Phase 2 N=6 Choroideremia Gene Therapy Clinical Trial
Choroideremia
Primary: Change in Best Corrected Visual Acuity From Baseline — 3.0 Letters
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Phase 3 N=169 Efficacy and Safety of BIIB111 for the Treatment of Choroideremia
Choroideremia
Primary: Percentage of Participants With a ≥15 -Letter Improvement From Baseline in Best Corrected Visual Acuity (BCVA) at Month 12 as Measured by the Early Treatment of Diabetic…
Phase 2 N=60 A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111
Choroideremia
Primary: Mean Best-Corrected Visual Acuity (BCVA) as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart in Letters at Month 12 — 68.0; 76.5; 82.3; 81.2…
Phase 2 N=15 Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations
Choroideremia · CHM (Choroideremia) Gene Mutations
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 5; 5; 5 Participants
Phase 1 N=7 Ranibizumab in Hemorrhagic Choroidal Neovascularization Trial
Choroidal Neovascularization
Primary: Number of Subjects Avoiding 15 or More Letter Loss of Best Corrected Visual Acuity From Baseline to 12 Months on an Early Treatment Diabetic Retinopathy Study (ETDRS)…
Phase 2 N=22 Sildenafil for Treatment of Choroidal Ischemia
Choroidal Ischemia · Vitelliform Macular Dystrophy · Age-related Macular Degeneration
Primary: Change in Visual Acuity (Sildenafil Group) — 0.00; -0.03 logMAR
Phase 2 N=23 Gene Therapy for Achromatopsia (CNGB3)
Achromatopsia
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to…
Phase 2 N=11 Gene Therapy for Achromatopsia (CNGA3)
Achromatopsia
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to…
Phase 3 N=295 Study of Brolucizumab in Adult Patients With Suboptimal Anatomically Controlled Neovascular Age-related Macular Degeneration
Neovascular Age-Related Macular Degeneration
Primary: Number of Patients With no Disease Activity at Week 16 in the Study Eye — 89 Participants
Phase 2 N=78 DHA and X-Linked Retinitis Pigmentosa
Retinitis Pigmentosa · X-linked Genetic Diseases
Primary: Rate of LOSS of 31 Hertz Cone Electroretinographic Function — 0.028; 0.022 log microvolts/year — p=0.30
Phase 2 N=15 Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
Leber Congenital Amaurosis
Primary: Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event — 0; 0; 1; 0 Participants
Phase 2 N=88 Study Evaluating Intravitreal hI-con1™ in Patients With Choroidal Neovascularization Secondary to Age-related Macular Degeneration
Choroidal Neovascularization · Age-related Macular Degeneration
Primary: Change From Baseline in Best Corrected Visual Acuity (BCVA) in the Study Eye at Month 3 — .3; 6.8; 7.6 Letters
Phase 2 N=45 Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
X-Linked Retinitis Pigmentosa
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 9 Weeks Following Administration, at Least Possibly Related to…
Phase 2 N=146 A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa
Retinitis Pigmentosa
Primary: Change in Humphrey Visual Fields - Total Sensitivity — 1.4; 16.8; -164.8; -66.7 dB
Phase 2 N=134 A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa
Retinitis Pigmentosa
Primary: The Primary Outcome is the Change in Best-corrected Visual Acuity (BCVA) Using the Electronic Visual Acuity (EVA) Technology at Month 12. — 1; 1; 3; 1 participants
Phase 2 N=27 Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)
X-linked Retinoschisis
Primary: Number of Participants Experiencing Adverse Events — 6; 7; 12 Participants
Phase 2 N=44 Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa
Retinitis Pigmentosa · Usher Syndrome Type 2 · Usher Syndrome Type 3
Primary: Mean Change in Cone Spacing in Arcminutes (Z Score) of 2 Baseline Values Were Compared With Measurements Obtained at Post-op Month 36 — 1.13; 0.85 Difference in change…
N/A N=442 Cerebrotendinous Xanthomatosis (CTX) Prevalence Study
Cerebrotendinous Xanthomatosis (CTX)
Primary: Number of Participants Who Had Positive Genetic Test Results — 4 Participants
Phase 2 N=8 A Study in Subjects With Retinitis Pigmentosa
Retinitis Pigmentosa
Primary: Number of Subjects With Serious Adverse Events — 0; 0 Participants
Phase 3 N=29 Rhopressa for Corneal Edema Associated With Fuchs Dystrophy
Fuchs Endothelial Dystrophy
Primary: Change in Central Corneal Thickness — -23; -2 microns
N/A N=5 Gene Analysis and Treatment Optimization in Chinese Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia
Primary: Number of LDLR Gene Mutations — 7 gene mutations
Phase 1 N=6 Trial of Subretinal Injection of (rAAV2-VMD2-hMERTK)
Retinal Disease · Retinitis Pigmentosa
Primary: Systemic and Ocular Safety — 0; 0; 1; 0 Participants
Phase 2 N=12 Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis
Retinoschisis · X-Linked
Primary: Adverse Events (AEs) Affecting Ocular Function That Differ Clinically From Those Expected in the Normal Course of Progression of XLRS — 3; 3; 12; 6 events
N/A N=54 Alpha MSH in Ocular Disease
Advanced Dry Macular Degeneration
Primary: Level of Alpha Melanocyte Stimulating Hormone as Measured by Enzyme-linked Immunosorbent Assay
Phase 2 N=23 Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
Stargardt Disease · Macular Atrophy
Primary: Change in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram — -12.23; 68.00; 96.69 percent suppression
Phase 2 N=139 Efficacy and Safety on SOM3355 in Huntington's Disease Chorea
Huntington Chorea
Primary: Change in Total Maximal Chorea (TMC) Score of the UHDRS® for Subjects Not Taking Neuroleptics During the Trial (mITT - N=122) — -2.19; -2.42; -3.46 units on a scale…
Phase 2 N=22 A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED)
Fuchs' Corneal Endothelial Dystrophy (FCED)
Primary: Incidence and Severity of Ocular TEAEs. — 3; 6; 1; 1 Participants
Phase 4 N=49 Indocyanine Angiographic Changes of Choroidal Neovascularization by Ranibizumab
Exudative Age-related Macular Degeneration
Primary: Caliber of Choroidal New Vessel (CNV) — 43.53 micrometer
Phase 3 N=122 VEGF Trap-Eye in Choroidal Neovascularization Secondary to Pathologic Myopia (mCNV)
Myopia, Pathological
Primary: Mean Change in Best Corrected Visual Acuity (BCVA) as Measured by Early Treatment Diabetic Retinopathy Study (ETDRS) From Baseline to Week 24 - Last Observation Carried…
Phase 2 N=57 Study in Subjects With Small Primary Choroidal Melanoma
Uveal Melanoma · Ocular Melanoma · Choroidal Melanoma
Primary: Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability] of Light-activated AU-011 — 3; 3; 3; 3 Participants