Clinical trials: Pigmentary Retinopathy
Non-syndromic Retinitis Pigmentosa
Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa
The objective of this study is to evaluate the safety and tolerability of escalating doses of a gene therapy called GS030-DP (injected study treatment) administered via a single intravitreal injection and repeated light…
- Trial name
- PIONEER (NCT03326336)
- Testing
- Gene therapy: GS030-DP AND Medical device: GS030-MD
- Where
- 3 sites in United States, France and 1 more
- Volunteers
- 10 people
- Sponsor
- GenSight Biologics
X-Linked Retinitis Pigmentosa
4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
This is a Phase 1/2 multicenter study with two parallel parts: an observational natural history cohort and an open-label, prospective interventional trial in males with non-syndromic X-linked retinitis pigmentosa (XLRP) due to…
- Trial name
- NCT04517149
- Testing
- 4D-125 IVT Injection, Observational
- Where
- 8 sites in United States
- Volunteers
- 21 people
- Sponsor
- 4D Molecular Therapeutics
X-Linked Retinitis Pigmentosa
Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
- Trial name
- NCT04671433
- Testing
- Genetic: AAV5-hRKp.RPGR
- Where
- 28 sites in United States, Belgium and 9 more
- Volunteers
- 105 people
- Sponsor
- MeiraGTx Ocular UK Ltd
Retinitis Pigmentosa
BS01 in Patients With Retinitis Pigmentosa
A Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP (AAV2-CAG-ChronosFP) in Patients with Retinitis Pigmentosa
- Trial name
- NCT04278131
- Testing
- BS01
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Bionic Sight LLC
Bardet-Biedl Syndrome (BBS), Obesity
An Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)
An open-label, single-arm, multicenter, Expanded Access Protocol [EAP] designed to provide treatment access to setmelanotide (3 mg, administered subcutaneously [SC], once daily) for eligible patients with BBS who have no…
- Trial name
- NCT05183802
- Testing
- Setmelanotide, administered subcutaneously [SC], once daily.
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Retinitis Pigmentosa, Retinal Degeneration
Safety and Efficacy of Subretinal Implants for Partial Restoration of Vision in Blind Patients
Patients suffering from hereditary retinal degeneration receive a retinal implant to restore sight. Subretinal implant "ON" results in significant visual acuity improvement, when compared to "OFF" condition.
- Trial name
- NCT01024803
- Testing
- Device name: "Retina Implant model Alpha". Surgical implantation of medical device into eye
- Where
- 6 sites in Germany, Hungary and 1 more
- Volunteers
- 39 people
- Sponsor
- Retina Implant AG
X-Linked Retinitis Pigmentosa
A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.
- Trial name
- NCT06275620
- Testing
- AGTC-501 (high dose and standard corticosteroid regimen), AGTC-501 (low dose and standard corticosteroid regimen), AGTC-501 (high dose and modified corticosteroid regimen)
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Beacon Therapeutics
X-Linked Retinitis Pigmentosa
An Observational Study of Japanese Participants With X-linked Retinitis Pigmentosa
The purpose of the study is to identify a cohort of Japanese participants with X-linked retinitis pigmentosa (XLRP) associated with pathogenic variants in the retinitis pigmentosa GTPase regulator (RPGR) gene and to investigate…
- Trial name
- NCT04868916
- Where
- Japan
- Volunteers
- 15 people
- Sponsor
- Janssen Pharmaceutical K.K.
Retinitis Pigmentosa
Expanded Access Program for RP in Adults
This intermediate-size patient population expanded access program is to provide access to investigational product OCU400 for up to 75 patients with Retinitis Pigmentosa (RP) outside of the ongoing clinical trial Phase 3 program…
- Trial name
- NCT06574997
- Testing
- OCU400
- Where
- 5 sites in United States
- Sponsor
- Ocugen
Retinitis Pigmentosa
Safety Issues of Peribulbar Injection of UC-MSC in Patients With Retinitis Pigmentosa
The study will perform UC-MSCs and CM transplantation. The first group will be injected by UCMSC+NaCl. the 2nd group will be injected by UC-MSC+CM. the 3rd group will be injected by CM. Each group consists of 6 subjects.
- Trial name
- RP (NCT04315025)
- Testing
- Umbilical Cord Mesenchymal Stem Cell (UC-MSC), Conditioned Medium (CM)
- Where
- 2 sites in Indonesia
- Volunteers
- 18 people
- Sponsor
- PT. Prodia Stem Cell Indonesia
Retinitis Pigmentosa, Usher Syndromes
Prospective Open Clinical and Genetic Study of Patients With Retinitis Pigmentosa
This study is aimed to characterize Russian population of Retinitis Pigmentosa
- Trial name
- RU-RP (NCT03901391)
- Testing
- Whole Exome Sequencing
- Where
- 2 sites in Russia
- Volunteers
- 130 people
- Sponsor
- Sensor Technology for Deafblind
Retinitis Pigmentosa, Retinal Degeneration
NR600 System Retinal Prosthesis for Patients With Retinal Degenerative Diseases
The study objective is to demonstrate safety of the NR 600 System, and to evaluate the performance of the device in restoring visual activity of daily living in subjects with retinal degenerative diseases and severe visual…
- Trial name
- NR (NCT04295304)
- Testing
- Retinal surgery with implantation of epi-retinal device
- Where
- 6 sites in Belgium, Israel and 1 more
- Volunteers
- 9 people
- Sponsor
- Nano Retina
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 (RP), Age Related Macular Degeneration
Evaluating a New Peptide Therapy for Retinal Diseases: AMD, Diabetic Retinopathy, and Dystrophies
Summary of the Study This clinical trial evaluates a novel peptide-based therapy for treating retinal dystrophies, age-related macular degeneration (AMD), and diabetic retinopathy (DR).
- Conditions
- Retinitis Pigmentosa (RP), Age Related Macular Degeneration, Diabetic Retinopathy
- Trial name
- Retino (NCT06787482)
- Testing
- ACE Retino
- Where
- 2 sites in Egypt
- Volunteers
- 100 people
- Sponsor
- Ace Cells Lab Limited
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
Retinitis Pigmentosa (RP)
Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
The purpose of this study is: To evaluate whether 7-day treatment with oral QLT091001 can improve visual function in RP subjects with an autosomal dominant mutation in RPE65.
- Trial name
- NCT01543906
- Testing
- QLT091001
- Where
- 2 sites in Canada, Ireland
- Volunteers
- 5 people
- Sponsor
- QLT Inc.
Retinitis Pigmentosa, Usher Syndrome Type 2
Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene
The purpose of this study is to evaluate the 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.
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- Stellar (NCT03780257)
- Testing
- QR-421a
- Where
- 7 sites in United States, Canada and 1 more
- Volunteers
- 20 people
- Sponsor
- ProQR Therapeutics
X-Linked Retinitis Pigmentosa
Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.
- Trial name
- HORIZON (NCT03316560)
- Testing
- rAAV2tYF-GRK1-RPGR
- Where
- 4 sites in United States
- Volunteers
- 29 people
- Sponsor
- Beacon Therapeutics
X-Linked Retinitis Pigmentosa (XLRP)
A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa
The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of…
- Trial name
- Landscape (NCT07174726)
- Testing
- Adeno-associated virus vector expressing a human RPGR gene
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Beacon Therapeutics
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