Clinical trials: Stage 3 AMD
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.
Pompe Disease, Glycogen Storage Disease Type II (GSD II)
Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
The primary objective of this study was to characterize the pharmacokinetics (PK) of alglucosidase alfa manufactured at the 4000 L scale in participants who had a confirmed diagnosis of Pompe disease.
- Trial name
- PAPAYA (NCT01410890)
- Testing
- alglucosidase alfa
- Where
- 12 sites in United States, Bulgaria and 4 more
- Volunteers
- 21 people
- Sponsor
- Genzyme, a Sanofi Company
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
Inherited Ophthalmic Diseases, Hypopigmentation Disorder
National Ophthalmic Genotyping and Phenotyping Network (eyeGENE (Registered Trademark)), Stage 3 - Expansion of DNA and Data Repositories for Rare Inherited Ophthalmic Diseases
Background: The eyeGENE (Registered Trademark) program is a research resource for inherited eye conditions which includes genotypic and phenotypic data, imaging, and a corresponding biobank of DNA samples from people with a…
- Conditions
- Inherited Ophthalmic Diseases, Hypopigmentation Disorder, Corneal Dystrophy, Blue-cone Monochromacy
- Trial name
- NCT06491615
- Where
- 2 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- National Eye Institute (NEI)
Wet AMD
Investigates Improvement in Vision Following Switching From Avastin and Macugen Over to Lucentis
The purpose of this study is to determine whether people who have received intermittent anti-VEGF therapy have an improvement in vision after being switched to monthly injections of Lucentis.
- Trial name
- NCT00804934
- Testing
- Lucentis
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Retina Institute of Hawaii
Stargardt Disease 1
Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease
The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1
- Trial name
- DRAGON (NCT05244304)
- Testing
- Tinlarebant
- Where
- 19 sites in United States, Australia and 9 more
- Volunteers
- 104 people
- Sponsor
- Belite Bio, 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.
STGD1, Stargardt Disease 1
A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease
The goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease
- Trial name
- DRAGON II (NCT06388083)
- Testing
- Tinlarebant
- Where
- 18 sites in United States, Japan and 1 more
- Volunteers
- 60 people
- Sponsor
- Belite Bio, Inc
Glycogen Storage Disease Type II;Pompe's Disease
Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease
Primary Objective: To determine the effect of avalglucosidase alfa treatment on respiratory muscle strength measured by percent (%) predicted forced vital capacity (FVC) in the upright position, as compared to alglucosidase alfa.
- Trial name
- COMET (NCT02782741)
- Testing
- Avalglucosidase alfa (GZ402666), Alglucosidase alfa (GZ419829)
- Where
- 69 sites in United States, Argentina and 23 more
- Volunteers
- 101 people
- Sponsor
- Genzyme, a Sanofi Company
Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II)
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease.
- Conditions
- Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency
- Trial name
- NCT01597596
- Testing
- alglucosidase alfa
- Where
- 17 sites in United States, Germany and 1 more
- Volunteers
- 4 people
- Sponsor
- Genzyme, a Sanofi Company