Clinical trials: Stargardt Macular Degeneration
Stargardt Disease-1
Study of STG-001 in Subjects With Stargardt Disease
This is an open-label, multicenter study in subjects with Stargardt disease, comparing 2 doses of STG-001 with regard to safety, pharmacokinetics and pharmacodynamics.
- Trial name
- NCT04489511
- Testing
- STG-001
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Stargazer Pharmaceuticals, Inc.
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
Stargardt Disease 1, STGD1
A Phase 1, First-in-Human Study of CITY-RBP4 in Healthy Volunteers and STGD1 Participants
This is a first-in-human (FIH), multicenter, randomized, double-masked, placebo-controlled adaptive clinical study evaluating the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) of CITY-RBP4, following…
- Conditions
- Stargardt Disease 1, STGD1, Healthy Volunteers
- Trial name
- NCT07836452
- Testing
- CITY-RBP4
- Where
- Australia
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- City Therapeutics
Stargardt's Macular Dystrophy
Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular Dystrophy
This is a safety and tolerability trial to evaluate the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with Stargardt's Macular Dystrophy (SMD).
- Trial name
- NCT01345006
- Testing
- MA09-hRPE
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Astellas Institute for Regenerative Medicine
Stargardt Disease, Stargardt Macular Degeneration
Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease
The purpose of this study is to determine the long term safety and tolerability of ALK-001 (C20-D3-retinyl acetate), and to explore the effects of ALK-001 on the progression of Stargardt disease in patients between the ages of 8…
- Conditions
- Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
- Trial name
- TEASE (NCT02402660)
- Testing
- ALK-001
- Where
- 14 sites in United States
- Volunteers
- 160 people
- Sponsor
- Alkeus Pharmaceuticals, Inc.
Stargardt's Macular Dystrophy
A Follow up Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
The purpose of this study is to evaluate the long term safety and tolerability of hESC-RPE cellular therapy in patients with advanced SMD from 1 to 5 years following the surgical procedure to implant the hESC-RPE cells.
- Trial name
- NCT02941991
- Testing
- hESC-RPE
- Where
- 2 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Astellas Institute for Regenerative Medicine
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
Stargardt Disease
A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease
Phase 2/3 Pivotal Confirmatory Clinical Trial is a randomized, outcome assessor-masked, multicenter study, that will enroll fifty-one (51) subjects.
- Trial name
- GARDian3 (NCT05956626)
- Testing
- OCU410ST
- Where
- 14 sites in United States
- Volunteers
- 51 people
- Sponsor
- Ocugen
Stargardt Disease, Stargardt Macular Degeneration
Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)
The purpose of this open-label, multicenter study is to determine the long-term safety, pharmacokinetics and effects of ALK-001 (C20-D3-retinyl acetate) on the progression of Stargardt disease.
- Conditions
- Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
- Trial name
- NCT04239625
- Testing
- ALK-001
- Where
- 8 sites in United States
- Volunteers
- 200 people
- Sponsor
- Alkeus Pharmaceuticals, Inc.
Stargardt's Macular Dystrophy
Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
The purpose of this study is: To evaluate the safety and tolerability of RPE cellular therapy in patients with SMD . To evaluate potential efficacy endpoints to be used in future studies RPE cellular therapy.
- Trial name
- NCT01469832
- Testing
- MA09-hRPE
- Where
- 3 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Astellas Institute for Regenerative Medicine
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.
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.
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.
Stargardt Disease
Safety and Efficacy of Emixustat in Stargardt Disease
The purpose of this study is to determine if emixustat hydrochloride reduces the rate of progression of macular atrophy compared to placebo in subjects with Stargardt disease. Funding Source -- FDA OOPD
- Trial name
- SeaSTAR (NCT03772665)
- Testing
- Emixustat
- Where
- 29 sites in United States, Brazil and 9 more
- Volunteers
- 194 people
- Sponsor
- Kubota Vision Inc.
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
Stargardt Disease
A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)
The purpose of the 039-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-039 in participants with Stargardt disease secondary to a biallelic mutation of the ABCA4 gene.
- Trial name
- CELESTE (NCT07161544)
- Testing
- AAVB-039
- Where
- 11 sites in United States, Norway and 1 more
- Volunteers
- 75 people
- Sponsor
- AAVantgarde Bio Srl
Stargardt, Stargardt's Disease
An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene
This is an Observational Study to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene This is a multicenter study which will enroll approximately 75…
- Conditions
- Stargardt, Stargardt's Disease, Stargardt Disease, STGD1
- Trial name
- POLARIS (NCT06435000)
- Where
- 20 sites in United States, Germany and 1 more
- Volunteers
- 75 people
- Sponsor
- Splice Bio
Stargardt Disease
A Study of DC6001 Tablet in Healthy Chinese Adult Subjects
This study adopts a randomized, double-blind, parallel placebo-controlled dose-escalation design, consisting of two parts: Part 1 includes a single ascending dose (SAD) study plus a food effect (FE) study, and Part 2 is a…
- Trial name
- NCT07417566
- Testing
- DC6001
- Where
- China
- Volunteers
- 76 people, healthy volunteers welcome
- Sponsor
- Heronova Pharmaceuticals
Stargardt Disease
Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease
A Phase 1/2, open-label, non-randomized, dose-escalation study, where a minimum of 9 and a maximum of 18 eligible patients with Stargardt Disease will be enrolled sequentially in up to 3 dose cohorts of RTx-021.
- Trial name
- AURORA (NCT07439887)
- Testing
- RTx-021
- Where
- 3 sites in United States
- Volunteers
- 18 people
- Sponsor
- Ray Therapeutics, Inc.
Stargardt's Disease
Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration
Primary Objective: To evaluate the long-term safety and tolerability of SAR422459 in patients with Stargardt's Macular Degeneration. Secondary Objective: To assess: Safety Biological activity
- Trial name
- NCT01736592
- Testing
- Long term follow up in all patients who received SAR422459 in previous study TDU13583
- Where
- 2 sites in United States, France
- Volunteers
- 27 people
- Sponsor
- Sanofi