Clinical trials: Retinal Dystrophy
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
- Zhongmou Therapeutics
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.
Inherited Retinal Dystrophy
Fellow-eye Study (FE) of LX101 in Subjects With Inherited Retinal Dystrophy
Up to nine subjects who have participated in the earlier LX101 clinical study, and who meet all study eligibility criteria, will receive LX101 administration in the previously uninjected, contralateral eye to evaluate the safety…
- Trial name
- NCT06212297
- Testing
- LX101
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Innostellar Biotherapeutics Co.,Ltd
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
Stargardt Disease, Age-related Macular Degeneration
Phase 1 Safety Study of ALK-001 in Healthy Volunteers
This protocol is a phase 1 clinical study to assess the safety and pharmacokinetics of ALK-001 in healthy volunteers. Please contact trials@alkeus.com for any questions.
- Conditions
- Stargardt Disease, Age-related Macular Degeneration, Other Retinal Dystrophies
- Trial name
- NCT02230228
- Testing
- ALK-001 (No generic name)
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Alkeus Pharmaceuticals, Inc.
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl).
- Trial name
- NCT00999609
- Testing
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Spark Therapeutics, Inc.
Stargardt Disease
Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study
The current study is a non-interventional long-term safety follow-up of the subjects who completed STARLIGHT, in accordance with FDA guidance on recipients of human gene therapy products.
- Trial name
- SUSTAIN (NCT06048185)
- Testing
- Gene Therapy Product-MCO-010
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Nanoscope Therapeutics Inc.
Retinitis Pigmentosa, Retinitis
Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study
This study will be conducted following Good Clinical Practice (GCP) and International Conference on Harmonization (ICH) guidelines.
- Conditions
- Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases
- Trial name
- REMAIN (NCT06162585)
- Testing
- Gene Therapy product-MCO-010
- Where
- 6 sites in United States, Puerto Rico
- Volunteers
- 18 people
- Sponsor
- Nanoscope Therapeutics Inc.
Retinitis Pigmentosa, Choroideremia
Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration
The Vision Research and Assessment Institute (VRAI) was established with the purpose of serving as a testing facility for efficacy endpoints for patients with Low Vision.
- Conditions
- Retinitis Pigmentosa, Choroideremia, Stargardt Macular Dystrophy, Stargardt Disease
- Trial name
- NCT06375239
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- Ray Therapeutics, Inc.
Stargardt's Macular Dystrophy
Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
The purpose of this study is: To evaluate the safety and tolerability of RPE cellular therapy in patients with SMD . To evaluate potential efficacy endpoints to be used in future studies RPE cellular therapy.
- Trial name
- NCT01469832
- Testing
- MA09-hRPE
- Where
- 3 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Astellas Institute for Regenerative Medicine
Retinitis Pigmentosa, Retinitis
Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
The purpose of the study is to evaluate the safety and efficacy of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (MCO-010).
- Conditions
- Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases
- Trial name
- RESTORE (NCT04945772)
- Testing
- Gene Therapy Product-MCO-010
- Where
- 6 sites in United States, Puerto Rico
- Volunteers
- 27 people
- Sponsor
- Nanoscope Therapeutics Inc.
Retinitis Pigmentosa, Retinal Dystrophy
Observational Study to Assess the Reliability and Validity of the MLYMT and MLSDT
This is a prospective Multi-Center Observational Study to assess the reliability and validity of the Multi-Luminance Y-Mobility Test (MLYMT) and Multi-Luminance Shape Discrimination Study (MLSDT) Main Outcome Measures: (i)…
- Trial name
- NCT05820100
- Testing
- Observational
- Where
- 8 sites in United States, Puerto Rico
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Nanoscope Therapeutics Inc.
Stargardt Disease
Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease
The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease
- Trial name
- STARLIGHT (NCT05417126)
- Testing
- Gene Therapy-vMCO-010
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Nanoscope Therapeutics Inc.
Biallelic RPE65 Mutation-associated Retinal Dystrophy
Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy.
- Trial name
- NCT05858983
- Testing
- FT-001 Low Dose, FT-001 Mid Dose, FT-001 High Dose
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Frontera Therapeutics
Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies
Single Ascending Dose Study in Participants With LCA10
The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound…
- Conditions
- Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease
- Trial name
- NCT03872479
- Testing
- EDIT-101
- Where
- 5 sites in United States
- Volunteers
- 34 people
- Sponsor
- Editas Medicine, Inc.
Autosomal Dominant Retinitis Pigmentosa, Eye Diseases
A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene
This study evaluates the safety, tolerability and efficacy of QR-1123 injection in the eye (intravitreal; IVT) injections (one eye/unilateral) in subjects receiving a single dose or repeat doses.
- Conditions
- Autosomal Dominant Retinitis Pigmentosa, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies
- Trial name
- AURORA (NCT04123626)
- Testing
- QR-1123
- Where
- 5 sites in United States
- Volunteers
- 11 people
- Sponsor
- ProQR Therapeutics
Leber Congenital Amaurosis 10, Blindness
An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.
PQ-110-005 (BRIGHTEN) is an open-label, dose escalation and double-masked, randomized, controlled study evaluating safety and tolerability of sepofarsen administered via intravitreal (IVT) injection in pediatric subjects (<8…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- BRIGHTEN (NCT04855045)
- Testing
- sepofarsen
- Where
- 9 sites in Belgium, Brazil and 5 more
- Volunteers
- 15 people
- Sponsor
- ProQR Therapeutics
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
Stargardt's Macular Dystrophy
A Follow up Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
The purpose of this study is to evaluate the long term safety and tolerability of hESC-RPE cellular therapy in patients with advanced SMD from 1 to 5 years following the surgical procedure to implant the hESC-RPE cells.
- Trial name
- NCT02941991
- Testing
- hESC-RPE
- Where
- 2 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Astellas Institute for Regenerative Medicine
Retinal Disease, Macular Degeneration
Stem Cell Ophthalmology Treatment Study
This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease. http://mdstemcells.com/scots-ii/
- Conditions
- Retinal Disease, Macular Degeneration, Hereditary Retinal Dystrophy, Optic Nerve Disease
- Trial name
- SCOTS (NCT01920867)
- Testing
- RB (Retrobulbar), ST (Subtenon), IV (Intravenous)
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- MD Stem Cells