Clinical trials: Inherited Ophthalmic Diseases
Eyelid Diseases, Eyelid Movement Disorders
Feasibility of Dynamic Muscle Stimulation + Radiofrequency for Improving Blink Quality in Subjects with Dry Eye Disease
To demonstrate that DMSt + RF improves eye blink quality in subjects with dry eye disease
- Conditions
- Eyelid Diseases, Eyelid Movement Disorders, Dry Eye, Dry Eye Syndromes
- Trial name
- NCT05945069
- Testing
- TriLift device
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Lumenis Be Ltd.
Retinitis Pigmentosa
Clinical Trial of Intravitreal Injection of Autologous Bone Marrow Stem Cells in Patients With Retinitis Pigmentosa
The purpose of this study is to evaluate the safety of intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa by a prospective, single-center, randomized, parallel, double-blind, phase I…
- Trial name
- TC/RP (NCT02280135)
- Testing
- Intravitreal injection of Autologous bone marrow Stem Cell, Subconjunctival injection of saline
- Where
- Spain
- Volunteers
- 8 people
- Sponsor
- Red de Terapia Celular
Leber's Hereditary Optic Neuropathy
RHODOS Follow-up Single-visit Study
This study aims to evaluate the current visual acuity of SNT-II-003 participants and compare this with the last visit from the SNT-II-003 study.
- Trial name
- RHODOS-OFU (NCT01421381)
- Where
- 3 sites in Canada, Germany and 1 more
- Volunteers
- 60 people
- Sponsor
- Santhera Pharmaceuticals
Stargardt Disease 1, STGD1
A Phase 1, First-in-Human Study of CITY-RBP4 in Healthy Volunteers and STGD1 Participants
This is a first-in-human (FIH), multicenter, randomized, double-masked, placebo-controlled adaptive clinical study evaluating the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) of CITY-RBP4, following…
- Conditions
- Stargardt Disease 1, STGD1, Healthy Volunteers
- Trial name
- NCT07836452
- Testing
- CITY-RBP4
- Where
- Australia
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- City Therapeutics
Retinopathy of Prematurity (ROP)
Safety, PK and PD of FLQ-101 in Premature Neonates
The purpose of this study is to evaluate safety and efficacy outcomes following exposure to FLQ-101.
- Trial name
- tROPhy-1 (NCT07093255)
- Testing
- FLQ-101
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- FELIQS 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.
Choroideremia
Natural Disease Progression in Participants With Choroideremia
The primary objective of this study is to quantify disease progression measured by best corrected visual acuity (BCVA) in the real-world setting among Choroideremia (CHM) participants.
- Trial name
- NCT04795206
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 1,178 people
- Sponsor
- Biogen
Retinitis Pigmentosa
Prospective Natural History Study of Retinitis Pigmentosa
This is natural history study of rods and cones degenerations in patients with Retinitis Pigmentosa (RP) caused by pathogenic mutations in RHO, PDE6a or PDE6b gene mutations.
- Trial name
- PHENOROD2 (NCT04285398)
- Testing
- Ophthalmic examinations, Mobility Test
- Where
- 2 sites in United States, France
- Volunteers
- 82 people
- Sponsor
- SparingVision
Acute Noninfectious Posterior, Intermediate, or Pan Uveitis
Study of FTY720 in Patients With Uveitis
This study will assess the safety, tolerability, and efficacy of FTY720 in patients with acute, noninfectious intermediate, posterior and pan uveitis
- Trial name
- NCT01791192
- Testing
- FTY720, Oral Corticosteroid
- Sponsor
- Novartis Pharmaceuticals
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
Thyroid Eye Disease
An Efficacy, Safety, and Tolerability Study of VRDN-003 in Participants With Active Thyroid Eye Disease (TED)
This is a clinical trial assessing the efficacy, safety, and tolerability of an investigational drug, VRDN-003, in participants with active Thyroid Eye Disease (TED).
- Trial name
- REVEAL-1 (NCT06625411)
- Testing
- VRDN-003
- Where
- 48 sites in United States, Germany and 3 more
- Volunteers
- 132 people
- Sponsor
- Viridian Therapeutics, Inc.
Retinitis Pigmentosa
Phase III Efficacy and Safety Clinical Study of UF-021 for Treatment of Retinitis Pigmentosa
This study is a multicenter trial performed in Japan, consisting of a comparative study period and a continuous administration period.
- Trial name
- NCT01786395
- Testing
- UF-021
- Where
- 34 sites in Japan
- Volunteers
- 202 people
- Sponsor
- R-Tech Ueno, Ltd.
Retinitis Pigmentosa
Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)
The rod-cone dystrophies (often referred to as retinitis pigmentosa (RP)) are a clinically and genetically heterogeneous group of disorders in which there is progressive loss of rod and later cone photoreceptor function leading…
- Trial name
- NCT03349242
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 140 people
- Sponsor
- MeiraGTx UK II Ltd
Blindness, Leber Congenital Amaurosis 10
Natural History Study of CEP290-Related Retinal Degeneration
A prospective natural history study with systematic assessments and uniform follow-up to provide a high-quality dataset for assisting in the design of future clinical treatment trials involving patients with CEP290-related…
- Conditions
- Blindness, Leber Congenital Amaurosis 10, Vision Disorders, Eye Diseases
- Trial name
- NCT03396042
- Where
- 7 sites in United States, France and 2 more
- Volunteers
- 26 people
- Sponsor
- Editas Medicine, Inc.
Lysosomal Storage Disease, Lung Diseases
Biomarker for Maroteaux-Lamy Disease (BioMaroteaux)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Maroteaux-Lamy disease from blood
- Conditions
- Lysosomal Storage Disease, Lung Diseases, Obstructive Sleep Apnoea, Macroglossia
- Trial name
- BioMaroteaux (NCT01458613)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
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
Retinitis Pigmentosa, Retinitis
Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study
This study will be conducted following Good Clinical Practice (GCP) and International Conference on Harmonization (ICH) guidelines.
- Conditions
- Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases
- Trial name
- REMAIN (NCT06162585)
- Testing
- Gene Therapy product-MCO-010
- Where
- 6 sites in United States, Puerto Rico
- Volunteers
- 18 people
- Sponsor
- Nanoscope Therapeutics Inc.
Leber's Hereditary Optic Neuropathy
Study With Idebenone in Patients With Chronic Vision Loss Due to Leber's Hereditary Optic Neuropathy (LHON)
The objective of the study is to determine whether administration of idebenone can shorten the time to improvement of visual acuity in patients with chronic vision loss due to LHON.
- Trial name
- NCT01495715
- Testing
- Idebenone
- Sponsor
- Santhera Pharmaceuticals
Conjunctivitis, Atopic Dermatitis
Observational Study of Conjunctivitis in the Setting of DUPIXENT® Treatment for Atopic Dermatitis
The primary objective of the study is to characterize the clinical phenotype(s) of DUPIXENT®-associated conjunctivitis events. The secondary objectives of the study are to characterize the course of conjunctivitis events during…
- Trial name
- NCT04287608
- Testing
- dupilumab
- Where
- 15 sites in United States
- Volunteers
- 50 people
- Sponsor
- Regeneron Pharmaceuticals