Clinical trials: Inherited Retinal Diseases, open to volunteers
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
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
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
The Effect of Oral Administration of 9-cis Rich Powder of the Alga Dunaliella Bardawil on Visual Functions in Adolescent Patients With Retinitis Pigmentosa
The aim of this study is to determine whether 9-cis-beta Caroten rich D. Brdawiil extract is effective in the treatment of retinitis pigmentosa in adolescent patients.
- Trial name
- NCT02018692
- Testing
- Alga Dunaliella Bardawil powder
- Where
- Israel
- Volunteers
- 30 people
- Sponsor
- Sheba Medical Center
Retinal Disease, Age-Related Macular Degeneration
Stem Cell Ophthalmology Treatment Study II
This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.
- Conditions
- Retinal Disease, Age-Related Macular Degeneration, Retinitis Pigmentosa, Stargardt Disease
- Trial name
- SCOTS2 (NCT03011541)
- Testing
- Arm 1
- Where
- 4 sites in United States, Austria and 1 more
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
Neonatal Pain, Retinopathy of Prematurity (ROP)
Fentanyl Intranasal for Retinopathy of Prematurity Screening in Preterm Infants
The goal of this clinical trial is to learn whether intranasal fentanyl (a pain medicine given as a nasal spray) can reduce pain and is safe to use during routine eye examinations for retinopathy of prematurity (ROP) in preterm…
- Conditions
- Neonatal Pain, Retinopathy of Prematurity (ROP), Pain Management, Infant, Premature
- Trial name
- FIREFLY (NCT07112430)
- Testing
- Fentanyl Citrate (Intranasal)
- Where
- Canada
- Volunteers
- 58 people
- Sponsor
- Marsha Campbell-Yeo
Intermediate AMD
A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD
The proposed clinical investigation wants primary to validate the safety of the innovative light therapy approach and in second priority provide insight and confirmations on therapeutic effect.
- Trial name
- retinaSEES-PoC (NCT06557369)
- Testing
- reSEES
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- Oculox Technologies SA
X-Linked Retinitis Pigmentosa
A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.
- Trial name
- NCT06275620
- Testing
- AGTC-501 (high dose and standard corticosteroid regimen), AGTC-501 (low dose and standard corticosteroid regimen), AGTC-501 (high dose and modified corticosteroid regimen)
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Beacon Therapeutics
Retinitis Pigmentosa (RP), Rod Cone Dystrophy
Brain Stimulation Effects on Orientation and Mobility Skills in Adults With Vision Impairment
This pilot clinical trial evaluates whether non-invasive brain stimulation improves the orientation and mobility (O\&M) skills of individuals with constricted visual fields in both eyes. The study is composed of three visits.
- Conditions
- Retinitis Pigmentosa (RP), Rod Cone Dystrophy, Visually Impaired Persons, Peripheral Visual Field Defect of Both Eyes
- Trial name
- NCT07341763
- Testing
- Active hf-tRNS.
- Where
- Canada
- Volunteers
- 20 people
- Sponsor
- University of Waterloo
LCA5
Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
The goals of this clinical trial are assess the natural course of LCA5-IRD over 6 months and to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-001 in patients with inherited retinal degeneration…
- Trial name
- NCT05616793
- Testing
- AAV8.hLCA5
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Opus Genetics, Inc
Retinitis Pigmentosa (RP), Usher Syndrome
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
- NCT07290530
- Testing
- N-acetylcysteine amide
- Volunteers
- 80 people
- Sponsor
- Nacuity Pharmaceuticals, Inc.
Usher Syndromes
Natural History Study of Usher Syndrome ( Light4Deaf )
Clinical centres in the LIGHT4DEAF consortium have developed and will continue to improve a reliable, early molecular diagnosis and protocols for full clinical characterisation of Usher syndrome, which will be valuable for the…
- Trial name
- Light4Deaf (NCT04665726)
- Where
- 4 sites in France
- Volunteers
- 400 people
- Sponsor
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Retinopathy of Prematurity (ROP), Premature Birth
Long-term Ophthalmic Outcomes in Ex-premature Infants
There is limited knowledge on ophthalmological outcomes in the adult population that was born prematurely. This study aims to evaluate the ophthalmic outcomes of ex-premature infants that have reached adolescence or adulthood.
- Trial name
- LTOO-XP (NCT06649513)
- Testing
- eye drops (Tropicamid)
- Where
- Netherlands
- Volunteers
- 210 people
- Sponsor
- Erasmus Medical Center
Leber Congenital Amaurosis 10, Blindness
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A>G…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders
- Trial name
- HYPERION (NCT06891443)
- Testing
- sepofarsen
- Where
- 18 sites in United States, Australia and 8 more
- Volunteers
- 32 people
- Sponsor
- Laboratoires Thea
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 (RP), Diabetic Retinopathy (DR)
Functional Assessments in Vision Impairment
The aim of the research project is to validate the use of a novel functional assessment tool designed to document how participants with a vision impairment complete activities of daily living in a real world environment.
- Conditions
- Retinitis Pigmentosa (RP), Diabetic Retinopathy (DR), Age Related Macular Degeneration (AMD), Glaucoma
- Trial name
- NCT06908161
- Where
- Australia
- Volunteers
- 45 people
- Sponsor
- Center for Eye Research Australia
ROSAH
Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients
The purpose of this study is to evaluate the safety and tolerability of DF-003 in retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and migraine headache (ROSAH) syndrome patients.
- Trial name
- NCT06395285
- Testing
- DF-003
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 12 people
- Sponsor
- Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)
Retinoblastoma, Chemotherapy Effect
Topotecan and Melphalan for Retinoblastoma
The purpose of this study is to evaluate topotecan and melphalan for retinoblastoma patients.
- Trial name
- NCT04799002
- Testing
- Topotecan, Melphalan
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Sun Yat-sen University
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
Von Hippel Lindau, Von Hippel Lindau Disease
Real-World Effectiveness and Pharmacogenetics of Belzutifan in VHL Syndrome: The BELIEVE-VHL Trial
The BELIEVE-VHL Trial is a prospective real-life study designed to evaluate the therapeutic effects, benefits, and adverse effects of belzutifan, as well as the timing of treatment response and disease progression in patients…
- Conditions
- Von Hippel Lindau, Von Hippel Lindau Disease, Von Hippel Lindau-Deficient Clear Cell Renal Cell Carcinoma, Hemangioblastoma (HB) of the Central Nervous System (CNS)
- Trial name
- BELIEVE-VHL (NCT07167329)
- Testing
- Belzutifan
- Where
- Brazil
- Volunteers
- 100 people
- Sponsor
- José Claudio Casali da Rocha