Clinical trials: Inherited Retinal Disease
Stargardt Disease
Study of ALK-001 on the Progression of Stargardt Disease
This study evaluates the efficacy and safety of investigational study drug ALK-001 in participants 8 to 45 years of age, inclusive, with symptoms and signs of autosomal recessive Stargardt disease (STGD)
- Trial name
- NORTHSTAR (NCT07419334)
- Testing
- ALK-001
- Where
- 14 sites in United States
- Volunteers
- 230 people
- Sponsor
- Alkeus Pharmaceuticals, Inc.
Retinopathy of Prematurity
RAINBOW Study: RAnibizumab Compared With Laser Therapy for the Treatment of INfants BOrn Prematurely With Retinopathy of Prematurity
The purpose of this study was to determine if intravitreal ranibizumab is superior to laser ablation therapy in the treatment of retinopathy of prematurity (ROP).
- Trial name
- RAINBOW (NCT02375971)
- Testing
- Ranibizumab, Laser therapy
- Where
- 86 sites in United States, Austria and 24 more
- Volunteers
- 224 people
- Sponsor
- Novartis Pharmaceuticals
Retinopathy of Prematurity, ROP
Validation of i-ROP DL to Detect More Than Mild ROP
The purpose of the pivotal reader study is to assess the readers' accuracy in diagnosing plus disease versus no plus or pre-plus disease with or without the aid of the i-ROP DL.
- Trial name
- NCT06109285
- Testing
- iROP DL
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Siloam Vision
Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP)
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
To evaluate the natural history of visual function in subjects with IRD phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by RPE65 or LRAT gene mutations.
- Trial name
- NCT02575430
- Testing
- No treatment: retrospective chart review
- Where
- 9 sites in United States, Canada and 5 more
- Volunteers
- 59 people
- Sponsor
- QLT Inc.
Leber Congenital Amaurosis, Retinitis Pigmentosa
Treatment of RP and LCA by Primary RPE Transplantation
Early Phase I Study of the Safety and Preliminary Efficacy of Human primary Retinal Pigment Epithelial (HuRPE) Cells Subretinal Transplantation in Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) Patients
- Trial name
- NCT03566147
- Testing
- Human primary Retinal Pigment Epithelial (HuRPE) cells
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Eyecure Therapeutics Inc.
Inherited Retinal Diseases
Global Patient Registry of Inherited Retinal Diseases
The purpose of this study is to better understand the natural history of Inherited Retinal Disease (IRD) and help inform patient management.
- Trial name
- EYERD Registry (NCT05957276)
- Testing
- Standard of Care
- Where
- 75 sites in United States, Australia and 16 more
- Volunteers
- 886 people
- Sponsor
- Janssen Research & Development, LLC
Retinopathy of Prematurity (ROP)
Rainbow Extension Study
The purpose of this study was to evaluate the long term efficacy and safety of intravitreal ranibizumab compared with laser ablation therapy in patients who were treated for retinopathy of prematurity (ROP) in the core study…
- Trial name
- RainbowExt (NCT02640664)
- Testing
- Ranibizumab
- Where
- 73 sites in United States, Austria and 22 more
- Volunteers
- 180 people
- Sponsor
- Novartis Pharmaceuticals
Choroideremia, CHM (Choroideremia) Gene Mutations
Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations
This clinical study evaluates the safety and tolerability of AAV2-hCHM in participants with Choroideremia gene mutations.
- Trial name
- NCT02341807
- Testing
- AAV2-hCHM
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- Spark Therapeutics, Inc.
Diabetic Macular Edema, Diabetic Retinopathy
A Efficacy and Safety Study of Ranibizumab 10mg/ml Injection (Incepta) in Patients With Diabetic Macular Edema
Macular edema in diabetes, defined as retinal thickening within two disc diameters of the center of the macula, results from retinal microvascular changes that compromise the blood-retinal barrier, causing leakage of plasma…
- Conditions
- Diabetic Macular Edema, Diabetic Retinopathy, Macular Edema, Macular Degeneration
- Trial name
- NCT06305416
- Testing
- Ranibizumab 10mg/ml Injection, Lucentis
- Where
- Bangladesh
- Volunteers
- 70 people
- Sponsor
- Incepta Pharmaceuticals Ltd
Diabetic Macular Edema, Retinal Disease
Assess the Efficacy and Safety of Repeat Intravitreal Injections of Foselutoclax (UBX1325) in Patients With DME (ASPIRE)
The goal of this clinical trial is to assess the efficacy and safety of multiple doses of foselutoclax (UBX1325) in patients with Diabetic Macular Edema.
- Conditions
- Diabetic Macular Edema, Retinal Disease, Macular Edema, Diabetes Mellitus
- Trial name
- NCT06011798
- Testing
- Aflibercept, foselutoclax
- Where
- 19 sites in United States
- Volunteers
- 52 people
- Sponsor
- Unity Biotechnology, 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
Leber Hereditary Optic Neuropathy
REALITY LHON Registry
This study is a multi-country retrospective and cross-sectional observational study of affected LHON subjects, based on retrospective subjects' medical chart abstractions and cross-sectional administration of patient-reported…
- Trial name
- REALITY (NCT03295071)
- Testing
- Patient-reported outcomes (PROs)
- Where
- 11 sites in United States, France and 3 more
- Volunteers
- 44 people
- Sponsor
- GenSight Biologics
Geographic Atrophy, Stargardt Disease
Phase 1 Study of C.001 in Retinal Degeneration
This is a first-in-human Phase 1 study evaluating the safety and tolerability of C.001 in patients with retinal diseases involving degeneration of the retinal pigment epithelium.
- Conditions
- Geographic Atrophy, Stargardt Disease, RPE-mediated Maculopathy, Age Related Macular Degeneration
- Trial name
- NCT07594236
- Testing
- Injection
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- Cellio Therapeutics 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
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