Clinical trials: Inherited Retinal Disease
Retinopathy of Prematurity
Real World Study of the Effectiveness and Safety of Conbercept Ophthalmic Injection in the Treatment of Retinopathy of Prematurity - Multicenter, Retrospective and Observational Study Based on Real World Data
This study is a retrospective, multi-center real world study. The real world data comes from the electronic medical record system and disease database of the research centers .The patient's demographic information, disease…
- Trial name
- NCT04985448
- Where
- 2 sites in China
- Volunteers
- 1,000 people
- Sponsor
- Chengdu Kanghong Biotech Co., Ltd.
Usher Syndromes
Natural History Study of Usher Syndrome ( Light4Deaf )
Clinical centres in the LIGHT4DEAF consortium have developed and will continue to improve a reliable, early molecular diagnosis and protocols for full clinical characterisation of Usher syndrome, which will be valuable for the…
- Trial name
- Light4Deaf (NCT04665726)
- Where
- 4 sites in France
- Volunteers
- 400 people
- Sponsor
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Retinopathy of Prematurity (ROP), Premature Birth
Long-term Ophthalmic Outcomes in Ex-premature Infants
There is limited knowledge on ophthalmological outcomes in the adult population that was born prematurely. This study aims to evaluate the ophthalmic outcomes of ex-premature infants that have reached adolescence or adulthood.
- Trial name
- LTOO-XP (NCT06649513)
- Testing
- eye drops (Tropicamid)
- Where
- Netherlands
- Volunteers
- 210 people
- Sponsor
- Erasmus Medical Center
Cytomegalovirus Retinitis, HIV Infections
An Open Label Evaluation of the Safety and Pharmacokinetics of Ganciclovir in Children
To evaluate the pharmacokinetics of intravenous ganciclovir in children (ages 3 months - 12 years). To determine the safety and tolerance of a 2 to 3 week induction course of ganciclovir IV in immunocompromised children receiving…
- Trial name
- NCT00002015
- Testing
- Ganciclovir
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- Roche Global Development
Retinoblastoma
Carboplatin Periocular Injection for Retinoblastoma
This study will evaluate the clinical efficacy of periocular injections of carboplatin together with chemotherapy in the treatment of Retinoblastoma as compared to chemotherapy alone.
- Trial name
- NCT02137928
- Testing
- carboplatin periocular injection, chemotherapy
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Sun Yat-sen University
Retinoblastoma
Nitroglycerin for Intra-arterial Chemotherapy in Pediatric Retinoblastoma.
The primary objective of the study is to evaluate the effect of intravenously infused nitroglycerin on the incidence of the cardio-respiratory side effects during the intra-arterial chemotherapy for retinoblastoma in pediatric…
- Trial name
- NCT04564521
- Testing
- Nitroglycerin, Normal saline
- Where
- South Korea
- Volunteers
- 36 people
- Sponsor
- Seoul National University Hospital
Stargardt Disease 1
Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease
The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1
- Trial name
- DRAGON (NCT05244304)
- Testing
- Tinlarebant
- Where
- 19 sites in United States, Australia and 9 more
- Volunteers
- 104 people
- Sponsor
- Belite Bio, Inc
Bronchopulmonary Dysplasia, Chronic Lung Disease of Prematurity
A Clinical Efficacy and Safety Study of OHB-607 in Preventing Bronchopulmonary Dysplasia in Extremely Premature Infants
The purpose of this study is to determine if an investigational drug can prevent Bronchopulmonary Dysplasia, reducing the burden of chronic lung disease in extremely premature infants, as compared to extremely premature infants…
- Conditions
- Bronchopulmonary Dysplasia, Chronic Lung Disease of Prematurity, Intraventricular Hemorrhage, Retinopathy of Prematurity (ROP)
- Trial name
- NCT03253263
- Testing
- OHB-607
- Where
- 62 sites in United States, Australia and 11 more
- Volunteers
- 295 people
- Sponsor
- OHB Neonatology Ltd.
Leber Congenital Amaurosis 10, Blindness
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A>G…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders
- Trial name
- HYPERION (NCT06891443)
- Testing
- sepofarsen
- Where
- 18 sites in United States, Australia and 8 more
- Volunteers
- 32 people
- Sponsor
- Laboratoires Thea
Retinopathy of Prematurity (ROP)
Aflibercept for Retinopathy of Prematurity - Intravitreal Injection Versus Laser Therapy
The purpose of this study is to demonstrate how well aflibercept works in babies with ROP, comparing it with laser therapy. The study also has the objective to demonstrate how safe aflibercept is when used in babies, and describe…
- Trial name
- FIREFLEYE (NCT04004208)
- Testing
- Eylea (Aflibercept, BAY86-5321), Laser photocoagulation
- Where
- 90 sites in Argentina, Austria and 25 more
- Volunteers
- 113 people
- Sponsor
- Bayer
Retinal Dystrophies, Color Vision Defects
Natural History Study of Inherited Retinal Diseases
This prospective, observational investigation seeks to delineate the interplay between chromatic vision deficits and both functional visual outcomes and anatomical retinal biomarkers in individuals affected by Inherited Retinal…
- Conditions
- Retinal Dystrophies, Color Vision Defects, Vision Disorders, Macular Degeneration
- Trial name
- MojiLVCDT (NCT07085533)
- Where
- China
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Augelux Therapeutics
Retinopathy of Prematurity, Pain Management
Pain Management During Screening for Retinopathy of Prematurity
Abstract Background: Screening examinations for retinopathy of prematurity (ROP) is critical to reduce ROP-related vision loss, however, the procedure is painful and uncomfortable, and topical anesthetics do not completely…
- Trial name
- NCT04767178
- Testing
- Ibuprofen (Sanofi, Istanbul, Turkey), Paracetamol (GlaxoSmithKline, Istanbul, Turkey)
- Where
- Turkey (Türkiye)
- Volunteers
- 44 people
- Sponsor
- Istanbul Medeniyet University
Retinopathy of Prematurity
Refractive Error and Biometry in Retinopathy of Prematurity
The investigators compared long-term refractive and biometric outcomes in children with retinopathy of prematurity who received two different anti-vascular endothelial growth factor agents.
- Trial name
- NCT03334513
- Testing
- Bevacizumab, Ranibizumab
- Volunteers
- 35 people
- Sponsor
- San Ni Chen
Ewing's Family Tumors, Renal Tumors
Auto Transplant for High Risk or Relapsed Solid or CNS Tumors
This is a standard of care treatment guideline for high risk or relapsed solid tumors or CNS tumors consisting of a busulfan, melphalan, thiotepa conditioning (for solid tumors) or carboplatin and thiotepa conditioning (for CNS…
- Conditions
- Ewing's Family Tumors, Renal Tumors, Hepatoblastoma, Rhabdomyosarcoma
- Trial name
- NCT01505569
- Testing
- Ifosfamide, Etoposide, Mesna
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Retinoblastoma, Chemotherapy Effect
Topotecan and Melphalan for Retinoblastoma
The purpose of this study is to evaluate topotecan and melphalan for retinoblastoma patients.
- Trial name
- NCT04799002
- Testing
- Topotecan, Melphalan
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Sun Yat-sen University
Von Hippel Lindau, Von Hippel Lindau Disease
Real-World Effectiveness and Pharmacogenetics of Belzutifan in VHL Syndrome: The BELIEVE-VHL Trial
The BELIEVE-VHL Trial is a prospective real-life study designed to evaluate the therapeutic effects, benefits, and adverse effects of belzutifan, as well as the timing of treatment response and disease progression in patients…
- Conditions
- Von Hippel Lindau, Von Hippel Lindau Disease, Von Hippel Lindau-Deficient Clear Cell Renal Cell Carcinoma, Hemangioblastoma (HB) of the Central Nervous System (CNS)
- Trial name
- BELIEVE-VHL (NCT07167329)
- Testing
- Belzutifan
- Where
- Brazil
- Volunteers
- 100 people
- Sponsor
- José Claudio Casali da Rocha
Early-Onset Sepses, Neonatal, Periventricular Haemorrhage Neonatal
Zinc Supplementation on Very Low Birth Weight Infant
Premature birth is a major cause of neonatal death in addition to neonatal asphyxia and infections. Early in life, premature babies must get aggressive nutrition so that there is no extrauterine growth restriction (EUGR) in the…
- Conditions
- Early-Onset Sepses, Neonatal, Periventricular Haemorrhage Neonatal, Bronchopulmonary Dysplasia, Retinopathy of Prematurity
- Trial name
- NCT04050488
- Testing
- Zinc Sulfate
- Volunteers
- 364 people, healthy volunteers welcome
- Sponsor
- Fakultas Kedokteran Universitas Indonesia
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
The objective of this study is to collect long-term safety information (i.e., for 5 years after treatment) associated with voretigene neparvovec-rzyl (vector and/or transgene), its subretinal injection procedure, the concomitant…
- Trial name
- NCT03597399
- Testing
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Where
- 10 sites in United States
- Volunteers
- 87 people
- Sponsor
- Spark Therapeutics, Inc.
Retinopathy of Prematurity (ROP)
To Assess the Effect of Dexamethasone Eye Drops on the Retinopathy of Prematurity Outcome
The purpose is to compare the treatment frequency of preterm infants who developed severe Retinopathy of Prematurity (ROP) during the years 2015-2018 with 2020-2021 in the country, using the statistical method 'difference in…
- Trial name
- NCT06775353
- Testing
- Dexamethasone eyedrop
- Where
- Sweden
- Volunteers
- 2,017 people, healthy volunteers welcome
- Sponsor
- Region Skane
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