Clinical trials: Retinal Dystrophy
Spinal Cord Injury, Spinal Cord Involvement
Clinical Trial of a Serious Game for Individuals With SCI/D
This study will evaluate the efficacy of a newly developed serious game, SCI HARD, to enhance self-management skills, self-reported health behaviors, and quality of life among adolescents and young adults with spinal cord injury…
- Conditions
- Spinal Cord Injury, Spinal Cord Involvement, Spina Bifida, Transverse Myelitis
- Trial name
- NCT02341950
- Testing
- SCI Hard
- Where
- United States
- Volunteers
- 184 people
- Sponsor
- University of Michigan
Retinal Dystrophy Due to Biallelic ABCA4 Mutations, Stargardt Disease 1
Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt…
- Trial name
- NCT07002398
- Testing
- VG801
- Where
- China
- Volunteers
- 18 people
- Sponsor
- VeonGen Therapeutics GmbH
Amaurosis, Retinal Diseases
Genetic Study of Patients Suffering From Congenital Amaurosis of Leber or From an Early Severe Retinal Dystrophy
Retinal dystrophies are responsible for numerous cases of blindness, and there are no therapeutic possibilities today. Gene therapy is efficient in a dog model concerning dystrophy linked to a mutation of the rpe65 gene.
- Trial name
- NCT00422721
- Testing
- realization of a family tree, refractometry, evaluation of the presence of a nystagmus
- Where
- France
- Volunteers
- 360 people
- Sponsor
- Nantes University Hospital
Mucolipidosis Type IV
The Natural History of Mucolipidosis Type IV
The purpose of this study is to define the natural history of Mucolipidosis Type IV and identify potential clinical outcome measures.
- Trial name
- NCT01067742
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Baylor Research Institute
Leber Congenital Amaurosis 2, Retinitis Pigmentosa 20
Natural History of Patients With Inherited Retinal Diseases Due to Mutations in RPE65 Gene
Rationale: In preparation for treatment with gene therapy, this study is being conducted in order to investigate the natural history of Inherited Retinal Dystrophies (IRDs) due to mutations in RPE65 gene.
- Trial name
- RPE65-NHS (NCT04525261)
- Where
- 9 sites in Italy
- Volunteers
- 60 people
- Sponsor
- University of Campania Luigi Vanvitelli
Retinitis Pigmentosa, Maculopathy, Age Related
High Resolution Retina Imaging
To increase the clinical experience of using the rtx1 camera in various retinal disorders and to follow the evolution of structural alterations during retinal diseases using adaptive optics imaging with the rtx1 camera
- Conditions
- Retinitis Pigmentosa, Maculopathy, Age Related, Macular Dystrophy, Macular Edema
- Trial name
- IHR (NCT04128150)
- Where
- 3 sites in France
- Volunteers
- 1,000 people
- Sponsor
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Retinitis Pigmentosa, Stem Cell Transplant
Spheroidal Mesenchymal Stem Cells in Retinitis Pigmentosa
Purpose: This prospective clinical case series aimed to evaluate the effect of suprachoroidal implantation of mesenchymal stem cells (MSCs) in the form of spheroids as a stem cell therapy for retinitis pigmentosa (RP) patients…
- Trial name
- NCT05712148
- Testing
- Stem cell implantation
- Where
- Turkey (Türkiye)
- Volunteers
- 15 people
- Sponsor
- Acibadem University
LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
The purpose of this study is: To evaluate the safety of up to 3 additional courses of oral QLT091001 administered once daily for 7 days in subjects treated previously with a single 7-day course of QLT091001 in Study RET IRD 01 To…
- Trial name
- NCT01521793
- Testing
- QLT091001
- Where
- 6 sites in United States, Canada and 3 more
- Volunteers
- 27 people
- Sponsor
- QLT Inc.
Macular Oedema, Retinitis Pigmentosa
Aflibercept for Macular Oedema With Underlying Retinitis Pigmentosa (AMOUR) Study
The purpose of this study is to assess the safety and efficacy of intravitreal injections of Aflibercept (Eylea) in treating Cystoid Macula Oedema (CMO) in patients with underlying Retinitis Pigmentosa (RP).
- Trial name
- AMOUR (NCT02661711)
- Testing
- Aflibercept
- Where
- United Kingdom
- Volunteers
- 30 people
- Sponsor
- Moorfields Eye Hospital NHS Foundation Trust
Retinitis Pigmentosa (RP)
Retrospective 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
- PHENOROD1 (NCT03975543)
- Where
- France
- Volunteers
- 113 people
- Sponsor
- SparingVision
Retinitis Pigmentosa, Usher Syndrome Type 2
An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa
PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability and efficacy of QR 421a (ultevursen) administered via intravitreal (IVT) injection in one or both eyes, in subjects ≥ 12 years of age with…
- Trial name
- HELIA (NCT05085964)
- Testing
- RNA antisense oligonucleotide for intravitreal injection
- Where
- 7 sites in United States, Canada and 1 more
- Volunteers
- 21 people
- Sponsor
- Laboratoires Thea
Inherited Retinal Dystrophies
Multi-center Observation of the Natural Course of Inherited Retinal Dystrophies
Based on a clinical multi-center study of the natural course of inherited retinal dystrophies in Chinese population, screening fundus imaging indicators for patients with Chinese inherited retinal dystrophies, describing the…
- Trial name
- RID (NCT03691168)
- Where
- China
- Volunteers
- 500 people
- Sponsor
- Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Stargardt Disease
ABCA4-associated Disease in Childhood and Adolescence - a Phenotype Study
Retrospective chart review study to elucidate the phenotype and genotype of children with ABCA4-associated Stargardt disease.
- Trial name
- NCT06377150
- Where
- Germany
- Volunteers
- 39 people
- Sponsor
- University Hospital Tuebingen
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
Hereditary Retinal Dystrophies
Prospective Analysis of "Genotype-phenotype" Correlations Observed in a Large Cohort of Patients With Hereditary Retinal Dystrophies - GEPHIRD
This study will carry out a detailed descriptive analysis of a large population of patients with hereditary retinal dystrophies (HRD): clinical, paraclinical and genetic data.
- Trial name
- GEPHIRD (NCT03662386)
- Testing
- OCT-A, Visual acuity (ETDRS)
- Where
- France
- Volunteers
- 103 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Retinitis Pigmentosa
Clinical and Genetic Findings in Patients With PRPF31-associated Retinitis Pigmentosa
Retrospective chart review study to elucidate the genotype and phenotype of patients with PRPF31-associated retinitis pigmentosa and asymptomatic carriers of the respective variant(s)
- Trial name
- NCT06368375
- Where
- Germany
- Volunteers
- 87 people
- Sponsor
- University Hospital Tuebingen
Familial Mediterranean Fever (FMF), Autoinflammation
Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics
This study is designed to explore the genetics and pathophysiology of diseases presenting with intermittent fever, including familial Mediterranean fever, TRAPS, hyper-IgD syndrome, and related diseases.
- Conditions
- Familial Mediterranean Fever (FMF), Autoinflammation, Periodic Fever, Fever
- Trial name
- NCT00001373
- Where
- 5 sites in United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- National Human Genome Research Institute (NHGRI)
Biallelic RPE65 Mutation-associated Retinal Dystrophy
Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy
The purpose of this study is to provide safety and efficacy data for voretigene neparvovec, administered as subretinal injection, in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy.
- Trial name
- NCT04516369
- Testing
- voretigene neparvovec
- Where
- Japan
- Volunteers
- 4 people
- Sponsor
- Novartis Pharmaceuticals
Cone-Rod Degeneration, Rod-Cone Degeneration
Virtual Reality Mobility Assessment of Functional Vision in Retinal Disease
Background: The retina is a thin layer of tissue at the back of the eye. Retinal disease usually reduces a person s mobility because it affects how he or she moves through familiar and unfamiliar environments.
- Trial name
- NCT04289571
- Testing
- VR Mobility Tool
- Where
- 2 sites in United States, Australia
- Volunteers
- 165 people, healthy volunteers welcome
- Sponsor
- National Eye Institute (NEI)
Inherited Retinal Dystrophy Primarily Involving Sensory Retina, Inherited Retinal Dystrophy Primarily Involving Retinal Pigment Epithelium
Establishment of the National Registry for Inherited Retinal Dystrophy in Iran
Purpose: To establish of the national Inherited Retinal Dystrophy Registry (IRDR) in Iran. Methods: This study is a community-based participatory research that is approved by the Ministry of Health and Medical Education of Iran…
- Trial name
- NCT04131400
- Testing
- Visual Acuity Testing and Retinal Imaging
- Where
- Iran
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Shahid Beheshti University of Medical Sciences