Clinical trials: Hereditary Retinal Dystrophy
- Condition Hereditary Retinal Dystrophy
- Funder type Industry (pharma, biotech, devices)
- Has posted results yes
Leber Hereditary Optic Neuropathy
Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
The goal of this clinical trial is to assess the efficacy and safety of GS010 gene therapy - (lenadogene nolparvovec) in subjects with LHON due to the G11778A ND4 mitochondrial mutation with a vision loss up to one year.
- Trial name
- REFLECT (NCT03293524)
- Testing
- GS010
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 98 people
- Sponsor
- GenSight Biologics
Retinitis Pigmentosa
Transcorneal Electrostimulation for Therapy of Retinitis Pigmentosa
The purpose of this study is to determine the long-term effects of transcorneal electrostimulation (TcES) in patients with retinitis pigmentosa (RP).
- Trial name
- NCT01837901
- Testing
- Transcorneal electrostimulation
- Where
- Germany
- Volunteers
- 88 people
- Sponsor
- Okuvision GmbH
Stargardt's Macular Dystrophy
Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)
The purpose of this study is to evaluate the safety and efficacy of avacincaptad pegol intravitreal injection compared to Sham in participants with autosomal recessive Stargardt disease 1 (STGD1).
- Trial name
- NCT03364153
- Testing
- avacincaptad pegol
- Where
- 41 sites in United States, France and 6 more
- Volunteers
- 121 people
- Sponsor
- Astellas Pharma Global Development, Inc.
Retinitis Pigmentosa
A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP)
The primary objective of the study is to assess the safety and tolerability of two dose regimens of recombinant human nerve growth factor (rhNGF) eye drops solution administered over 6 months versus a vehicle control in patients…
- Trial name
- Lumos (NCT02110225)
- Testing
- rhNGF 60 µg/ml eye drops solution, rhNGF 180 µg/ml eye drops solution
- Where
- 5 sites in Italy
- Volunteers
- 50 people
- Sponsor
- Dompé Farmaceutici S.p.A
Retinitis Pigmentosa
Feasibility Study of a Chronic Retinal Stimulator in Retinitis Pigmentosa
The objective of this study is to evaluate the safety and efficacy of the retinal stimulation system by evaluating the data after chronic implantation.
- Trial name
- (A16) (NCT00279500)
- Testing
- Argus 16 Retinal Stimulation System
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Second Sight Medical Products
Retinitis Pigmentosa (RP)
Safety of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Retinitis Pigmentosa
This study evaluates the safety and potential activity of a single dose of live human retinal progenitor cells (jCell) administered to adults with retinitis pigmentosa.
- Trial name
- NCT02320812
- Testing
- human retinal progenitor cells
- Where
- 2 sites in United States
- Volunteers
- 28 people
- Sponsor
- jCyte, Inc
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
Object Finder for a Retinal Prosthesis
The proposed project seeks to provide object recognition as a feature in a retinal implant system. Participants will be able to direct an object recognition application to find a desired object in the field of view of the…
- Trial name
- NCT04319809
- Testing
- Object recognition subsystem
- Where
- United States
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Minnesota HealthSolutions
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
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.
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
Retinitis Pigmentosa
Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa
This study evaluates the changes in visual function at 12 months following a single injection of human retinal progenitor cells compared to sham treated controls in a cohort of adult subjects with RP.
- Trial name
- NCT03073733
- Testing
- human retinal progenitor cells, Mock injection
- Where
- 3 sites in United States
- Volunteers
- 84 people
- Sponsor
- jCyte, Inc
Retinitis Pigmentosa, Usher Syndrome Type 2
Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)
The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with…
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- NCT05176717
- Testing
- QR-421a
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 5 people
- Sponsor
- Laboratoires Thea
Retinitis Pigmentosa
A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late 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
- NCT00447993
- Testing
- Low Dose NT-501, High Dose NT-501
- Where
- 11 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neurotech Pharmaceuticals
Retinitis Pigmentosa
Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa
The primary objective of the study is to assess the safety of repeat injection of human retinal progenitor cells (jCell) in adult subjects with RP that have previously been treated with jCell.
- Trial name
- NCT04604899
- Testing
- human retinal progenitor cells
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- jCyte, Inc
Leber Congenital Amaurosis
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
- Trial name
- OPTIRPE65 (NCT02781480)
- Testing
- AAV RPE65
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 15 people
- Sponsor
- MeiraGTx UK II Ltd
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.
Retinitis Pigmentosa, Usher Syndrome Type 2
Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)
The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- NCT05158296
- Testing
- Ultevursen
- Where
- 15 sites in United States, Germany and 2 more
- Volunteers
- 7 people
- Sponsor
- Laboratoires Thea
Usher Syndrome, Retinitis Pigmentosa
Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B
To evaluate the safety and tolerability of ascending doses of subretinal injections of SAR421869 in participants with Usher syndrome type 1B. To evaluate for possible biological activity of SAR421869.
- Trial name
- NCT01505062
- Testing
- SAR421869
- Where
- 2 sites in United States, France
- Volunteers
- 9 people
- Sponsor
- Sanofi
Advanced Retinitis Pigmentosa
AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa
All participants in phase 1 and phase 2a had hand motion visual acuity or worse. If efficacy was demonstrated from phase 1, better vision subjects could be enrolled; however, efficacy was not demonstrated.
- Trial name
- NCT02556736
- Testing
- AGN-151597
- Where
- 4 sites in United States
- Volunteers
- 14 people
- Sponsor
- AbbVie