Clinical trials: Inherited Retinal Diseases
Von Hippel-Lindau Syndrome
A Phase I/II Trial for Intravitreous Treatment of Severe Ocular Von Hippel-Lindau Disease Using a Combination of the PDGF Antagonist E10030 and the VEGF Antagonist Ranibizumab
Background: People with Von-Hippel-Lindau (VHL) disease may experience significant vision loss as a result of retinal capillary hemangiomas (RCH), the most common and often earliest manifestation of VHL.
- Trial name
- NCT02859441
- Testing
- Ranibizumab, E10030
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- National Eye Institute (NEI)
Retinal Degeneration, Primary Open-angle Glaucoma
Intravitreal Mesenchymal Stem Cell Transplantation in Advanced Glaucoma.
Bone marrow-derived mesenchymal stem cells (MSC) therapy is a promising treatment for several degenerative diseases, including retinopathies and glaucoma, however no previous safety study involving humans has been conducted.
- Trial name
- NCT02330978
- Testing
- Intravitreal transplantation of mesenchymal stem cell, Culture and isolation of autologous bone-marrow mesenchymal stem cells
- Where
- Brazil
- Volunteers
- 2 people
- Sponsor
- University of Sao Paulo
Retinopathy of Prematurity (ROP)
Long-term Safety and Efficacy Outcome Study Comparing Children Previously Enrolled in Study ROPP-2008-01 for the Prevention of Retinopathy of Prematurity (ROP)
The main purpose of this study is to evaluate the long-term efficacy and safety outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (NCT01096784).
- Trial name
- PEDAL (NCT02386839)
- Testing
- rhIGF-1/rhIGFBP-3
- Where
- 16 sites in United States, Italy and 4 more
- Volunteers
- 76 people
- Sponsor
- Shire
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
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.
Age-Related Macular Degeneration (AMD), Macular Degeneration
X-82 to Treat Age-related Macular Degeneration
The purpose of this study is to evaluate the safety and efficacy of X-82 in the treatment of vision loss due to wet AMD.
- Conditions
- Age-Related Macular Degeneration (AMD), Macular Degeneration, Exudative Age-related Macular Degeneration, AMD
- Trial name
- NCT02348359
- Testing
- X-82, Anti-VEGF
- Where
- 35 sites in United States
- Volunteers
- 157 people
- Sponsor
- Tyrogenex
Age-related Macular Degeneration, Stargardt Disease
Efficacy of Acupuncture in Macular Diseases
This is a single-arm study designed to assess the efficacy of acupuncture (Traditional Chinese Medicine) for chronic macular diseases of several types.
- Conditions
- Age-related Macular Degeneration, Stargardt Disease, Macular Dystrophy
- Trial name
- AMAD (NCT02255981)
- Testing
- Acupuncture and massage
- Where
- 2 sites in Colombia
- Volunteers
- 33 people
- Sponsor
- Escuela Neijing
Stargardt Disease
A Natural History of the Progression of Stargardt Disease: Retrospective and Prospective Studies
Stargardt disease is currently an incurable and untreatable macular dystrophy that causes severe visual loss in children and young adults, thereby causing enormous morbidity with economic, psychological, emotional, and social…
- Trial name
- ProgSTAR (NCT01977846)
- Where
- 9 sites in United States, France and 2 more
- Volunteers
- 259 people
- Sponsor
- Foundation Fighting Blindness
Non-infectious Uveitis
Safety and Efficacy of AIN457 in Noninfectious Uveitis
This study was performed to evaluate the efficacy and safety of AIN457 for patients with active uveitis that requires systemic immunosuppression.
- Trial name
- NCT00685399
- Testing
- AIN457, AIN 457
- Where
- 23 sites in United States, Germany
- Volunteers
- 76 people
- Sponsor
- Novartis Pharmaceuticals
Dry Age-related Macular Degeneration, Macular Degeneration
FOCUS: A Phase I/II First in Human Study to Evaluate the Safety and Efficacy of GT005 Administered in Subjects With Dry AMD
This was an open label first in human Phase I/II multicentre study of GT005 in subjects with Macular Atrophy due to Age-related macular degeneration (AMD).
- Conditions
- Dry Age-related Macular Degeneration, Macular Degeneration, Retinal Disease, Eye Diseases
- Trial name
- NCT03846193
- Testing
- GT005, GT005 / Device: Orbit™ Subretinal Delivery System
- Where
- 13 sites in United States, United Kingdom
- Volunteers
- 56 people
- Sponsor
- Gyroscope Therapeutics Limited
Age-Related Maculopathy, Age-Related Maculopathies
Safety And Tolerability Study Of RN6G In Subjects With Advanced Dry, Age-Related Macular Degeneration Including Geographic Atrophy
The purpose of this study is to determine the safety and tolerability of multiple doses of RN6G in subjects with advanced dry, age-related macular degeneration including geographic atrophy.
- Conditions
- Age-Related Maculopathy, Age-Related Maculopathies, Eye Diseases, Retinal Degeneration
- Trial name
- NCT01003691
- Testing
- RN6G
- Where
- 24 sites in United States
- Volunteers
- 24 people
- Sponsor
- Pfizer
Diabetic Macular Edema
A Study to Evaluate the Efficacy, Durability, and Safety of KSI-301 Compared to Aflibercept in Participants With Diabetic Macular Edema (DME)
This Phase 3 study will evaluate the efficacy, durability, and safety of KSI-301 compared to aflibercept in participants with treatment-naïve DME.
- Trial name
- GLIMMER (NCT04603937)
- Testing
- KSI-301, Aflibercept
- Where
- 72 sites in United States, Czechia and 6 more
- Volunteers
- 459 people
- Sponsor
- Kodiak Sciences Inc
Retinopathy of Prematurity (ROP)
Aflibercept for Retinopathy of Prematurity - Intravitreal Injection Versus Laser Therapy
The purpose of this study is to demonstrate how well aflibercept works in babies with ROP, comparing it with laser therapy. The study also has the objective to demonstrate how safe aflibercept is when used in babies, and describe…
- Trial name
- FIREFLEYE (NCT04004208)
- Testing
- Eylea (Aflibercept, BAY86-5321), Laser photocoagulation
- Where
- 90 sites in Argentina, Austria and 25 more
- Volunteers
- 113 people
- Sponsor
- Bayer
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.
Age-related Macular Degeneration
Study to Assess the Efficacy and Safety of Brolucizumab 6mg Compared to Aflibercept 2 mg in a Treat-to-control Regimen (TALON)
This was a 64-week randomized, double-masked, multi-center, active-controlled, two-arm study in patients with neovascular age related macular degeneration (nAMD) who have not previously received anti- vascular endothelial growth…
- Trial name
- TALON (NCT04005352)
- Testing
- Brolucizumab 6 mg, Aflibercept 2 mg
- Where
- 118 sites in United States, Argentina and 18 more
- Volunteers
- 734 people
- Sponsor
- Novartis Pharmaceuticals
Leber Congenital Amaurosis
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
The purpose of the study is to evaluate the safety and efficacy of an adeno-associated virus vector expressing RPE65 in patients with Leber congenital amaurosis caused by mutations in the RPE65 gene. Funding Source - FDA OOPD
- Trial name
- NCT00749957
- Testing
- rAAV2-CB-hRPE65
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Beacon Therapeutics
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.
Wet Age-related Macular Degeneration
A Study to Evaluate the Efficacy and Safety of Intravitreal KSI-301 Compared With Intravitreal Aflibercept in Participants With Neovascular (Wet) Age-related Macular Degeneration (wAMD)
This Phase 3 study will evaluate the efficacy and safety of KSI-301 compared to aflibercept, in participants with neovascular (wet) age-related macular degeneration (wAMD)
- Trial name
- DAYLIGHT (NCT04964089)
- Testing
- KSI-301, Aflibercept
- Where
- 66 sites in United States, Puerto Rico
- Volunteers
- 557 people
- Sponsor
- Kodiak Sciences Inc
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.