Clinical trials: Inherited Ophthalmic Diseases
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
Dry Age-related Macular Degeneration
EXPLORE: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005
The purpose of this clinical study was to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with geographic atrophy secondary to age-related macular degeneration…
- Trial name
- EXPLORE (NCT04437368)
- Testing
- GT005
- Where
- 55 sites in United States, Australia and 6 more
- Volunteers
- 98 people
- Sponsor
- Gyroscope Therapeutics Limited
Behçet Disease
A Study to Evaluate the Efficacy and Safety of Hemay005 in the Treatment of Behçet Disease
This is a phase 2, multi-center, randomized, placebo-controlled, double-blind, parallel-group study with an equal randomization among the Hemay005 high dose, lower dose and placebo treatment groups.
- Trial name
- NCT04609397
- Testing
- Hemay005
- Where
- 31 sites in China
- Volunteers
- 89 people
- Sponsor
- Tianjin Hemay Pharmaceutical Co., Ltd
Dry Age-related Macular Degeneration, Geographic Atrophy
Investigating Geographic Atrophy Insights (i-GAIN) Natural History Study
An observational study to investigate the natural history and evaluate biomarkers of participants with geographic atrophy secondary to age-related macular degeneration
- Conditions
- Dry Age-related Macular Degeneration, Geographic Atrophy, Retinal Disease, Macular Degeneration
- Trial name
- i-GAIN (NCT05797896)
- Where
- 22 sites in United States, United Kingdom
- Volunteers
- 234 people
- Sponsor
- Complement Therapeutics
Age-Related Macular Degeneration
NG101 AAV Gene Therapy in Subjects With Wet Age-Related Macular Degeneration
This study will evaluate the safety, tolerability, and preliminary efficacy of NG101 AAV gene therapy administered by subretinal injections into a single selected eye as a single selected dose for patients with wet age-related…
- Trial name
- NCT05984927
- Testing
- NG101 AAV gene therapy
- Where
- 4 sites in United States, Canada
- Volunteers
- 20 people
- Sponsor
- Elisigen, Inc.
Thyroid Eye Disease
Efficacy and Safety of Lonigutamab in Subjects With Thyroid Eye Disease (TED)
Phase 1/2, multicenter, multiple dose clinical study designed to evaluate lonigutamab in subjects with TED.
- Trial name
- TED (NCT05683496)
- Testing
- lonigutamab
- Where
- 11 sites in United States, Australia
- Volunteers
- 31 people
- Sponsor
- ACELYRIN Inc.
AMD, nAMD
Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD
ABBV-RGX-314 (also known as RGX-314) is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD or nAMD).
- Conditions
- AMD, nAMD, Wet Age-related Macular Degeneration, wAMD
- Trial name
- ATMOSPHERE (NCT04704921)
- Testing
- ABBV-RGX-314, Ranibizumab (LUCENTIS®)
- Where
- 89 sites in United States
- Volunteers
- 671 people
- Sponsor
- AbbVie
Fuchs' Endothelial Dystrophy, Fuchs Dystrophy
TTHX1114(NM141) in Combination With DWEK/DSO
Open label, single-treatment, with a concurrent non-treatment control
- Conditions
- Fuchs' Endothelial Dystrophy, Fuchs Dystrophy, Fuchs
- Trial name
- NCT04676737
- Testing
- TTHX1114(NM141)
- Where
- 11 sites in United States
- Volunteers
- 49 people
- Sponsor
- Trefoil Therapeutics, Inc.
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
Retinopathy of Prematurity
Regulatory PMS Study for Lucentis® in Patients With Retinopathy of Prematurity
This study was an open-labeled, multicenter, single arm, observational post-marketing surveillance study under routine clinical practice with no mandated treatments, visits or assessments.
- Trial name
- NCT05304949
- Testing
- Lucentis
- Where
- 6 sites in South Korea
- Volunteers
- 69 people
- Sponsor
- Novartis Pharmaceuticals
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
Safety, Tolerability
A Study of GenSci098 in Subjects With Active Thyroid Eye Disease
To evaluate the safety and tolerability of single and multiple ascending subcutaneous doses of GenSci098 in patients with thyroid eye disease (TED)
- Conditions
- Safety, Tolerability, GenSci098, Thyroid Eye Disease (TED)
- Trial name
- NCT06569758
- Testing
- GenSci098
- Where
- China
- Volunteers
- 76 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Thyroid Eye Disease
A Study of TEPEZZA® Treatment in Participants With Thyroid Eye Disease
The main goal of this study is to learn how teprotumumab will be processed in the body (Pharmacokinetics) subcutaneously and whether it is safe and tolerable after administration into adult patients with thyroid eye disease.
- Trial name
- NCT06389578
- Testing
- Teprotumumab
- Where
- 3 sites in United States
- Volunteers
- 16 people
- Sponsor
- Amgen
Carcinoid, Neuroendocrine
Safety Study of Seneca Valley Virus in Patients With Solid Tumors With Neuroendocrine Features
The primary purpose of the study is to determine if Seneca Valley Virus may be administered safely to patients with certain types of advanced cancer.
- Trial name
- NCT00314925
- Testing
- Seneca Valley Virus (biological agent)
- Where
- 10 sites in United States
- Volunteers
- 60 people
- Sponsor
- Neotropix
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
Lower Eyelid Steatoblepharon (Excess Eyelid Fat)
Phase 2 Study of XAF5 (XOPH5) Ointment for Reduction of Excess Eyelid Fat (Steatoblepharon)
This study will test whether XAF5 (XOPH5) ointment, applied once daily to the lower eyelids for 10 weeks, reduces the prominence of lower eyelid fat in adults with moderate to severe steatoblepharon (excess eyelid fat).
- Trial name
- NCT02230761
- Testing
- XOPH5 Ointment
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Topokine Therapeutics, Inc.
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
Thyroid Eye Disease
A Safety, Tolerability, and Efficacy Study of Veligrotug (VRDN 001) in Participants With Thyroid Eye Disease (TED)
The investigational drug, veligrotug (VRDN-001), is a monoclonal antibody that inhibits the activity of a cell surface receptor called insulin-like growth factor-1 receptor (IGF-1R).
- Trial name
- THRIVE (NCT05176639)
- Testing
- Veligrotug
- Where
- 53 sites in United States, Australia and 5 more
- Volunteers
- 113 people
- Sponsor
- Viridian Therapeutics, Inc.
Leber Hereditary Optic Neuropathy
Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.
- Trial name
- NCT02064569
- Testing
- GS010
- Where
- France
- Volunteers
- 19 people
- Sponsor
- GenSight Biologics