Clinical trials: Inherited Ophthalmic Diseases
Thyroid Eye Disease (TED)
Open-Label Extension Study of MHB018A in Subjects With Thyroid Eye Disease
This is a multicenter, open-label extension (OLE) study of MHB018A in subjects with moderate-to-severe TED.
- Trial name
- NCT07262476
- Testing
- MHB018A injection
- Where
- China
- Volunteers
- 258 people
- Sponsor
- Minghui Pharmaceutical (Hangzhou) Ltd
Fabry Disease
Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients.
Fabry disease is a recessively inherited disorder due to systemic storage of abnormal metabolites (ceramide trihexocide, in particular) caused by lack of the lysosomal enzyme α-galactosidase.
- Trial name
- NCT01997489
- Testing
- Enzyme replacement
- Where
- 2 sites in Denmark
- Volunteers
- 39 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Retinitis Pigmentosa, Retinal Diseases
Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa
The purpose of this study is to evaluate the efficacy and safety of oral valproic acid to slow the progression of visual function and/or to improve the visual function in patients with retinitis pigmentosa (RP).
- Conditions
- Retinitis Pigmentosa, Retinal Diseases, Eye Diseases, Eye Disease, Hereditary
- Trial name
- VPA_RP (NCT01399515)
- Testing
- Valproic Acid
- Where
- South Korea
- Volunteers
- 200 people
- Sponsor
- Seoul National University Hospital
Albinism, Oculocutaneous Albinism
Vision Response to Dopamine Replacement
The purpose of the study is to evaluate and document physiologic and functional changes in visual performance and retinal function of patients diagnosed with albinism (a dopamine deficiency state) following a trial of oral…
- Trial name
- NCT01663935
- Testing
- Levodopa/carbidopa
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Wisconsin, Madison
Wolfram Syndrome
Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1
Wolfram syndrome (WFS:OMIM 222300) is a group of inherited disorders that usually appear in childhood and cause diabetes, optic atrophy leading to loss of vision, deafness and often diabetes insipidus.
- Trial name
- NCT05659368
- Testing
- Tirzepatide
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- Ospedale San Raffaele
Fuchs Endothelial Corneal Dystrophy, Cataract (Post-operative Cataract Surgery Follow-up)
Postoperative CCT in FECD vs. Non-FECD
This single-centre, prospective, controlled, non-interventional observational clinical study aims to characterize early postoperative corneal thickness behaviour after routine phacoemulsification cataract surgery in eyes with…
- Trial name
- NCT07731945
- Volunteers
- 98 people, healthy volunteers welcome
- Sponsor
- Vienna Hospital Association
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
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
Pre-Term, Respiratory Distress Syndrome, Newborn
NeO2Matic-Pilot Trial
The main goal of this trial is to test if: automated adjustment of supplemental oxygen to preterm infants in noninvasive respiratory support based on feedback from a measurement of blood-oxygen saturation results in more stable…
- Conditions
- Pre-Term, Respiratory Distress Syndrome, Newborn, Retinopathy of Prematurity, Bronchopulmonary Dysplasia
- Trial name
- NEO2MATIC (NCT05629910)
- Testing
- O2Matic
- Where
- Denmark
- Volunteers
- 30 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Optical Coherence Tomography, Anterior Chamber Angle
Optical Coherence Tomography of the Irido-Corneal Angle Before and After Goniotomy and Trabeculotomy in Primary Congenital Glaucoma
The research aimed to improve surgical techniques and long-term outcomes by utilizing this advanced imaging technology to illuminate the structural abnormalities of the anterior segment in eyes with primary congenital glaucoma.
- Conditions
- Optical Coherence Tomography, Anterior Chamber Angle, Goniotomy, Trabeculotomy
- Trial name
- NCT07012252
- Testing
- Optical coherence tomography
- Where
- Egypt
- Volunteers
- 32 people
- Sponsor
- Cairo University
Parkinson Disease, Multiple System Atrophy
Research of Biomarkers in Parkinson Disease
The main goal of the GENEPARK consortium is to employ innovative haemogenomic approaches to determine gene expression profiles specific for genetic and idiopathic Parkinson's disease (PD) patients.
- Conditions
- Parkinson Disease, Multiple System Atrophy, Progressive Supranuclear Palsy, Huntington Disease
- Trial name
- Genepark (NCT00465790)
- Where
- 5 sites in Croatia, France and 2 more
- Volunteers
- 219 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Type 1 Diabetes, Diabetes Insipidus
Tracking Neurodegeneration in Early Wolfram Syndrome
The goal of this study is to determine the pattern of early neurodegenerative changes in WFS (Wolfram Syndrome). The investigator will perform cross-sectional and longitudinal assessments of youth with WFS, targeting sensitive…
- Conditions
- Type 1 Diabetes, Diabetes Insipidus, Diabetes Mellitus, Wolfram Syndrome
- Trial name
- TRACK (NCT02455414)
- Where
- United States
- Volunteers
- 101 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Thyroid Eye Disease, TED
Psychological Burden of Thyroid Eye Disease
This interview study aims to look in depth at the quality of life of people living with TED and their caregivers, with emphasis on impact on mood, relationships, work/education, and leisure.
- Trial name
- NCT07444190
- Testing
- focus groups and individual interviews as well as filling out questionnaires
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Thomas Jefferson University
Behcet's Syndrome, Intestinal Type
Pathogenesis of Intestinal Behcet's Syndrome
There is a direct interaction between food, intestinal flora and the intestinal barrier composed of intestinal epithelial cells and mucosal immunity, whose composition and functional alterations are theorized to be closely…
- Trial name
- NCT06026020
- Testing
- Biological samples collection
- Where
- China
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Peking Union Medical College Hospital
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Cyclosporine Eye Drops in Preventing Graft-Versus-Host Disease of the Eye in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer or Bone Marrow Failure Disorder
RATIONALE: Cyclosporine eye drops may prevent graft-versus-host disease of the eye in patients who have undergone donor stem cell transplant for hematologic cancer or bone marrow failure disorder.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00755040
- Testing
- cyclosporine ophthalmic emulsion
- Where
- 5 sites in United States
- Volunteers
- 164 people
- Sponsor
- Vanderbilt-Ingram Cancer Center
Retinitis Pigmentosa
Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients
This is zM-02's safety, tOlerability, and efficacy in retinitis pigmentOsa first-in-humaN study (MOON). This trial is meant to evaluate the safety and efficacy of ZM-02 in Retinitis pigmentosa (RP) patients.
- Trial name
- MOON (NCT06292650)
- Testing
- ZM-02-L, ZM-02-H, ZM-02-S
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Augelux Therapeutics
Vasculitis, Behcet's Disease
Vasculitis Pregnancy Registry
The purpose of this study is to learn about the experience of women with vasculitis who become pregnant. In particular, the study will consist of several online surveys to assess 1.
- Conditions
- Vasculitis, Behcet's Disease, CNS Vasculitis, Cryoglobulinemic Vasculitis
- Trial name
- V-PREG (NCT02593565)
- Testing
- Online questionnaires
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- University of Pennsylvania
Graves' Dysthyroid Ophthalmopathy, Thyroid Related Orbitopathy
Rituximab Treatment of Graves' Dysthyroid Ophthalmopathy
This study is designed to treat patients with Graves' disease with Rituximab in an attempt to prevent or reverse the physically deforming and debilitating consequences of this disease.
- Trial name
- NCT00424151
- Testing
- Rituximab
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Silkiss, Rona Z., M.D., FACS
Fuchs' Endothelial Dystrophy, Cataract
Risk Assessment for Progression to DMEK Following Cataract Surgery in Fuchs Endothelial Corneal Dystrophy
Justification. Fuchs' corneal endothelial dystrophy (FECD) is one of the main causes of corneal transplantation. In many cases, the corneal decompensation derived from this dystrophy is triggered as a consequence of cataract…
- Trial name
- NCT04072029
- Testing
- Phacoemulsification with intraocular lens implantation
- Where
- Spain
- Volunteers
- 68 people
- Sponsor
- Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal
Dry Age-related Macular Degeneration
HORIZON: 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
- NCT04566445
- Testing
- GT005
- Where
- 62 sites in United States, Australia and 5 more
- Volunteers
- 255 people
- Sponsor
- Gyroscope Therapeutics Limited
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