Clinical trials: Inherited Ophthalmic Diseases
- Condition Inherited Ophthalmic Diseases
- Funder type Industry (pharma, biotech, devices)
- Has posted results yes
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- RESCUE (NCT02652767)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 39 people
- Sponsor
- GenSight Biologics
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- REVERSE (NCT02652780)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 37 people
- Sponsor
- GenSight Biologics
Retinopathy of Prematurity (ROP)
Long-term Safety and Efficacy Outcome Study Comparing Children Previously Enrolled in Study ROPP-2008-01 for the Prevention of Retinopathy of Prematurity (ROP)
The main purpose of this study is to evaluate the long-term efficacy and safety outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (NCT01096784).
- Trial name
- PEDAL (NCT02386839)
- Testing
- rhIGF-1/rhIGFBP-3
- Where
- 16 sites in United States, Italy and 4 more
- Volunteers
- 76 people
- Sponsor
- Shire
Thyroid Eye Disease, Graves Orbitopathy
A Study of the Efficacy and Safety of Secukinumab 300 mg in Patients With Thyroid Eye Disease (TED)
Thyroid eye disease (TED) is a rare autoimmune, inflammatory disorder of the orbit and represents the most common extra-thyroidal manifestation of Graves' disease (GD).
- Trial name
- ORBIT (NCT04737330)
- Testing
- Secukinumab
- Where
- 5 sites in Germany
- Volunteers
- 28 people
- Sponsor
- Novartis Pharmaceuticals
Thyroid Eye Disease
An Open-label Study (OLE) for Non-responders of VRDN-001-101 and VRDN-001-301
The investigational drug, 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
- OLE (NCT06179875)
- Testing
- Veligrotug
- Where
- 45 sites in United States, France and 6 more
- Volunteers
- 139 people
- Sponsor
- Viridian Therapeutics, Inc.
Behçet Disease
Dusquetide for the Treatment of Behcet's Disease
This is a clinical study to see if dusquetide can treat flares of oral and genital ulcers caused by Behcet's Disease. Study participants will receive an infusion of dusquetide twice a week for 4 weeks (8 treatments total), with…
- Trial name
- NCT06386744
- Testing
- Dusquetide
- Where
- Turkey (Türkiye)
- Volunteers
- 8 people
- Sponsor
- Soligenix
Retinitis Pigmentosa
Feasibility Study of a Chronic Retinal Stimulator in Retinitis Pigmentosa
The objective of this study is to evaluate the safety and efficacy of the retinal stimulation system by evaluating the data after chronic implantation.
- Trial name
- (A16) (NCT00279500)
- Testing
- Argus 16 Retinal Stimulation System
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Second Sight Medical Products
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
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
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.
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.
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl).
- Trial name
- NCT00999609
- Testing
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Spark Therapeutics, Inc.
Thyroid Eye Disease, Graves' Orbitopathy
Treatment of Graves' Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis With Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study
The overall objective is to investigate the efficacy, tolerability, and safety of teprotumumab (a fully human monoclonal antibody [mAb] inhibitor of the insulin-like growth factor-1 receptor [IGF-1R]) administered once every 3…
- Trial name
- OPTIC (NCT03298867)
- Testing
- Teprotumumab
- Where
- 14 sites in United States, Germany and 1 more
- Volunteers
- 83 people
- Sponsor
- Amgen
Non-infectious Uveitis
Safety and Efficacy of AIN457 in Noninfectious Uveitis
This study was performed to evaluate the efficacy and safety of AIN457 for patients with active uveitis that requires systemic immunosuppression.
- Trial name
- NCT00685399
- Testing
- AIN457, AIN 457
- Where
- 23 sites in United States, Germany
- Volunteers
- 76 people
- Sponsor
- Novartis Pharmaceuticals
Dry Age-related Macular Degeneration, Macular Degeneration
FOCUS: A Phase I/II First in Human Study to Evaluate the Safety and Efficacy of GT005 Administered in Subjects With Dry AMD
This was an open label first in human Phase I/II multicentre study of GT005 in subjects with Macular Atrophy due to Age-related macular degeneration (AMD).
- Conditions
- Dry Age-related Macular Degeneration, Macular Degeneration, Retinal Disease, Eye Diseases
- Trial name
- NCT03846193
- Testing
- GT005, GT005 / Device: Orbit™ Subretinal Delivery System
- Where
- 13 sites in United States, United Kingdom
- Volunteers
- 56 people
- Sponsor
- Gyroscope Therapeutics Limited
Fuchs Endothelial Corneal Dystrophy
A Multicenter Study Assessing the Efficacy and Safety of STN1010904 Ophthalmic Suspension 0.03% and 0.1% Compared With Vehicle in Subjects With Fuchs Endothelial Corneal Dystrophy (FECD)
This is a Phase IIa study to assess efficacy and safety of STN1010904 ophthalmic suspension (0.03%, and 0.1 %), twice daily dosing when compared to Placebo in subjects diagnosed with Fuchs Endothelial Corneal Dystrophy (FECD).
- Trial name
- PHANTOM (NCT05376176)
- Testing
- STN1010904 ophthalmic suspension 0.03% BID, STN1010904 ophthalmic suspension 0.1% BID
- Where
- 15 sites in United States, France and 1 more
- Volunteers
- 86 people
- Sponsor
- Santen Inc.
Age-Related Macular Degeneration
Study of Safety and Efficacy of Brolucizumab 6 mg Dosed Every 4 Weeks Compared to Aflibercept 2 mg Dosed Every 4 Weeks in Patients With Retinal Fluid Despite Frequent Anti-VEGF Injections
This clinical study was designed to compare the safety and efficacy of brolucizumab 6 mg dosed every 4 weeks to aflibercept 2 mg dosed every 4 weeks in those neovascular age-related macular degeneration (nAMD) patients with…
- Trial name
- MERLIN (NCT03710564)
- Testing
- Brolucizumab, Aflibercept
- Where
- 61 sites in United States, Puerto Rico
- Volunteers
- 535 people
- Sponsor
- Novartis Pharmaceuticals
Corneal Endothelial Dystrophy, Fuchs Endothelial Corneal Dystrophy
A Phase 1/ Phase 2 Study of TTHX1114(NM141)
Prospective, multicenter, randomized, masked, vehicle-controlled, dose-escalation study
- Conditions
- Corneal Endothelial Dystrophy, Fuchs Endothelial Corneal Dystrophy, Pseudophakic Bullous Keratopathy, Endothelial Dysfunction
- Trial name
- INTREPID (NCT04520321)
- Testing
- TTHX1114(NM141)
- Where
- 8 sites in United States
- Volunteers
- 22 people
- Sponsor
- Trefoil Therapeutics, Inc.
Age-Related Macular Degeneration (AMD), Macular Degeneration
X-82 to Treat Age-related Macular Degeneration
The purpose of this study is to evaluate the safety and efficacy of X-82 in the treatment of vision loss due to wet AMD.
- Conditions
- Age-Related Macular Degeneration (AMD), Macular Degeneration, Exudative Age-related Macular Degeneration, AMD
- Trial name
- NCT02348359
- Testing
- X-82, Anti-VEGF
- Where
- 35 sites in United States
- Volunteers
- 157 people
- Sponsor
- Tyrogenex