1123
- Where
- 5 sites in United States
- Volunteers
- 11 people
- Sponsor
- ProQR Therapeutics
Stargardt Disease, Stargardt Macular Degeneration
Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)
The purpose of this open-label, multicenter study is to determine the long-term safety, pharmacokinetics and effects of ALK-001 (C20-D3-retinyl acetate) on the progression of Stargardt disease.
- Conditions
- Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
- Trial name
- NCT04239625
- Testing
- ALK-001
- Where
- 8 sites in United States
- Volunteers
- 200 people
- Sponsor
- Alkeus Pharmaceuticals, Inc.
Usher Syndrome, Congenital Deafness
Clinical and Genetic Testing of Patients With Usher Syndrome
This study is aimed to characterize Russian population of Usher patients.
- Conditions
- Usher Syndrome, Congenital Deafness, Retinitis Pigmentosa
- Trial name
- NCT03319524
- Where
- 6 sites in Russia
- Volunteers
- 28 people
- Sponsor
- Sensor Technology for Deafblind
X-Linked Retinitis Pigmentosa
A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112
The objective of the study is to evaluate the safety, tolerability and efficacy of a single sub-retinal injection of BIIB112 in participants with X-linked retinitis pigmentosa (XLRP).
- Trial name
- XIRIUS (NCT03116113)
- Testing
- BIIB112
- Where
- 8 sites in United States, United Kingdom
- Volunteers
- 50 people
- Sponsor
- Biogen
Retinitis Pigmentosa, Usher Syndrome Type 2
Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)
The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with…
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- NCT05176717
- Testing
- QR-421a
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 5 people
- Sponsor
- Laboratoires Thea
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
Retinitis Pigmentosa
Safety and Tolerability of hRPC in Retinitis Pigmentosa
hRPC is a cell therapy for retinitis pigmentosa. This is a first-in-human, dose escalation study in which participants with retinitis pigmentosa will receive a single subretinal injection of hRPC cells in one eye to evaluate…
- Trial name
- hRPCRP (NCT02464436)
- Testing
- hRPC
- Where
- 5 sites in United States, Spain and 1 more
- Volunteers
- 29 people
- Sponsor
- ReNeuron Limited
Leber Congenital Amaurosis, LCA
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
Primary Objective: To evaluate the safety and tolerability of ascending doses of ATSN-101 administered as a unilateral subretinal injection in patients with Leber Congenital Amaurosis (LCA) caused by autosomal recessive guanylate…
- Conditions
- Leber Congenital Amaurosis, LCA, LCA1
- Trial name
- NCT03920007
- Testing
- ATSN-101, ATSN-101 Diluent Solution, Prednisone
- Where
- 2 sites in United States
- Volunteers
- 15 people
- Sponsor
- Atsena Therapeutics Inc.
Retinitis Pigmentosa, Choroideremia
Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa or Choroideremia Patients (ENVISION)
A Phase 1, open-label, non-randomized, dose-escalation study, where approximately 18 eligible patients with retinitis pigmentosa or choroideremia will be enrolled sequentially in up to 4 dose cohorts of RTx-015.
- Trial name
- NCT06460844
- Testing
- RTx-015
- Where
- 4 sites in United States
- Volunteers
- 10 people
- Sponsor
- Ray 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 (RP), Usher Syndrome Type 2
Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
The purpose of this Phase 2 Open-label study is to evaluate the long-term safety and tolerability of Ultevursen administered via intravitreal injection (IVT) in participants with Retinitis Pigmentosa due to mutations in exon 13…
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- CAPELLA (NCT07796646)
- Testing
- No intervention, will not receive any active study intervention, Intravitreal Injection of Ultevursen
- Volunteers
- 81 people
- Sponsor
- Laboratoires Thea
Stargardt Disease, Age-related Macular Degeneration
Phase 1 Safety Study of ALK-001 in Healthy Volunteers
This protocol is a phase 1 clinical study to assess the safety and pharmacokinetics of ALK-001 in healthy volunteers. Please contact trials@alkeus.com for any questions.
- Conditions
- Stargardt Disease, Age-related Macular Degeneration, Other Retinal Dystrophies
- Trial name
- NCT02230228
- Testing
- ALK-001 (No generic name)
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Alkeus Pharmaceuticals, Inc.
Stargardt's Macular Dystrophy
Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)
The purpose of this study is to evaluate the safety and efficacy of avacincaptad pegol intravitreal injection compared to Sham in participants with autosomal recessive Stargardt disease 1 (STGD1).
- Trial name
- NCT03364153
- Testing
- avacincaptad pegol
- Where
- 41 sites in United States, France and 6 more
- Volunteers
- 121 people
- Sponsor
- Astellas Pharma Global Development, 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.
Retinal Degeneration, Inherited Retinal Dystrophy Primarily Involving Sensory Retina
Impact on Daily Life of Patients Using the Subretinal Implant RETINA IMPLANT Alpha AMS
This study aims to assess the impact of the subretinal implant RETINA IMPLANT Alpha AMS on the patient's daily life, using validated activities of daily living and questionnaires.
- Trial name
- NCT03561922
- Testing
- RETINA IMPLANT Alpha AMS
- Where
- 2 sites in France
- Volunteers
- 2 people
- Sponsor
- Retina Implant AG
Retinitis Pigmentosa
Development of Visual Function Evaluation Method
The objective of this study is to develop the comprehensive visual function evaluation method in severe visually impaired patient
- Trial name
- NCT03281005
- Where
- Japan
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Astellas Pharma Inc