Clinical trials: Inherited Retinal Dystrophies
Retinitis Pigmentosa
Phase III Efficacy and Safety Clinical Study of UF-021 for Treatment of Retinitis Pigmentosa
This study is a multicenter trial performed in Japan, consisting of a comparative study period and a continuous administration period.
- Trial name
- NCT01786395
- Testing
- UF-021
- Where
- 34 sites in Japan
- Volunteers
- 202 people
- Sponsor
- R-Tech Ueno, Ltd.
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
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, 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
Stargardt Disease, Stargardt Disease 1
Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)
This is an observational prescreening study. Individuals who are eligible for prescreening will undergo testing procedures that may be used to determine eligibility in ACDN-01 clinical trials.
- Conditions
- Stargardt Disease, Stargardt Disease 1, Cone Rod Dystrophy, Juvenile Macular Degeneration
- Trial name
- NCT06445322
- Testing
- Prescreening Assessments
- Where
- 9 sites in United States
- Volunteers
- 50 people
- Sponsor
- Ascidian Therapeutics, Inc
Retinitis Pigmentosa, Choroideremia
A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)
A phase I/II dose-escalating study of the safety, tolerability and efficacy of KIO-301 administered intravitreally to patients with retinitis pigmentosa and choroideremia (ABACUS).
- Conditions
- Retinitis Pigmentosa, Choroideremia, Non-Syndromic Rod-Dominant Inherited Retinal Diseases
- Trial name
- NCT05282953
- Testing
- KIO-301
- Where
- 3 sites in Australia
- Volunteers
- 32 people
- Sponsor
- Kiora Pharmaceuticals, Inc.
Retinitis Pigmentosa 11, Retinal Degeneration
A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001
This is a Phase 1/2 repeat-dose, open-label, two-arm, parallel group safety and efficacy study of two doses of VP-001 (30 μg and 75 μg) in participants with confirmed PRPF31 mutation-associated retinal dystrophy, including…
- Conditions
- Retinitis Pigmentosa 11, Retinal Degeneration, Retinal Disease, Eye Diseases Hereditary
- Trial name
- DINGO (NCT06852963)
- Testing
- VP-001
- Where
- 6 sites in United States
- Volunteers
- 17 people
- Sponsor
- PYC Therapeutics
Retinitis Pigmentosa, Retinitis Pigmentosa Syndrome
Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis Pigmentosa
This is an open-label, multiple ascending dose study to evaluate the safety, tolerability, and efficacy of EA-2353 in subjects with RP. Unilateral intravitreal injections (IVT) will be administered into the subject's Study Eye.
- Trial name
- NCT05392751
- Testing
- EA-2353
- Where
- 5 sites in United States
- Volunteers
- 14 people
- Sponsor
- Endogena Therapeutics, Inc
Diabetic Retinopathy, Retinal Vein Occlusion
Computer Aided Diagnosis of Multiple Eye Fundus Diseases From Color Fundus Photograph
Blindness can be caused by many ocular diseases, such as diabetic retinopathy, retinal vein occlusion, age-related macular degeneration, pathologic myopia and glaucoma.
- Conditions
- Diabetic Retinopathy, Retinal Vein Occlusion, Retinal Artery Occlusion, Central Serous Chorioretinopathy
- Trial name
- NCT04723160
- Testing
- Software assisted imaging diagnosis
- Where
- 5 sites in China
- Volunteers
- 748 people, healthy volunteers welcome
- Sponsor
- Visionary Intelligence Ltd.
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
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
- Augelux Therapeutics
Retinitis Pigmentosa, Retinal Diseases
Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa
The purpose of the study is to evaluate the safety and tolerability of a single intravitreal injection of virally-carried Multi-Characteristic Opsin I (vMCO-I)
- Conditions
- Retinitis Pigmentosa, Retinal Diseases, Retinal Degeneration
- Trial name
- NCT04919473
- Testing
- Gene Therapy product:vMCO-I
- Where
- India
- Volunteers
- 11 people
- Sponsor
- Nanoscope Therapeutics Inc.
Retinitis Pigmentosa
A Study to Evaluate the Safety of CNTO 2476 in Patients With Advanced Retinitis Pigmentosa
The main purpose of this study is to evaluate the preliminary safety and immunogenicity (ability of an antigen to provoke immune response in the human body) of CNTO 2476, administered subretinally, in participants with advanced…
- Trial name
- NCT00458575
- Testing
- CNTO 2476
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Centocor, Inc.
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.
Stargardt Disease, Stargardt Macular Dystrophy
A Study to Learn How Stargardt-type Eye Conditions Progress in Children and Adults
Macular dystrophies are a group of inherited eye conditions that affect the macula. The macula is in the center of the retina, the light sensitive part at the back of the eye.
- Conditions
- Stargardt Disease, Stargardt Macular Dystrophy, Stargardt-like Macular Dystrophy
- Trial name
- EVOLVE (NCT07425574)
- Testing
- No Intervention
- Where
- 15 sites in United States
- Volunteers
- 90 people
- Sponsor
- Astellas Pharma Global Development, 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
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
- Augelux Therapeutics
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
A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
The purpose of this first-in-human study is to explore the maximum tolerated dose (MTD) of CPK850 as determined by the single ascending dose ranging portion of the study.
- Trial name
- NCT03374657
- Testing
- CPK850
- Where
- Sweden
- Volunteers
- 12 people
- Sponsor
- Novartis Pharmaceuticals
Retinal Dystrophy Due to Biallelic ABCA4 Mutations, Stargardt Disease 1
Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt…
- Trial name
- NCT07002398
- Testing
- VG801
- Where
- China
- Volunteers
- 18 people
- Sponsor
- VeonGen Therapeutics GmbH