Clinical trials by disease

Choroideremia

REP1 Gene Replacement Therapy for Choroideremia 212.26 Ko 07/24/2020
STAR 233.50 Ko 11/12/2018
A Multicenter Prospective Observational « Natural History » Study in Patients With Choroideremia 201.42 Ko 07/16/2020
Gemini 203.60 Ko 07/16/2020
A Randomised, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial Of Retinal Gene Therapy For Choroideremia Using An Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1) 216.10 Ko 07/16/2020
SOLSTICE 209.87 Ko 05/16/2019
Night 216.60 Ko 07/16/2020
CIL 34546-NSR-REP-01 212.57 Ko 05/16/2019
THOR (Choroideremia (phenotype + approved molecular diagnosis)) 221.28 Ko 06/21/2018

Retinitis Pigmentosa

XIRIUS (X-linked RP) 211.63 Ko 05/16/2019
Rate of Progression in USH2A Related Retinal Degeneration (RUSH2A) (Retinitis pigmentosa, Usher Syndrome) 231.16 Ko 05/16/2019
Clinical implication of Retinitis Pigmentosa molecular diagnostic using high throughput sequencing (RP-SEQ-HD) 227.18 Ko 06/13/2018
Therapeutic management of patients with cystoid macular edema secondary (retinitis pigmentosa) 252.80 Ko 05/16/2018
Argus® II Retinal Stimulation System Feasibility Protocol (retinotosis pigmentosa) 2 212.46 Ko 07/09/2020
Argus® II Retinal Prosthesis System: Post-Market Study 200.83 Ko 07/10/2020
Study of the Efficacy and Safety of QLT091001 in Subjects with Inherited Retinal Disease (IRD) Caused by Mutation in Retinal Pigment Epithelium Protein 65 (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) (Retinitis pigmentosa; Inherited Retinal Disease 222.87 Ko 05/16/2018
Retinitis pigmentosa: molecular diagnosis by next generation sequencing (inherited retinal dystrophy) 251.00 Ko 05/16/2018
Study of UshStat in Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B (Retinitis Pigmentosa Associated With Usher Syndrome Type 1B) 201.27 Ko 07/10/2020
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT 215.14 Ko 09/17/2020
Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65) 201.73 Ko 07/09/2020
Clinical Characterization on PDE6A-related Retinitis Pigmentosa in Preparation to a Gene Therapy Trial 201.03 Ko 07/16/2020
PIGMENT – PDE6A gene therapy for retinitis pigmentosa 200.91 Ko 07/20/2020
TES for the Treatment of Retinitis Pigmentosa (RP) 209.43 Ko 07/20/2020
Rate of Progression in EYS Related Retinal Degeneration (Pro-EYS) 214.02 Ko 06/29/2020
Natural history of the progression of X-linked retinitis pigmentosa 514.39 Ko 07/09/2020
Transcorneal Electrostimulation for Therapy of Retinitis Pigmentosa 199.06 Ko 07/16/2020
Transcorneal Electrical Stimulation - Multicenter Safety Study 202.87 Ko 07/16/2020
Safety and Efficacy of the Alpha AMS Subretinal Implant 199.51 Ko 07/24/2020
Safety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients With Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression 208.42 Ko 05/16/2019
Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01) 214.50 Ko 09/17/2020
VITAL (USHER syndrome induced by MYO7A mutation and retinotosis pigmentosa) 239.78 Ko 07/05/2018
Retrospective Natural History Study of Retinitis Pigmentosa Rare Eye 198.92 Ko 07/16/2020
Prospective Natural History Study of Retinitis Pigmentosa (PHENOROD2 214.81 Ko 06/29/2020
Autosomal Dominant Retinitis Pigmentosa: Prevalence of Known Genes Identification of New Loci / Genes 212.88 Ko 07/10/2020
Validation of standardized test protocols to assess the impact of visual pathologies in daily life activities 212.48 Ko 06/29/2020
A Post-Authorization, Multicenter, Multinational, Longitudinal,Observational Safety Registry Study for Patients Treated withVoretigene Neparvovec 199.94 Ko 06/29/2020
Design of a mobility protocol allowing to assess the impact of visual pathologies in activities of daily living for subjects with advanced visual deficits 221.12 Ko 06/29/2020
Safety and Efficacy of Subretinal Implants for Partial Restoration of Visionin Blind Patients 206.70 Ko 07/16/2020
Retina Implant Pilot Trial to Evaluate Safety & Efficacy in Blind Patients Having Degenerated Photo-receptors 205.83 Ko 07/16/2020
A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP) 208.35 Ko 07/20/2020
Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation 201.22 Ko 07/16/2020
Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa (PIONEER) 213.42 Ko 05/23/2019
Study to Evaluate Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa Due to Mutations in Exon 13 of the USH2A Gene (Stellar) 211.63 Ko 05/23/2019
Visual and Functional Assessment in Low Vision Patients 209.54 Ko 07/24/2020

Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) Historical Case Record Survey 201.15 Ko 07/10/2020
Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy 199.37 Ko 07/10/2020
Historical Case Record Survey of Visual Acuity Data From Patients With Leber’s Hereditary Optic Neuropathy (LHON) 2 229.60 Ko 07/16/2020
A Phase I, Randomized, Double Blind, Placebo-controlled, Dose-escalating Clinical Trial With KH176 200.82 Ko 07/09/2020
Observational Registry Study of Leber Hereditary Optic Neuropathy (LHON) Affected Patients 212.60 Ko 05/23/2019
Non-interventional Study of Clinical Experience in Patients Prescribed Raxone® for the Treatment of Leber's Hereditary Optic Neuropathy (LHON) (Leber's Hereditary Optic Neuropathy) 224.43 Ko 05/16/2018
REVERSE 212.38 Ko 05/16/2019
Efficacy & Safety Study of Bilateral Intravitreal Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year (REFLECT) 213.62 Ko 05/23/2019
An open label dose escalation clinical trial to evaluate the safety and the tolerability of gs010 (rAAV2/2-ND4) in patients with leber hereditary optic neuropathy due to mutations in the mitochondrial nadh dehydrogenase 4 gene 201.31 Ko 07/10/2020
Efficacy & Safety Study of Bilateral Intravitreal Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year (REFLECT) 204.57 Ko 07/10/2020
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation 202.35 Ko 09/02/2020
RESCUE AND REVERSE 210.93 Ko 09/02/2020

Leber Congenital Amaurosis

Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT 215.14 Ko 07/09/2020
Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 199.83 Ko 07/10/2020
Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01) 214.50 Ko 07/09/2020
Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis 206.96 Ko 07/20/2020
Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families 199.56 Ko 07/10/2020
Extension Study to Study PQ-110-001 (NCT03140969) 200.98 Ko 07/16/2020
Efficacy and safety of QLT091001 in subjects with Inherited Retinal Disease (IRD) caused by gene mutations 233.93 Ko 05/16/2019
RET-IRD-04 233.93 Ko 05/16/2018
Genetic Study of Patients Suffering From Congenital Amaurosis of Leber or From an Early Severe Retinal Dystrophy 200.64 Ko 09/17/2020
A double-masked, randomized, controlled, multiple-dose study to evaluate theefficacy, safety, tolerability and systemic exposure of QR-110 in Subjects withLeber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene (Il 218.39 Ko 07/09/2020
Study to Evaluate QR-110 in Subjects With Leber’s Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene 201.73 Ko 07/09/2020
Genetic Study of Patients Suffering From Congenital Amaurosis of Leber or From an Early Severe Retinal Dystrophy 200.64 Ko 07/24/2020

Stargardt Disease

Hereditary retinal disease

Impact on Daily Life of Patients Using the Subretinal Implant RETINA IMPLANT Alpha AMS 199.88 Ko 07/16/2020
Cornea Ectasia Excimer Laser Treatment 196.56 Ko 07/20/2020
CURETINA personalized medicine in hereditary retinal degenerations 209.77 Ko 07/16/2020
Clinical and Molecular Studies in Families With Inherited Eye Disease 197.07 Ko 07/20/2020
ReCOVER (This trial does use Clemastine fumarat as a remyelinating agent targeting the muscarinic receptor on oligodendrocytes around the optic nerve) 210.55 Ko 05/16/2019
Prospective Analysis of «Genotype-phenotype» Correlations Observed in a Large Cohort of Patients With Hereditary Retinal Dystrophies - GEPHIRD 210.42 Ko 07/09/2020
Phase I Trial of Gene Vector to Patients with Retinal Disease due to RPE65 mutations 199.70 Ko 07/09/2020
Argus II retinal prosthesis system post- market surveillance study protocol (inherited retinal dystrophy & choroidal dystrophy) - Copie 260.69 Ko 07/24/2020
Nanometer-controlled cut-off filters in real-life self-reported quality of vision of patients with retinitis pigmentosa (inherited retinal dystrophy) 251.76 Ko 05/16/2018

Neurotrophic Keratitis

Evaluation of safety and efficacy of r-hNGF (Neurotrophic Keratitis) 211.29 Ko 11/28/2018

CNGA3-linked achromatopsia

CNGA3 (CNGA3-linked achromatopsia) 2 228.44 Ko 05/16/2019
Safety and efficacy of a single subretinal injection of rAAV.hCNGA3 in patients with CNGA3-linked achromatopsia investigated in an exploratory, dose-escalation trial 223.28 Ko 07/16/2020

Argus II

Argus® II Retinal Stimulation System Feasibility Protocol (retinotosis pigmentosa) 2 - Copie 212.46 Ko 07/24/2020
Argus® II Retinal Prosthesis System: Post-Market Study - Copie 200.83 Ko 07/24/2020
Self-confidence Study in Patients With Argus II Artificial Retina Rare Eye 198.42 Ko 07/10/2020
Argus II retinal prosthesis system post- market surveillance study protocol (inherited retinal dystrophy & choroidal dystrophy) 260.69 Ko 07/24/2020

Glaucoma

Long-Term Non-Interventional Latanoprost Study 270.68 Ko 07/05/2018

Usher Syndrome

Light4deaf study: study of the natural history of the syndrome of usher in a cohort of patients followed longitudinally for 5 years (Usher Syndrome) 224.37 Ko 05/16/2018
Rush2A 249.81 Ko 11/08/2018
Natural History Study in Subjects With Usher Syndrome 2 195.96 Ko 07/20/2020
Clinical and Genetic Examination of Usher Syndrome Patients’ Cohort in Europe 200.22 Ko 07/10/2020
Open-Label Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat® in Patients With Usher Syndrome Type 1B (Usher Syndrome) 225.23 Ko 05/16/2018
VITAL (USHER syndrome induced by MYO7A mutation and retinotosis pigmentosa) - Copie 239.78 Ko 07/24/2020
Study to Evaluate Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa Due to Mutations in Exon 13 of the USH2A Gene (Stellar) - Copie 211.63 Ko 07/24/2020

Bardet-Biedl

Bardet-Biedl Syndrome Study: Clinical and Genetic Epidemiology Study in Adults 201.36 Ko 07/10/2020
Treatment of Bardet-Biedl-Syndrome With Metformin for Evaluation of a Possible Visual Improvement 202.18 Ko 07/16/2020
Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity 204.17 Ko 06/29/2020

Alström Syndrome

Clinical Study of a single ciliopathy: Alström Syndrome 2 210.92 Ko 07/10/2020
Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity 204.17 Ko 07/24/2020

Chorioretinopathy

SPECTRA trial (central serous chorioretinopathy) 2 207.19 Ko 07/20/2020

Keratoconus

Measurement of High Order Aberrations in Late Stages Keratoconus 199.80 Ko 07/16/2020
Iontophoretic Transepithelial Corneal Cross-linking in Pediatric Patients 196.90 Ko 07/20/2020
Transepithelial Corneal Collagen Crosslinking in Eyes With Progressive Keratoconus 199.47 Ko 07/16/2020
Iontophoretic Transepithelial Collagen Cross-linking Versus Epithelium-off Collagen Cross-linking in Pediatric Patients. Three Year Follow up. 197.63 Ko 07/20/2020

Congenital cataract registry

Longitudinal observation of factors affecting sight development in congenital cataract patients, long term observation of IOL implantation outcomes 226.24 Ko 07/10/2020

Ichthyosis

Prevalence of Ophthalmological Abnormalities in Children and Adults suffering from Hereditary Ichthyosis 201.20 Ko 07/02/2020

Trisomy 21

Trisomy 21 in Adulthood : a medical and social situation in Alsace 227.20 Ko 06/13/2018

Other

High resolution and high speed Multimodal Ophthalmic Imaging (IMA-MODE) 200.21 Ko 06/29/2020
High Resolution Retina Imaging (IHR) Rare Eye 198.46 Ko 06/29/2020
A Study to Determine the Safety and Efficacy of RenexusÂ® in Macular Telangiectasia Type 2 202.20 Ko 07/16/2020
Advanced Characterization of Autosomal Dominant Optic Atrophy 200.96 Ko 07/09/2020
Interest of Intravitreal Injections of Anti-VEGF as Initial and Adjuvant Treatment in Coats Disease 202.89 Ko 07/16/2020
EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment 199.09 Ko 07/20/2020

Clinical trials ongoing in ERN-EYE Members Expertise Centers

by Disease

Choroideremia

Retinitis Pigmentosa

Leber Hereditary Optic Neuropathy (LHON)

Leber Congenital Amaurosis

Stargardt Disease

Hereditary retinal disease

Neurotrophic Keratitis

CNGA3-linked achromatopsia

Argus II

Glaucoma

Usher Syndrome

Bardet-Biedl

Alström Syndrome

Chorioretinopathy

Keratoconus

Congenital cataract registry

Ichthyosis

Trisomy 21

Other

This ERN is supported by:

Clinical trials ongoing in ERN-EYE Members Expertise Centers

by Disease

Choroideremia

Retinitis Pigmentosa

Leber Hereditary Optic Neuropathy (LHON)

Leber Congenital Amaurosis

Stargardt Disease

Hereditary retinal disease

Neurotrophic Keratitis

CNGA3-linked achromatopsia

Argus II

Glaucoma

Usher Syndrome

Bardet-Biedl

Alström Syndrome

Chorioretinopathy

Keratoconus

Congenital cataract registry

Ichthyosis

Trisomy 21

Other

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