Clinical trials: Optic Atrophy
Increased Lordosis/Scoliosis, Hyporeflexia
Biomarker for Duchenne Muscular Dystrophy
International, multicenter, observational, longitudinal study to identify biomarker/s for Duchenne Muscular Dystropy (DMD) and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s.
- Conditions
- Increased Lordosis/Scoliosis, Hyporeflexia, Duchenne Muscular Dystrophy, Red-Green Color Blindness
- Trial name
- BioDuchenne (NCT02994030)
- Where
- 12 sites in Albania, Egypt and 6 more
- Volunteers
- 103 people
- Sponsor
- CENTOGENE GmbH Rostock
Age-Related Macular Degeneration
Genetic Study of Age-Related Macular Degeneration
This study will examine skin and blood cells for genetic changes related to the development of age-related macular degeneration, an eye disease that can significantly impair the ability to read, drive, and carry out daily…
- Trial name
- NCT00357578
- Where
- United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- National Eye Institute (NEI)
Lung Adenocarcinoma
Expression of Optic Atrophy Type 1 (OPA1) Protein in Lung Adenocarcinoma
Optic atrophy type 1(OPA1) is a nuclear dynamin-related GTPase, targeted to the inner mitochondrial membrane, which plays a role in mitochondrial fusion. Mitochondria fusion is associated with process of apoptosis. .
- Trial name
- NCT01249053
- Where
- Taiwan
- Volunteers
- 100 people
- Sponsor
- China Medical University Hospital
Macular Edema, Diabetic Macular Edema
A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of IVT MHU650 in Macular Edema Patients
This was an open-label, multi-center, FIH study with a single ascending dose (SAD) design that assessed the safety, tolerability and pharmacokinetics (PK) of a single IVT dose of MHU650 in up to 24 participants with macular edema.
- Conditions
- Macular Edema, Diabetic Macular Edema, Neovascular Age-related Macular Degeneration, Retinal Vein Occlusion
- Trial name
- NCT04635800
- Testing
- MHU650
- Where
- 6 sites in United States, Puerto Rico
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
Multiple System Atrophy, Progressive Supranuclear Palsy (PSP)
Synaptic Loss in Multiple System Atrophy
In this study the investigators would like to investigate the degree of damage of the synapses, an important part of the neurons vital for the communications between neurons, in Multiple System Atrophy (MSA), and pathology…
- Trial name
- NCT05121012
- Testing
- Study procedures for MSA patients, Study procedures for PSP patients
- Where
- United Kingdom
- Volunteers
- 36 people
- Sponsor
- University of Exeter
Wolfram Syndrome, Fecal Incontinence
Mechanisms of Fecal Incontinence in Wolfram Syndrome
In order to clarify the mechanism of fecal incontinence in in Wolfram syndrome and to characterize its risk factors, the examiner will reviewed the files of patients followed in a Reference Center for Rare Diseases in…
- Trial name
- NCT07313085
- Testing
- Retrospective case-control analysis
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Hôpital Necker-Enfants Malades
LGMD2I, LGMDR9
Global FKRP Registry
Mutations in the Fukutin Related Protein (FKRP) gene cause the condition Limb Girdle Muscular Dystrophy type R9 (LGMDR9) also known as LGMD2I, and the rarer conditions Congenital Muscular Dystrophy (MDC1C), Muscle Eye Brain…
- Conditions
- LGMD2I, LGMDR9, Limb Girdle Muscular Dystrophy, Congenital Muscular Dystrophy
- Trial name
- NCT04001595
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 1,000 people
- Sponsor
- Newcastle University
Dry Age-related Macular Degeneration
EXPLORE: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005
The purpose of this clinical study was to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with geographic atrophy secondary to age-related macular degeneration…
- Trial name
- EXPLORE (NCT04437368)
- Testing
- GT005
- Where
- 55 sites in United States, Australia and 6 more
- Volunteers
- 98 people
- Sponsor
- Gyroscope Therapeutics Limited
Age Related Macular Degeneration, Geographic Atrophy
Project AMD: Comprehensive Characterisation of Age-Related Macular Degeneration and Its Progression
Age-related macular degeneration (AMD) is the leading cause of irreversible vision loss worldwide, and nearly two million Australians have some signs of AMD.
- Conditions
- Age Related Macular Degeneration, Geographic Atrophy, AMD, Age-related Macular Degeneration
- Trial name
- Project AMD (NCT04739319)
- Testing
- N/A Observational study
- Where
- Australia
- Volunteers
- 2,500 people, healthy volunteers welcome
- Sponsor
- Center for Eye Research Australia
Leber Hereditary Optic Neuropathy
RESCUE and REVERSE Long-term Follow-up
The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in…
- Trial name
- RESTORE (NCT03406104)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 62 people
- Sponsor
- GenSight Biologics
Geographic Atrophy
Study of Pegcetacoplan (APL-2) Therapy in Patients With Geographic Atrophy
The primary objectives of the study are to assess the safety, tolerability and evidence of activity of multiple intravitreal (IVT) injections of pegcetacoplan in subjects with Geographic Atrophy associated with Age-Related…
- Trial name
- FILLY (NCT02503332)
- Testing
- Pegcetacoplan
- Where
- 46 sites in United States, Australia and 1 more
- Volunteers
- 246 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Leber Hereditary Optic Neuropathy (LHON), Leber's Hereditary Optic Neuropathy (LHON)
Pilot Study of the Efficacy of Nicotinamide (Vitamin B3) in Leber's Hereditary Optic Neuropathy
Leber Hereditary Optic Neuropathy (LHON) is a rare genetic disease that causes sudden and severe vision loss, usually in young adults.
- Conditions
- Leber Hereditary Optic Neuropathy (LHON), Leber's Hereditary Optic Neuropathy (LHON), Mitochondrial Disease, Optic Nerve Disease
- Trial name
- NICOLHON (NCT07258667)
- Testing
- Nicotinamide treatment
- Where
- France
- Volunteers
- 13 people
- Sponsor
- University Hospital, Angers
Parkinson Disease, Essential Tremor
Brain Network Activation in Patients With Movement Disorders
The diagnosis and management of movement disorders, such as Parkinson's disease (PD), parkinson-plus syndromes (PPS), dystonia, essential tremor (ET), normal pressure hydrocephalus (NPH) and others is challenging given the lack…
- Conditions
- Parkinson Disease, Essential Tremor, Dystonia, Normal Pressure Hydrocephalus
- Trial name
- BNA-MDi (NCT03269201)
- Where
- Israel
- Volunteers
- 300 people
- Sponsor
- Sheba Medical Center
Geographic Atrophy
Study to Evaluate the Safety of Intravitreal APL-2 in Patients Diagnosed With Geographic Atrophy
Safety Assessment of APL-2 in Patients with Geographic Atrophy
- Trial name
- NCT03777332
- Testing
- PEGCETACOPLAN (APL-2)
- Where
- 5 sites in United States
- Volunteers
- 19 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Parkinson's Disease, Parkinsonism
Modifiable Variables in Parkinsonism (MVP)
We are trying to identify factors associated with improved quality of life and fewer PD symptoms. We are attempting to identify practices, beliefs, and therapies used by individuals who report excellent quality of life, few PD…
- Conditions
- Parkinson's Disease, Parkinsonism, MSA - Multiple System Atrophy, Progressive Supranuclear Palsy
- Trial name
- NCT02194816
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- Bastyr University
Mitochondrial Diseases
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study…
- Trial name
- NCT02473445
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- Amgen
Geographic Atrophy, Age-related Macular Degeneration
Intravitreal LFG316 in Patients With Age-related Macular Degeneration (AMD)
This study was conducted in two parts; Part A and Part B: Part B was initially planned to include two cohorts. Cohort 2 was cancelled following an interim analysis for efficacy in Part A of the study, and not due to any safety…
- Trial name
- NCT01527500
- Testing
- LFG316, LFG316 Lower dose
- Where
- 19 sites in United States
- Volunteers
- 158 people
- Sponsor
- Novartis Pharmaceuticals
Age-Related Macular Degeneration, Geographic Atrophy
Multicenter Clinical Study of the SING-IMT in Patients with Late-stage AMD
The objective of this study is to determine the safety and effectiveness of the smaller incision, new generation (SING), implantable miniature telescope (IMT) in patients with moderate-severe central vision loss due to late-stage…
- Trial name
- CONCERTO (NCT05438732)
- Testing
- smaller incision, new generation implantable miniature telescope (SING-IMT)
- Where
- 10 sites in United States
- Volunteers
- 125 people
- Sponsor
- VisionCare, Inc.
Retina Detachment, Eye Injuries
Multi-center Clinical Observation of FCVB in Guangdong Province
This study provides further theoretical guidance for clinical application of FCVB through observing and collecting various indicators before and after surgical treatment of the FCVB in the real world.
- Conditions
- Retina Detachment, Eye Injuries, Eye Atrophy, Silicone Emulsion
- Trial name
- NCT03908515
- Testing
- Foldable Capsular Vitreous Body (FCVB)
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Sun Yat-sen University
Parkinson's Disease, Alzheimer's Disease
Biomarkers in Neural Disorders
This study seeks to establish the sensitivity and specificity of what appears to be a unique brainstem biomarker of Parkinson's Disease (PD) - an electrically induced olygosynaptic nasotrigeminal reflex response - in…
- Conditions
- Parkinson's Disease, Alzheimer's Disease, Progressive Supranuclear Palsy, Essential Tremor
- Trial name
- NCT02761707
- Testing
- Electrical Brainstem Responses, Olfactory Tests, Visual Deprivation
- Where
- United States
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania