Clinical trials: Inherited Ophthalmic Diseases
Leber's Congenital Amaurosis
Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.
- Trial name
- NCT03140969
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 11 people
- Sponsor
- Laboratoires Thea
Gout, Rheumatoid Arthritis
Assessment of Physician Consideration of EPRO's, from Patients with Gout, Rheumatoid Arthritis, Sjogren's Syndrome or Systemic Lupus, on the Frequency of Therapeutic Adjustments
Inflammatory rheumatic diseases affect 1% of the population. Treatment of such diseases should be based on disease activity, safety issues and other patient characteristics such as comorbidities (EULAR, 2022), leading to a higher…
- Conditions
- Gout, Rheumatoid Arthritis, Sjogren's Syndrome, Systemic Lupus Erythematosus
- Trial name
- CAPTAIN (NCT05927688)
- Testing
- Therapeutic management following access by the physician to his patient's ePROs
- Where
- France
- Volunteers
- 352 people
- Sponsor
- University Hospital, Strasbourg, France
Thyroid Associated Ophthalmopathy
Establishment of Multiparametric Prediction Models for Moderate to SevereThyroid Associated Ophthalmopathy
Thyroid-associated ophthalmopathy (TAO) is an organ-specific autoimmune disease closely related to thyroid disease, which leads the incidence of orbital disease in adults and is the most common cause of diffuse toxic goiter…
- Trial name
- NCT06590220
- Where
- China
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Shanghai Changzheng Hospital
Fuchs Endothelial Corneal Dystrophy
Targeting Reactive Oxygen Species Production as a Novel Therapeutic in Fuch's Endothelial Corneal Dystrophy
This protocol will investigate whether topical application of N-acetyl cysteine (NAC) eye drops decreases oxidative stress and confers cytoprotection in patients with FECD.
- Trial name
- NCT04440280
- Testing
- N-acetyl cysteine (NAC) 10% solution, N-acetyl cysteine (NAC) 20% solution, Visine Dry Eye Relief eye drops
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
Graves Ophthalmopathy
T and B Cells in Graves' Orbitopathy
Graves orbitopathy (GO) is an inflammatory eye disease associated in 95% of patients with Graves' hyperthyroidism (GH), in ~3-4% with hypothyroid autoimmune thyroiditis, and in ~1-2% with thyroid autoimmunity in the absence of…
- Trial name
- LYMPHGO (NCT03195296)
- Testing
- No intervention
- Volunteers
- 20 people
- Sponsor
- University of Pisa
Clinical and Nutritional Safety.
Oat Products in the Treatment of Coeliac Disease in Children
In many Northern European countries oat-based products have been used in the dieto-therapy of coeliac disease for many years. The purpose of this study is to evaluate clinical tolerance and liking of gluten-free products…
- Trial name
- NCT00808301
- Testing
- Gluten-free products
- Where
- 7 sites in Italy
- Volunteers
- 300 people
- Sponsor
- Heinz Italia SpA
Intellectual Disability, Rubinstein-Taybi Syndrome
Functional Tests to Resolve Unsolved Rare Diseases. Rares.
Rares diseases are a heterogeneous group of conditions which need important tools for diagnosis. The use of high-throughput sequencing is able to diagnose half of the patients.
- Conditions
- Intellectual Disability, Rubinstein-Taybi Syndrome, Cystic Fibrosis, Congenital Heart Defect
- Trial name
- RID (NCT05696912)
- Testing
- Ex-vivo approach concerning 25 patients, In-vitro approach concerning 25 patients
- Where
- France
- Volunteers
- 50 people
- Sponsor
- University Hospital, Bordeaux
Retinopathy of Prematurity
umBilical Or Adult Donor Red Blood Cells in Extremely Low Gestational Age Neonates and Retinopathy of Prematurity (BORN)
Extremely low gestational age neonates (ELGAN, i.e., born before 28 gestation weeks) are among the most heavily transfused pediatric patients.
- Trial name
- BORN (NCT05100212)
- Testing
- cord blood-RBC concentrates, adult donor RBC concentrates
- Where
- 10 sites in Italy
- Volunteers
- 146 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Cytomegalovirus Retinitis, HIV Infections
A Randomized Controlled Study of the Efficacy and Safety of Maintenance Treatment With Oral Ganciclovir for Newly Diagnosed Cytomegalovirus Retinitis in People With AIDS
To compare the time to progression of CMV retinitis between oral ganciclovir and IV ganciclovir during 20 weeks of maintenance treatment.
- Trial name
- NCT00002257
- Testing
- Ganciclovir
- Where
- 13 sites in United States, Canada
- Volunteers
- 150 people
- Sponsor
- Hoffmann-La Roche
Stargardt Disease
Safety and Efficacy of Emixustat in Stargardt Disease
The purpose of this study is to determine if emixustat hydrochloride reduces the rate of progression of macular atrophy compared to placebo in subjects with Stargardt disease. Funding Source -- FDA OOPD
- Trial name
- SeaSTAR (NCT03772665)
- Testing
- Emixustat
- Where
- 29 sites in United States, Brazil and 9 more
- Volunteers
- 194 people
- Sponsor
- Kubota Vision Inc.
Behcet's Disease Uveitis
Efficacy and Safety Study of Gevokizumab to Treat Behcet's Disease Uveitis
The objective of this study is to assess the efficacy and safety of gevokizumab in treating Behcet's disease uveitis (BDU).
- Trial name
- EYEGUARD™-US (NCT02258867)
- Testing
- Gevokizumab
- Where
- 8 sites in United States
- Volunteers
- 4 people
- Sponsor
- XOMA (US) LLC
Retinopathy of Prematurity
Iron and Retinopathy of Prematurity (ROP)
The purpose of this study is to determine whether increased transferrin saturation in plasma (that reflects iron overload and/or low transferrin) is an independent risk factor for ROP development and severity.
- Trial name
- Fer-ROP (NCT05133999)
- Testing
- Plasma determination of iron, transferrin and ferritin, Fundus Examination by wide field digital imaging camera (PanocamTM camera)
- Where
- 3 sites in France
- Volunteers
- 175 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Cytomegalovirus Retinitis, HIV Infections
A Treatment Protocol for the Use of Intravenous Ganciclovir in AIDS Patients With Immediately Sight-Threatening CMV Retinitis
To determine the safety and effectiveness of intravenous ganciclovir (also known as DHPG) in the treatment of sight-threatening cytomegalovirus (CMV) retinitis in patients with AIDS.
- Trial name
- NCT00000698
- Testing
- Ganciclovir
- Where
- United States
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Retinopathy of Prematurity
Heredity of Retinal Vessel Tortuosity
The purpose of this study is to asses the relative influence of genetic and environmental influence on the tortuosity of retinal arterioles in eyes of 218 healthy Danish twins aged 20-46 years.
- Trial name
- NCT00427063
- Where
- Denmark
- Volunteers
- 218 people, healthy volunteers welcome
- Sponsor
- Glostrup University Hospital, Copenhagen
Macular Degeneration, Age-Related Maculopathies
Safety and Tolerability Study of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AMD)
This Phase 1 clinical research study will examine the safety and tolerability of an experimental gene transfer agent, AAV2-sFLT01, in patients with Neovascular Age-Related Macular Degeneration (AMD).
- Conditions
- Macular Degeneration, Age-Related Maculopathies, Age-Related Maculopathy, Maculopathies, Age-Related
- Trial name
- NCT01024998
- Testing
- AAV2-sFLT01
- Where
- 4 sites in United States
- Volunteers
- 19 people
- Sponsor
- Genzyme, a Sanofi Company
LGMD1B, LGMD1C
Long-Term Development of Muscular Dystrophy Outcome Assessments
This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD).
- Conditions
- LGMD1B, LGMD1C, LGMD1D, LGMD1E
- Trial name
- GRASP-01-005 (NCT05989620)
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Virginia Commonwealth University
Retinoblastoma
PCI Imaging System in Pediatric Ophthalmology
The purpose of this study is to compare the RetCam (Clarity Medical Systems, Pleasanton, CA) to a new prototype pediatric imaging system, COSMOS, produced by Phoenix Clinical Incorporated (PCI) (Pleasanton, CA).
- Trial name
- NCT02617862
- Testing
- Examination under anaesthetic (EUA), RetCam, COSMOS
- Where
- Canada
- Volunteers
- 6 people
- Sponsor
- Brenda Gallie
Retinoblastoma, Pineoblastoma
Morphological Analysis of the Pineal Gland in Pediatric Retinoblastoma Patients Using Magnetic Resonance Imaging
MRI is useful for diagnosing pinealoblastoma in retinoblastoma patients
- Trial name
- NCT01884194
- Where
- Germany
- Volunteers
- 80 people
- Sponsor
- Charite University, Berlin, Germany
Retinopathy of Prematurity
Imaging Retinal Vasculature in Infant Eyes
Retinopathy of prematurity is a leading cause of childhood blindness worldwide. The fovea, a critical location in the retina determining visual acuity and visual function, and the blood vessels around it, are abnormally developed…
- Trial name
- NCT05558059
- Testing
- Handheld Optical Coherence Tomography with OCT Angiography
- Where
- 2 sites in United States
- Volunteers
- 16 people
- Sponsor
- Duke University
Thyroid Ophthalmopathy
A Study to Evaluate the Efficacy and Safety of HBM9161 on Moderate to Severe Thyroid Eye Disease
Primary Objective: To preliminarily evaluate the efficacy of the treatment regimen of HBM9161 680 mg administered subcutaneously weekly and sequentially every other week for 12 weeks in Chinese patients with active moderate to…
- Trial name
- NCT05015127
- Testing
- HBM9161 680 mg qw by q2w from week 13, HBM9161 680 mg qw by q2w from week 7
- Where
- China
- Volunteers
- 36 people
- Sponsor
- Harbour BioMed (Guangzhou) Co. Ltd.
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