Clinical trials: Optic Atrophy
LGMD1B, LGMD1C
Long-Term Development of Muscular Dystrophy Outcome Assessments
This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD).
- Conditions
- LGMD1B, LGMD1C, LGMD1D, LGMD1E
- Trial name
- GRASP-01-005 (NCT05989620)
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Virginia Commonwealth University
Eye Diseases Hereditary, Retinal Disease
Inherited Retinal Degenerative Disease Registry
The My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation.
- Conditions
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome
- Trial name
- MRTR (NCT02435940)
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Foundation Fighting Blindness
Wolfram Syndrome
Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1
Wolfram syndrome (WFS:OMIM 222300) is a group of inherited disorders that usually appear in childhood and cause diabetes, optic atrophy leading to loss of vision, deafness and often diabetes insipidus.
- Trial name
- NCT05659368
- Testing
- Tirzepatide
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- Ospedale San Raffaele
Geographic Atrophy, Age-Related Macular Degeneration
Geographic Atrophy Treatment Evaluation
The purpose of this study was to evaluate the safety and efficacy of AL-8309B Ophthalmic Solution versus Vehicle administered as a topical ocular drop for the treatment for geographic atrophy (GA) secondary to age-related macular…
- Trial name
- GATE (NCT00890097)
- Testing
- AL-8309B Ophthalmic Solution, AL-8309B Vehicle
- Volunteers
- 772 people
- Sponsor
- Alcon Research
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
Epiretinal Membrane, Dry Age Related Macular Degeneration
(CIRRUS-2023-1) CLINICAL CLIN Assessment of PathFinder 1.0 on the CIRRUS 6000 and the CIRRUS 5000
This study will measure how well PathFinder 1.0 detects retinal abnormalities on macular cube OCT B-scans by assessing its sensitivity and specificity.
- Conditions
- Epiretinal Membrane, Dry Age Related Macular Degeneration, Macular Abnormalities, Retina Disease
- Trial name
- NCT07680244
- Testing
- PathFinder 1.0 on CIRRUS HD-OCT
- Where
- 6 sites in United States
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- Carl Zeiss Meditec-Dublin CoCe
Wolfram Syndrome
Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome
This trial aims to investigate the efficacy, safety and tolerability of sodium valproate in the treatment of patients with Wolfram syndrome.
- Trial name
- NCT03717909
- Testing
- Sodium Valproate 200Mg E/C Tablet
- Where
- 6 sites in France, Poland and 2 more
- Volunteers
- 63 people
- Sponsor
- University of Birmingham
Stargardt Disease
An Observational Study in Children and Adults With Stargardt Disease
This multicenter, prospective, longitudinal, observational study in approximately 80 subjects with Stargardt disease secondary to biallelic mutations in the ABCA4 gene (STGD1) aims to evaluate prognostic factors of disease…
- Trial name
- NCT06591806
- Where
- 2 sites in United States, Norway
- Volunteers
- 80 people
- Sponsor
- AAVantgarde Bio Srl
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
Age-related Macular Degeneration
Tools to Optimize Patient Presentation After Onset of Exudative Age-Related Macular Degeneration (AMD)
Exudative age-related macular degeneration ("wet" AMD) continues to be a leading cause of central vision loss in the US for those over fifty years of age, despite the availability of several effective interventions to contain…
- Trial name
- NCT01337414
- Testing
- VMS diary booklet, Standard of Care
- Where
- United States
- Volunteers
- 324 people
- Sponsor
- Johns Hopkins University
Age-Related Macular Degeneration, Geographic Atrophy
Central micrOperimetry and Radial OCT Evaluation in Geographic Atrophy (CORE-GA): a Prospective Pilot Study
This study is being conducted to assess two imaging methods-central microperimetry and radial OCT (Optical Coherence Tomography)-in patients with Geographic Atrophy (GA) or those who are at risk of developing this condition.
- Trial name
- CORE-GA (NCT07556406)
- Testing
- Optical coherence tomography, Microperimetry
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- University of California, San Francisco
Geographic Atrophy of the Macula
A Phase 2 Efficacy and Safety Study of GAL-101, 2% Ophthalmic Solution in Non-foveal Geographic Atrophy Secondary to Non-neovascular AMD
Age-related macular degeneration (AMD) affects millions of elderly patients. When advanced, there is Geographic Atrophy (GA) in the retina.
- Trial name
- eDREAM (NCT06659549)
- Testing
- GAL-101
- Where
- 24 sites in United States, Armenia and 6 more
- Volunteers
- 110 people
- Sponsor
- Galimedix Therapeutics Inc
Leber Hereditary Optic Neuropathy (LHON)
Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations
The objective of this clinical study is to evaluate the safety and efficacy of NR082 in the treatment of LHON caused by mitochondrial ND4 gene mutation.
- Trial name
- NCT05293626
- Testing
- NR082 Injection, Injection needle
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Neurophth Therapeutics Inc
Leber Congenital Amaurosis 10, Blindness
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- ILLUMINATE (NCT03913143)
- Testing
- sepofarsen
- Where
- 14 sites in United States, Belgium and 7 more
- Volunteers
- 36 people
- Sponsor
- ProQR Therapeutics
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
Neurodegenerative Diseases, Parkinson Disease
Neurodegenerative Diseases Progression Markers (MARKERS-NDD)
MARKERS-NDD is a prospective, observational, longitudinal study, which aims to collect data from patients affected by neurodegenerative diseases (NDD) followed longitudinally for routine examinations performed as part of normal…
- Conditions
- Neurodegenerative Diseases, Parkinson Disease, Synucleinopathies, Lewy Body Disease
- Trial name
- MARKERS-NDD (NCT06596746)
- Testing
- Observation
- Where
- Italy
- Volunteers
- 600 people
- Sponsor
- Casa di Cura San Raffaele Cassino
Geographic Atrophy, Wet Macular Degeneration
A Population-based Study of Macular Choroidal Neovascularization in a Chinese Population
To investigate pathomorphological and functional variations of choroidal neovascularization (CNV) in age-related macular degeneration (AMD) in a Chinese population using optical coherence tomography (OCT) to find which kinds of…
- Conditions
- Geographic Atrophy, Wet Macular Degeneration, Retinal Drusen
- Trial name
- NCT01666821
- Testing
- Follow-up observation
- Where
- China
- Volunteers
- 59 people, healthy volunteers welcome
- Sponsor
- Shanghai University of Traditional Chinese Medicine
Parkinson Disease, Progressive Supranuclear Palsy
Biomarkers in Parkinsonian Syndromes
This is a prospective observational study to identify biomarkers in parkinson syndromes. Patients with parkinsonian syndromes at the early stages of disease will be recruited and will be followed up until their established…
- Conditions
- Parkinson Disease, Progressive Supranuclear Palsy, Multiple System Atrophy, Atypical Parkinsonism
- Trial name
- PROATYP (NCT06501469)
- Where
- Greece
- Volunteers
- 200 people
- Sponsor
- Non-profit organization for scientific research in Parkinson's disease and related disorders
Healthy
Clinical Assessment of Barley and Oat Phytochemicals
The goal of this placebo-controlled, 3-way crossover study is to determine the acute (24-h) bioavailability and pharmacokinetics of the major phytochemicals found whole barley and oats, as well as their effects on selected…
- Trial name
- NCT01303562
- Testing
- Whole grain oats and barley
- Where
- United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Tufts University
Dry Age-related Macular Degeneration, Geographic Atrophy
A Phase IIb Randomized, Multicenter Trial of Subretinal CPCB-RPE1 in Advanced Dry AMD (Geographic Atrophy)
This is a Phase IIb randomized, clinical trial designed to assess the safety and efficacy of unilateral implantation of the CPCB-RPE1 implant in subjects with geographic atrophy involving the fovea.
- Trial name
- PATCH-AMD (NCT06557460)
- Testing
- Surgical implantation of the CPCB-RPE1 implant
- Where
- 5 sites in United States
- Volunteers
- 24 people
- Sponsor
- Regenerative Patch Technologies, LLC