Clinical trials: Retinal Dystrophies
Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP)
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
To evaluate the natural history of visual function in subjects with IRD phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by RPE65 or LRAT gene mutations.
- Trial name
- NCT02575430
- Testing
- No treatment: retrospective chart review
- Where
- 9 sites in United States, Canada and 5 more
- Volunteers
- 59 people
- Sponsor
- QLT Inc.
Retinitis Pigmentosa, Maculopathy, Age Related
High Resolution Retina Imaging
To increase the clinical experience of using the rtx1 camera in various retinal disorders and to follow the evolution of structural alterations during retinal diseases using adaptive optics imaging with the rtx1 camera
- Conditions
- Retinitis Pigmentosa, Maculopathy, Age Related, Macular Dystrophy, Macular Edema
- Trial name
- IHR (NCT04128150)
- Where
- 3 sites in France
- Volunteers
- 1,000 people
- Sponsor
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Retinitis Pigmentosa
The Perceptual Experience of Argus II Users
This Basic Experimental Studies Involving Humans (BESH) study investigates phosphene perception and performance factors in blind participants implanted with the Argus II retinal prosthesis system.
- Trial name
- NCT05285618
- Testing
- Argus II
- Where
- 3 sites in United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- University of California, Santa Barbara
Retinitis Pigmentosa
Stress and Vision Fluctuations in Retinitis Pigmentosa
Retinitis pigmentosa (RP) patients experience a slowly progressive, unpredictable loss of vision which eventually leads to bare or no light perception, posing a continuous threat to patients' independence.
- Trial name
- NCT00475254
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Johns Hopkins University
Retinitis Pigmentosa 11, Retinitis Pigmentosa
Natural History Study of Retinitis Pigmentosa Type 11
Observational study of patients with retinitis pigmentosa type 11
- Trial name
- ReSa (NCT04805658)
- Testing
- Clinical examinations
- Where
- Norway
- Volunteers
- 30 people
- Sponsor
- Oslo University Hospital
Retinitis Pigmentosa
Observational Study of the Argus® II Retinal Prosthesis System
The Argus II Retinal Implant is a revolutionary new device, which offers vision to patients who are blind from retinal degeneration - retinitis pigmentosa. These patients have no alternatives.
- Trial name
- NCT01999049
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- University Health Network, Toronto
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
Retinitis Pigmentosa
A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa
The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II \& III, and Choroideremia.
- Trial name
- NCT00447980
- Testing
- NT-501
- Where
- 12 sites in United States
- Volunteers
- 73 people
- Sponsor
- Neurotech Pharmaceuticals
Retinitis Pigmentosa
Promising ROd-cone DYstrophy Gene therapY
This is a two-step, multicenter, Phase I/II study including an open-label dose-escalation phase (Step 1) and a three-arm, controlled, double-masked, randomized extension phase (Step 2), in subjects with advanced RCD due to a…
- Trial name
- PRODYGY (NCT05748873)
- Testing
- SPVN06
- Where
- 6 sites in United States, France
- Volunteers
- 33 people
- Sponsor
- SparingVision
Stargardt Disease, Stargardt Disease 1
Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)
This is an observational prescreening study. Individuals who are eligible for prescreening will undergo testing procedures that may be used to determine eligibility in ACDN-01 clinical trials.
- Conditions
- Stargardt Disease, Stargardt Disease 1, Cone Rod Dystrophy, Juvenile Macular Degeneration
- Trial name
- NCT06445322
- Testing
- Prescreening Assessments
- Where
- 9 sites in United States
- Volunteers
- 50 people
- Sponsor
- Ascidian Therapeutics, Inc
Vision Functional Brain Networks in Patients With Hereditary Retinal Dystrophies
VIsual Cerebral ConnecTivity On Functional Magnetic Resonance Imaging in Patients With Hereditary REtinal Dystrophies
The Rothschild Foundation A Hospital follows a cohort of approximately 300 patients with hereditary retinal dystrophy. These patients are followed in ophthalmology consultation every year.
- Trial name
- VICTOIRE (NCT04648124)
- Testing
- IRMf-r (séquence resting state)
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Retinitis Pigmentosa Associated With CNGB1 Mutations
CNGB1 and Allied Disorders
Mutations in the rod-expressed gene, cyclic nucleotide-gated channel beta subunit (CNGB1) and associated inborn errors in metabolism are causes of retinal disease that causes progressive loss of vision.
- Trial name
- NCT04639635
- Testing
- No intervention, this is a natural history progression study
- Where
- 6 sites in United States, France and 2 more
- Volunteers
- 20 people
- Sponsor
- Columbia University
Mucolipidosis Type IV
The Natural History of Mucolipidosis Type IV
The purpose of this study is to define the natural history of Mucolipidosis Type IV and identify potential clinical outcome measures.
- Trial name
- NCT01067742
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Baylor Research Institute
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
BS01 in Patients With Retinitis Pigmentosa
A Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP (AAV2-CAG-ChronosFP) in Patients with Retinitis Pigmentosa
- Trial name
- NCT04278131
- Testing
- BS01
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Bionic Sight LLC
Retinitis Pigmentosa
Identifying Genes and Mutations Underlying Retinitis Pigmentosa and Allied Diseases
Retinitis Pigmentosa (RP) is the most common form of hereditary retinal degeneration, with a worldwide prevalence of 1:3500. It is one of the most genetically heterogenous conditions in humans, with over 100 causative genes and…
- Trial name
- NCT02309866
- Testing
- Genetic testing
- Volunteers
- 100 people
- Sponsor
- Hillel Yaffe Medical Center
Retinitis Pigmentosa, Retinitis Pigmentosa Syndrome
Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis Pigmentosa
This is an open-label, multiple ascending dose study to evaluate the safety, tolerability, and efficacy of EA-2353 in subjects with RP. Unilateral intravitreal injections (IVT) will be administered into the subject's Study Eye.
- Trial name
- NCT05392751
- Testing
- EA-2353
- Where
- 5 sites in United States
- Volunteers
- 14 people
- Sponsor
- Endogena Therapeutics, Inc
Retinitis Pigmentosa
Safety and Efficacy of Subretinal Transplantation of Clinical Human Embryonic Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Retinitis Pigmentosa
This project intends to transplant a clinical level human embryonic stem cells derived retinal pigment epithelium into subretinal space to treat eetinitis pigmentosa(RP) diseases.
- Trial name
- NCT03944239
- Testing
- Retinal pigment epitheliums transplantation
- Where
- China
- Volunteers
- 25 people
- Sponsor
- Qi Zhou
Leber Congenital Amaurosis
Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families.
The main objectives of this study are: 1. Improve genetic counseling by establishment of prevalences of each of genetic subtypes within a expanded population of patients with LCA taking into account ethnicity of families. 2.
- Trial name
- GENPHENACL (NCT02970266)
- Where
- France
- Volunteers
- 659 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Retinitis Pigmentosa
Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)
The rod-cone dystrophies (often referred to as retinitis pigmentosa (RP)) are a clinically and genetically heterogeneous group of disorders in which there is progressive loss of rod and later cone photoreceptor function leading…
- Trial name
- NCT03349242
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 140 people
- Sponsor
- MeiraGTx UK II Ltd