Clinical trials: Retinal Dystrophies
Retinitis Pigmentosa
Argus II Retinal Prosthesis System - Better Vision RP Study
The study is conducted to evaluate the safety and benefit of the Argus II System in a selected patient population with advanced Retinitis Pigmentosa who have a measurable central residual visual field smaller than or equal to 5…
- Trial name
- NCT03418116
- Testing
- Argus II Retinal Prosthesis
- Where
- 3 sites in Germany
- Sponsor
- Second Sight Medical Products
Retinitis Pigmentosa, Usher Syndrome Type 2
Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa
This clinical trial is a single-site, 30 patient study for participants who have early stage retinitis pigmentosa, or Usher syndrome (type 2 or 3). Funding Source - FDA OOPD and Foundation Fighting Blindness.
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Usher Syndrome Type 3
- Trial name
- NCT01530659
- Testing
- NT-501
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Neurotech Pharmaceuticals
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
Retinitis Pigmentosa
Development of Visual Function Evaluation Method (2)
The objective of this study is to develop the comprehensive visual function evaluation method in severe visually impaired patient.
- Trial name
- NCT03626207
- Where
- Japan
- Volunteers
- 8 people
- Sponsor
- Astellas Pharma Inc
Retinitis Pigmentosa
A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa
The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II \& III, and Choroideremia.
- Trial name
- NCT00447980
- Testing
- NT-501
- Where
- 12 sites in United States
- Volunteers
- 73 people
- Sponsor
- Neurotech Pharmaceuticals
Retinitis Pigmentosa
A Study in Subjects With Retinitis Pigmentosa
An open-label Phase II clinical trial, 8 (eight) subjects with retinitis pigmentosa due to rhodopsin mutations (including P23H) will be identified and treated with serial intravitreal injections of ADX-2191 in the worse seeing…
- Trial name
- NCT05392179
- Testing
- ADX-2191
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Aldeyra Therapeutics, Inc.
Retinitis Pigmentosa
A Study to Investigate the Safety of DSP-3077 After a Unilateral Eye Injection in Male and Female Participants 18 Years of Age or Older With Retinitis Pigmentosa
The Goal of this study is to evaluate the safety, tolerability, and clinical responses following single dose of DSP-3077. Study enrolls both male and female patients in 3 cohorts with each cohort defined by visual acuity (VA)…
- Trial name
- NCT06891885
- Testing
- DSP-3077 Retinal Sheet Cohort 1, DSP-3077 Retinal Sheet Cohort 2, DSP-3077 Retinal Sheet Cohort 3
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sumitomo Pharma America, Inc.
Retinitis Pigmentosa
Long-term Safety of UC-MSC Transplantation in Patients With Retinitis Pigmentosa
The study will perform to follow-up UC-MSCs and CM transplantation. 18 patients will be called back to be examined after 5 years of UC-MSC and/or CM transplantation.
- Trial name
- NCT05786287
- Testing
- Conditioned Medium (CM), Umbilical Cord Mesenchymal Stem Cell (UC-MSC)
- Where
- 2 sites in Indonesia
- Volunteers
- 18 people
- Sponsor
- PT. Prodia Stem Cell Indonesia
Stargardt Disease, Stargardt Macular Degeneration
Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)
The purpose of this open-label, multicenter study is to determine the long-term safety, pharmacokinetics and effects of ALK-001 (C20-D3-retinyl acetate) on the progression of Stargardt disease.
- Conditions
- Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
- Trial name
- NCT04239625
- Testing
- ALK-001
- Where
- 8 sites in United States
- Volunteers
- 200 people
- Sponsor
- Alkeus Pharmaceuticals, Inc.
Retinal Degeneration, Inherited Retinal Dystrophy Primarily Involving Sensory Retina
Impact on Daily Life of Patients Using the Subretinal Implant RETINA IMPLANT Alpha AMS
This study aims to assess the impact of the subretinal implant RETINA IMPLANT Alpha AMS on the patient's daily life, using validated activities of daily living and questionnaires.
- Trial name
- NCT03561922
- Testing
- RETINA IMPLANT Alpha AMS
- Where
- 2 sites in France
- Volunteers
- 2 people
- Sponsor
- Retina Implant AG
Retinal Disease, Age-Related Macular Degeneration
Stem Cell Ophthalmology Treatment Study II
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.
- Conditions
- Retinal Disease, Age-Related Macular Degeneration, Retinitis Pigmentosa, Stargardt Disease
- Trial name
- SCOTS2 (NCT03011541)
- Testing
- Arm 1
- Where
- 4 sites in United States, Austria and 1 more
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
Retinitis Pigmentosa
Retina Implant Pilot Trial to Evaluate Safety & Efficacy in Blind Patients Having Degenerated Photo-receptors
The outcome of this trial will reveal the possibilities of the retinal implant to improve the situation of patients with hereditary retinal blindness caused by degenerations of the outer retina.
- Trial name
- NCT00515814
- Testing
- Retina implant is surgically placed into subretinal position
- Where
- 2 sites in Germany
- Volunteers
- 15 people
- Sponsor
- Retina Implant AG
Retinitis Pigmentosa
A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa
The goal of the study is to investigate the safety, tolerability and efficacy of up to 3 doses of KIO-301 administered by intravitreal (IVT) injection bilaterally every 6 weeks in patients with late-stage retinitis pigmentosa…
- Trial name
- ABACUS-2 (NCT06628947)
- Testing
- 100 μg KIO-301, 50 μg KIO-301
- Where
- 5 sites in Australia
- Volunteers
- 36 people
- Sponsor
- Kiora Pharmaceuticals, Inc.
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
Retinitis Pigmentosa, Eye Diseases
Natural History Study of Patients With EYS-Associated RP
This natural history study of patients with EYS mutations from Russia and former CIS (Commonwealth of Independent States) territories will accelerate the development of outcome measures for clinical trials.
- Trial name
- RUS_EYS (NCT07228793)
- Testing
- Whole exome/genome sequencing
- Where
- Russia
- Volunteers
- 45 people
- Sponsor
- Sensor Technology for Deafblind
Leber Congenital Amaurosis, LCA
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
Primary Objective: To evaluate the safety and tolerability of ascending doses of ATSN-101 administered as a unilateral subretinal injection in patients with Leber Congenital Amaurosis (LCA) caused by autosomal recessive guanylate…
- Conditions
- Leber Congenital Amaurosis, LCA, LCA1
- Trial name
- NCT03920007
- Testing
- ATSN-101, ATSN-101 Diluent Solution, Prednisone
- Where
- 2 sites in United States
- Volunteers
- 15 people
- Sponsor
- Atsena Therapeutics Inc.
X-Linked Retinitis Pigmentosa
A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112
The objective of the study is to evaluate the safety, tolerability and efficacy of a single sub-retinal injection of BIIB112 in participants with X-linked retinitis pigmentosa (XLRP).
- Trial name
- XIRIUS (NCT03116113)
- Testing
- BIIB112
- Where
- 8 sites in United States, United Kingdom
- Volunteers
- 50 people
- Sponsor
- Biogen
Retinitis Pigmentosa
New Enrollment Post-Approval Study of the Argus® II Retinal Prosthesis System
This post-approval study is being implemented to monitor the use of Argus II System in a larger US population than available within pre-approval studies.
- Trial name
- NCT01860092
- Where
- 17 sites in United States
- Volunteers
- 53 people
- Sponsor
- Second Sight Medical Products
Retinitis Pigmentosa (RP), Usher Syndrome Type 2
Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
The purpose of this Phase 2 Open-label study is to evaluate the long-term safety and tolerability of Ultevursen administered via intravitreal injection (IVT) in participants with Retinitis Pigmentosa due to mutations in exon 13…
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- CAPELLA (NCT07796646)
- Testing
- No intervention, will not receive any active study intervention, Intravitreal Injection of Ultevursen
- Volunteers
- 81 people
- Sponsor
- Laboratoires Thea
Stargardt's Macular Dystrophy
Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular Dystrophy
This is a safety and tolerability trial to evaluate the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with Stargardt's Macular Dystrophy (SMD).
- Trial name
- NCT01345006
- Testing
- MA09-hRPE
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Astellas Institute for Regenerative Medicine