Clinical trials: Inherited Retinal Diseases
Inherited Retinal Diseases
Extension Study of the PUMCH-E101 Clinical Trial：Safety and Efficacy Study of PUMCH-E101 Injection in the Contralateral Eye
The goal of this clinical trial is to evaluate the safety and efficacy of PUMCH-E101 injection in the contralateral eye of subjects with RDH12 retinopathy.
- Trial name
- NCT07878572
- Testing
- PUMCH-E101 Injection(High dose)
- Where
- China
- Volunteers
- 8 people
- Sponsor
- Peking Union Medical College Hospital
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
Inherited Retinal Diseases
Global Patient Registry of Inherited Retinal Diseases
The purpose of this study is to better understand the natural history of Inherited Retinal Disease (IRD) and help inform patient management.
- Trial name
- EYERD Registry (NCT05957276)
- Testing
- Standard of Care
- Where
- 75 sites in United States, Australia and 16 more
- Volunteers
- 886 people
- Sponsor
- Janssen Research & Development, LLC
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
Inherited Retinal Dystrophy, Inherited Retinal Disease
Safety and Efficacy Study of PUMCH-E111 Injection in Subjects With RLBP1 Related Inherited Retinal Dystrophy
The goal of this clinical trial is to evaluate the safety and efficacy of PUMCH-E111 injection in subjects with RLBP1 related Inherited Retinal Dystrophy.
- Trial name
- NCT07656753
- Testing
- PUMCH-E111 Injection（Low dose）, PUMCH-E111 Injection（High dose）
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Peking Union Medical College Hospital
Retinitis Pigmentosa, Choroideremia
A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)
A phase I/II dose-escalating study of the safety, tolerability and efficacy of KIO-301 administered intravitreally to patients with retinitis pigmentosa and choroideremia (ABACUS).
- Conditions
- Retinitis Pigmentosa, Choroideremia, Non-Syndromic Rod-Dominant Inherited Retinal Diseases
- Trial name
- NCT05282953
- Testing
- KIO-301
- Where
- 3 sites in Australia
- Volunteers
- 32 people
- Sponsor
- Kiora Pharmaceuticals, Inc.
Inherited Retinal Diseases
Optimizing Low Vision Rehabilitation in Emotionally Distressed Patients With Inherited Retinal Diseases
The purpose of this study is to understand if combining Low Vision Rehabilitation (LVR) with Emotional Regulation Therapy (ERT) can help people with inherited retinal diseases (IRDs) that experience emotional distress related to…
- Trial name
- NCT06651736
- Testing
- Low Vision Rehabilitation (LVR), Emotion Regulation Therapy (ERT)
- Where
- United States
- Volunteers
- 180 people
- Sponsor
- University of Michigan
Inherited Retinal Diseases
An Assistant Model for IRD Care Needs: A Randomized Control Trial
we present FM-IRD（also designated as Retina4IRD）, the first foundation model-based AI system specifically designed for genotype diagnosis of IRD by emulating clinician decision-making reasoning process.
- Trial name
- IRD (NCT06839170)
- Testing
- FM-IRDs assisted, without FM-IRDs
- Where
- 2 sites in China
- Volunteers
- 300 people
- Sponsor
- Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Inherited Retinal Disease
Multicenter, Non-interventional Study, Describing Patients With Inherited Retinal Disease (IRD) in France
Genetic diagnostic testing becomes increasingly important for enhancing our understanding of the disease notably the genetics and providing the best care to the patients, and several initiatives seek to gather more data in order…
- Trial name
- EPI-genRET (NCT05122442)
- Testing
- Patients with inherited retinal disease
- Where
- France
- Volunteers
- 998 people
- Sponsor
- University Hospital, Strasbourg, France
Age - Related Macular Degeneration (AMD), Diabetic Macular Edema
Wide Field OCTA in Ocular Diseases
The main retinal diseases, whether or not associated with specific mutations genetic, cause progressive degeneration of vascular retinal structures and not vascular, resulting in decreased visual function.
- Conditions
- Age - Related Macular Degeneration (AMD), Diabetic Macular Edema, Diabetic Retinopathy, Myopia
- Trial name
- WOOD-2025 (NCT07298174)
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- IRCCS San Raffaele
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.
Retinitis Pigmentosa, Inherited Retinal Dystrophy
A Multicenter, Randomized, Double-Blind, Controlled Clinical Study of Minocycline for the Treatment of Retinitis Pigmentosa
This is a multicenter, double-blind, randomized controlled clinical trial to get high - level evidence on minocycline's efficacy and safety（100mg/d, 200mg/d) for Retinitis pigmentosa and to find the optimal treatment dose.
- Conditions
- Retinitis Pigmentosa, Inherited Retinal Dystrophy, Retina Disorder
- Trial name
- NCT07082855
- Testing
- Minocycline 100mg, Minocycline 200mg
- Where
- China
- Volunteers
- 126 people
- Sponsor
- Zhongshan Ophthalmic Center, Sun Yat-sen University
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- 2 sites in United States, Australia
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Inherited Retinal Diseases
Safety and Efficacy Study of PUMCH-E101 Injection in Subjects with RDH12 Retinopathy
The goal of this clinical trial is to evaluate the safety and efficacy of PUMCH-E101 injection in subjects with RDH12 retinopathy.
- Trial name
- NCT06749639
- Testing
- PUMCH-E101 Injection（Low dose）, PUMCH-E101 Injection（High dose）
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Peking Union Medical College Hospital
Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.
- Trial name
- NCT05906953
- Testing
- HG004
- Where
- 3 sites in United States, China
- Volunteers
- 20 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Retinitis Pigmentosa, Inherited Retinal Dystrophy
Minocycline Treatment in Retinitis Pigmentosa
The aim of this study is to evaluate the efficacy and safety of oral minocycline (100mg/d), administered for 6 months, for the treatment of patients with retinitis pigments(RP).
- Conditions
- Retinitis Pigmentosa, Inherited Retinal Dystrophy, Retina Disorder
- Trial name
- NCT04068207
- Testing
- Minocycline
- Where
- China
- Volunteers
- 35 people
- Sponsor
- Sun Yat-sen University
Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies
Single Ascending Dose Study in Participants With LCA10
The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound…
- Conditions
- Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease
- Trial name
- NCT03872479
- Testing
- EDIT-101
- Where
- 5 sites in United States
- Volunteers
- 34 people
- Sponsor
- Editas Medicine, Inc.
Inherited Ophthalmic Diseases, Inherited Retinal Degeneration
Interferon Gamma-1b Administered Topically for Macular Edema/Intraretinal Schisis Cysts in Rod-Cone Dystrophy (RCD) and Enhanced S-Cone Syndrome (ESCS)
Background: People with rod-cone dystrophy (RCD) or enhanced S-cone syndrome (ESCS) have excess fluid under the retina of their eye. This can cause vision loss.
- Trial name
- NCT02338973
- Testing
- Interferon gamma-1b
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- National Eye Institute (NEI)
Healthy Volunteer, Retinal Disease
Color Vision as a Measure for Inherited Retinal Diseases
Background: The purpose of this study is to find out whether color vision measured with the Cambridge Color Test is a good way to examine the severity of inherited retinal diseases (IRDs).
- Conditions
- Healthy Volunteer, Retinal Disease, Inherited Retinal Degeneration
- Trial name
- NCT01878032
- Where
- United States
- Volunteers
- 166 people
- Sponsor
- National Eye Institute (NEI)