Clinical trials: Retinal Dystrophies
Retinitis Pigmentosa
Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients
This is zM-02's safety, tOlerability, and efficacy in retinitis pigmentOsa first-in-humaN study (MOON). This trial is meant to evaluate the safety and efficacy of ZM-02 in Retinitis pigmentosa (RP) patients.
- Trial name
- MOON (NCT06292650)
- Testing
- ZM-02-L, ZM-02-H, ZM-02-S
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Zhongmou Therapeutics
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.
Retinitis Pigmentosa
Promising ROd-cone DYstrophy Gene therapY
This is a two-step, multicenter, Phase I/II study including an open-label dose-escalation phase (Step 1) and a three-arm, controlled, double-masked, randomized extension phase (Step 2), in subjects with advanced RCD due to a…
- Trial name
- PRODYGY (NCT05748873)
- Testing
- SPVN06
- Where
- 6 sites in United States, France
- Volunteers
- 33 people
- Sponsor
- SparingVision
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
Bietti Crystalline Dystrophy, Inherited Retinal Diseases
Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients With Bietti Crystalline Dystrophy
This is a phase 3 study to evaluate the efficacy and safety of VGR-R01 in subjects with Bietti Crystalline Dystrophy. This is a multicenter, randomized controlled study which will enroll 45 subjects.
- Trial name
- NCT06699108
- Testing
- VGR-R01
- Where
- China
- Volunteers
- 45 people
- Sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.
Retinitis Pigmentosa (RP), Usher Syndrome
24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
The goal of this clinical trial is to learn if NPI-001 works to prevent progression of retinitis pigmentosa in adults diagnosed with Usher syndrome. It will also provide information about the safety of NPI-001.
- Trial name
- NCT07290530
- Testing
- N-acetylcysteine amide
- Volunteers
- 80 people
- Sponsor
- Nacuity Pharmaceuticals, Inc.
Retinitis Pigmentosa, Stargardt Macular Dystrophy
Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD)
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, Stargardt Macular Dystrophy, Stargardt Disease, Geographic Atrophy From Age-related Macular Degeneration
- Trial name
- DEFINE-IRD (NCT07502664)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Ray Therapeutics, Inc.
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
Mitochondrial Diseases, Mitochondrial Respiratory Chain Deficiencies
A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease
This will be a double blind, randomised, placebo controlled, single and multiple oral dose study conducted in 3 parts: Part A, Part B and Part C.
- Conditions
- Mitochondrial Diseases, Mitochondrial Respiratory Chain Deficiencies, MELAS Syndrome, Mitochondrial Myopathies
- Trial name
- NCT03888716
- Testing
- KL1333
- Where
- 2 sites in United Kingdom
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Abliva AB
ROSAH
Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients
The purpose of this study is to evaluate the safety and tolerability of DF-003 in retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and migraine headache (ROSAH) syndrome patients.
- Trial name
- NCT06395285
- Testing
- DF-003
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 12 people
- Sponsor
- Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)
Retinitis Pigmentosa (RP), Rod Cone Dystrophy
Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy (SPVN20) in Subjects With Rod-Cone Dystrophy
This open-label, multi-center study is to evaluate the safety, tolerability, and efficacy of escalating doses of a gene therapy called SPVN20 administered via a single intravitreal injection in participants with advanced Rod-Cone…
- Trial name
- NYRVANA (NCT07807111)
- Testing
- SPVN20 (high dose), SPVN20 (medium dose), SPVN20 (low dose)
- Where
- 3 sites in Belgium, France and 1 more
- Volunteers
- 27 people
- Sponsor
- SparingVision
Retinitis Pigmentosa
Transcorneal Electrical Stimulation - Multicenter Safety Study
The purpose of this study is to expand the safety assessment of repeated applications of an electrical current from a DTL-like electrode in patients with RP.
- Trial name
- NCT01835002
- Testing
- Transcorneal electrostimulation
- Where
- 7 sites in Denmark, Germany and 1 more
- Volunteers
- 105 people
- Sponsor
- Okuvision GmbH
Retinitis Pigmentosa, Cone-Rod Dystrophy
IRIS PILOT - Extended Pilot Study With a Retinal Implant System
Investigate whether blind subjects that fulfil the patient criteria provided with a Retinal Implant are able to differentiate between simple patterns like horizontal bar, vertical bar and cross.
- Conditions
- Retinitis Pigmentosa, Cone-Rod Dystrophy, Choroideremia
- Trial name
- NCT00427180
- Testing
- Retinal Implant System (IRIS)
- Volunteers
- 20 people
- Sponsor
- Intelligent Medical Implants GmbH
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, 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
Retinitis Pigmentosa
Intravitreal Quantum Dots (QD) for Advanced Retinitis Pigmentosa (RP) (QUANTUM)
Pilot, randomized, observer and participant masked, sham and fellow eye controlled, interventional clinical device trail to evaluate the safety and effectiveness of the 2C-QD device to improve visual function in adults with…
- Trial name
- NCT05841862
- Testing
- 2C-QD
- Sponsor
- 2C Tech Corp
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
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 Disease, Stargardt Macular Degeneration
A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)
This Phase 1/2 study will evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt's Type 1 (STGD1).
- Conditions
- Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy
- Trial name
- ASTRA (NCT06942572)
- Testing
- SB-007
- Where
- 5 sites in United States
- Volunteers
- 57 people
- Sponsor
- Splice Bio
Stargardt Disease, Cone Rod Dystrophy
Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and…
- Conditions
- Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration, Stargardt Disease 1
- Trial name
- NCT06467344
- Testing
- ACDN-01
- Where
- 11 sites in United States
- Volunteers
- 15 people
- Sponsor
- Ascidian Therapeutics, Inc