Clinical trials: Inherited Retinal Diseases
Cytomegalovirus Retinitis, HIV Infections
A Randomized Study Comparing the Safety and Efficacy of Two Regimens of Oral Ganciclovir to Intravenous Ganciclovir Maintenance Therapy for Cytomegalovirus Retinitis in People With AIDS Who Have Received Prior Ganciclovir Therapy
To compare the safety and tolerance of oral ganciclovir at a double dose 3 times/day or a single dose 6 times/day to IV ganciclovir given for 20 weeks of maintenance therapy.
- Trial name
- NCT00002247
- Testing
- Ganciclovir
- Where
- 20 sites in United States, Canada
- Volunteers
- 225 people
- Sponsor
- Hoffmann-La Roche
Retinitis Pigmentosa
Observational Natural History Study of Autosomal Dominant Retinitis Pigmentosa (adRP)
The purpose of this study is to gain an understanding of how adRP progresses over time in patients with misfolded rod opsin mutations.
- Trial name
- NCT02926092
- Testing
- Observation
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Shire
Stargardt Disease
Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study
The current study is a non-interventional long-term safety follow-up of the subjects who completed STARLIGHT, in accordance with FDA guidance on recipients of human gene therapy products.
- Trial name
- SUSTAIN (NCT06048185)
- Testing
- Gene Therapy Product-MCO-010
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Nanoscope Therapeutics Inc.
Bietti Crystalline Dystrophy, Inherited Retinal Diseases
Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients With Bietti Crystalline Dystrophy
This is a phase 3 study to evaluate the efficacy and safety of VGR-R01 in subjects with Bietti Crystalline Dystrophy. This is a multicenter, randomized controlled study which will enroll 45 subjects.
- Trial name
- NCT06699108
- Testing
- VGR-R01
- Where
- China
- Volunteers
- 45 people
- Sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.
Leber Congenital Amaurosis 10, Blindness
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- ILLUMINATE (NCT03913143)
- Testing
- sepofarsen
- Where
- 14 sites in United States, Belgium and 7 more
- Volunteers
- 36 people
- Sponsor
- ProQR Therapeutics
Retinitis Pigmentosa, Choroideremia
Study of a Suprachoroidal Retinal Prosthesis
This study is a proof of principal, to evaluate a safety and efficacy of a prototype suprachoroidal retinal implant.
- Trial name
- NCT03406416
- Testing
- 44Ch Bionic Eye Device
- Where
- Australia
- Volunteers
- 4 people
- Sponsor
- Mobius Medical Pty Ltd.
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
Macular Degeneration, Geographic Atrophy
A Prospective Observational Study to Assess the Reliability and Validity of the MLSDT
The goal of this observational study is to assess the reliability and validity of the 9-object MLSDT for evaluation of participants with moderate to severe vision impairment when tested without a wearable low-vision magnifying…
- Conditions
- Macular Degeneration, Geographic Atrophy, Stargardt Disease
- Trial name
- NCT06805474
- Testing
- Observational
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Nanoscope Therapeutics Inc.
X-Linked Retinitis Pigmentosa
A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501/laruparetigene zovaparvovec )) to an untreated control group in male participants…
- Trial name
- NCT04850118
- Testing
- rAAV2tYF-GRK1-hRPGRco, Control
- Where
- 25 sites in United States, Australia and 2 more
- Volunteers
- 85 people
- Sponsor
- Beacon Therapeutics
Retinoblastoma
Retinoblastoma Phase II Expanded Access Clinical Trial
In this research study investigators want to learn more about treatment of advanced or recurrent retinoblastoma. For children with retinoblastoma that have an advanced stage of presentation in one eye or if they have failed all…
- Trial name
- NCT06679634
- Testing
- Chemoplaque also referred to as Episcleral Topotecan
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Targeted Therapy Technologies, LLC
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.
Macular Degeneration, Age-Related Maculopathies
Safety and Tolerability Study of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AMD)
This Phase 1 clinical research study will examine the safety and tolerability of an experimental gene transfer agent, AAV2-sFLT01, in patients with Neovascular Age-Related Macular Degeneration (AMD).
- Conditions
- Macular Degeneration, Age-Related Maculopathies, Age-Related Maculopathy, Maculopathies, Age-Related
- Trial name
- NCT01024998
- Testing
- AAV2-sFLT01
- Where
- 4 sites in United States
- Volunteers
- 19 people
- Sponsor
- Genzyme, a Sanofi Company
Retinitis Pigmentosa, Cone-Rod Dystrophy
IRIS PILOT - Extended Pilot Study With a Retinal Implant System
Investigate whether blind subjects that fulfil the patient criteria provided with a Retinal Implant are able to differentiate between simple patterns like horizontal bar, vertical bar and cross.
- Conditions
- Retinitis Pigmentosa, Cone-Rod Dystrophy, Choroideremia
- Trial name
- NCT00427180
- Testing
- Retinal Implant System (IRIS)
- Volunteers
- 20 people
- Sponsor
- Intelligent Medical Implants GmbH
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
X-Linked Retinitis Pigmentosa (XLRP)
A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa
The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of…
- Trial name
- Landscape (NCT07174726)
- Testing
- Adeno-associated virus vector expressing a human RPGR gene
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Beacon Therapeutics
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.
Diabetic Retinopathy
A Study to Evaluate the Efficacy and Safety of Tarcocimab Tedromer Compared With Sham Treatment in Participants With Diabetic Retinopathy (DR)
This study will demonstrate that tarcocimab 5 mg is superior to sham treatment in participants with DR.
- Trial name
- GLOW2 (NCT06270836)
- Testing
- Tarcocimab
- Where
- 39 sites in United States, Puerto Rico
- Volunteers
- 255 people
- Sponsor
- Kodiak Sciences Inc
Solid Tumors With PTCH1 Loss-of-function Mutations
A Study Evaluating the Safety and Efficacy of ENV-101 (Taladegib) in Patients With Advanced Solid Tumors Harboring PTCH1 Loss of Function Mutations
This study employs a 2-stage design that aims to evaluate the efficacy and safety of ENV- 101, a potent Hedgehog (Hh) pathway inhibitor, in patients with refractory advanced solid tumors characterized by loss of function (LOF)…
- Trial name
- NCT05199584
- Testing
- ENV-101 (taladegib)
- Where
- 16 sites in United States
- Volunteers
- 20 people
- Sponsor
- Endeavor Biomedicines, Inc.
Retinitis Pigmentosa
In Depth Observational Clinical Trial Of Retinitis Pigmentosa Patients
Typically, clinical research participation favors a specific demographic group, and little research exists on how trial attributes affect participation.
- Trial name
- NCT05849987
- Volunteers
- 500 people
- Sponsor
- Power Life Sciences Inc.
Outer Retinal Degeneration, Retinitis Pigmentosa
Argus® II Retinal Prosthesis System Post-Market Surveillance Study
This post-market surveillance study is conducted in the European Economic Area where Argus II has been CE certified for use in outer retinal degeneration patients.
- Trial name
- NCT01490827
- Where
- 10 sites in Germany, Italy
- Volunteers
- 52 people
- Sponsor
- Second Sight Medical Products