Clinical trials: Degeneration
Blindness, Leber Congenital Amaurosis 10
Natural History Study of CEP290-Related Retinal Degeneration
A prospective natural history study with systematic assessments and uniform follow-up to provide a high-quality dataset for assisting in the design of future clinical treatment trials involving patients with CEP290-related…
- Conditions
- Blindness, Leber Congenital Amaurosis 10, Vision Disorders, Eye Diseases
- Trial name
- NCT03396042
- Where
- 7 sites in United States, France and 2 more
- Volunteers
- 26 people
- Sponsor
- Editas Medicine, Inc.
Macular Degeneration
Eylea Post Marketing Surveillance(PMS)
The objective of this PMS is to gain information about safety and efficacy in real practice as a regulatory commitment required by MFDS
- Trial name
- NCT01783925
- Testing
- Aflibercept (Eylea, VEGF Trap-Eye, BAY86-5321)
- Where
- South Korea
- Volunteers
- 3,206 people
- Sponsor
- Bayer
LCA5
Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
The goals of this clinical trial are assess the natural course of LCA5-IRD over 6 months and to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-001 in patients with inherited retinal degeneration…
- Trial name
- NCT05616793
- Testing
- AAV8.hLCA5
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Opus Genetics, Inc
Amyotrophic Lateral Sclerosis
Italian Validation of the Beaumont Behavioural Inventory (BBI)
Up to 50% of patients affected with amyotrophic lateral sclerosis (ALS) can show behavioral dysfunctions within the spectrum of frontotemporal degenerations (FTD) - namely, apathy, disinhibition, loss of sympathy/empathy…
- Trial name
- BBI (NCT04440644)
- Testing
- BBI
- Where
- Italy
- Volunteers
- 190 people, healthy volunteers welcome
- Sponsor
- Istituti Clinici Scientifici Maugeri SpA
Parkinson Disease, Diabetes Mellitus, Type 1
Biological Sample Collection for Research and Biobanking
The New York Stem Cell Foundation (NYSCF) Research Institute is performing this research to study different conditions and diseases by using cells from the body (such as skin or blood cells).
- Conditions
- Parkinson Disease, Diabetes Mellitus, Type 1, Macular Degeneration, ALS
- Trial name
- NCT04270604
- Testing
- Biological sample collection
- Where
- United States
- Volunteers
- 1,279 people, healthy volunteers welcome
- Sponsor
- New York Stem Cell Foundation Research Institute
Friedreich Ataxia
Micronised Resveratrol as a Treatment for Friedreich Ataxia
The aim of this study is to assess the efficacy of micronised resveratrol as a treatment for FRDA, in terms of reducing the severity of ataxia symptoms at 24 weeks, through a randomised blinded, placebo controlled crossover trial.
- Trial name
- NCT03933163
- Testing
- Resveratrol
- Where
- 4 sites in Australia
- Volunteers
- 25 people
- Sponsor
- Murdoch Childrens Research Institute
FXN Gene, FRDA
Biomarker for Friedreich's Ataxia (BioFridA)
International, multicenter, observational, longitudinal monitoring study to identify biomarker/s for Friedreich's Ataxia and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s
- Conditions
- FXN Gene, FRDA, Hereditary Diseases
- Trial name
- BioFridA (NCT04548921)
- Where
- Lebanon
- Volunteers
- 1,000 people
- Sponsor
- CENTOGENE GmbH Rostock
LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations
The purpose of this study is: to evaluate the safety of oral QLT091001 to evaluate whether 7-day treatment with oral QLT091001 can improve visual function in subjects with LCA or RP due to RPE65 or LRAT mutations to evaluate…
- Trial name
- NCT01014052
- Testing
- QLT091001
- Where
- 7 sites in United States, Canada and 3 more
- Volunteers
- 32 people
- Sponsor
- QLT Inc.
Osteoarthritis
Far Infrared Radiation Treatment for Osteoarthritis
A study to evaluate the possibility of using far infrared radiation to manage pain due to osteoarthritis.
- Trial name
- NCT00573352
- Testing
- Far Infrared
- Where
- Canada
- Volunteers
- 2 people
- Sponsor
- GAAD Medical Research Institute Inc.
Friedreich Ataxia
Open Trail of γIFN for Friedreich Ataxia
The investigator proposes an open label pilot study to investigate the safety and efficacy of gamma interferon (γIFN) in patients with Friedreich's Ataxia (FRDA).
- Trial name
- NCT03888664
- Testing
- gamma interferon
- Volunteers
- 12 people
- Sponsor
- IRCCS Eugenio Medea
Macular Edema, Diabetic Retinopathy
Intravitreal Bevacizumab for Diabetic Macular Edema
Diabetic macular edema is a common cause of visual loss among diabetic patients. Studies have demonstrated the role of vascular endothelial growth factor (VEGF) in the pathogenesis of edema.
- Trial name
- NCT00703235
- Testing
- Bevacizumab
- Sponsor
- Isfahan Ophthalmology Research Center
Alzheimer Disease, Early Onset, Lewy Body Disease
Patients With Alzheimer's Disease or Related Youth Disease
This study aims to determine factors related to diagnosis delay for patients with young onset dementia (first symptoms before 60 years old) who live in North of France.
- Conditions
- Alzheimer Disease, Early Onset, Lewy Body Disease, Frontotemporal Lobar Degeneration
- Trial name
- PARMAAJJ (NCT03508024)
- Where
- France
- Volunteers
- 220 people
- Sponsor
- University Hospital, Lille
Usher Syndromes
Natural History Study of Usher Syndrome ( Light4Deaf )
Clinical centres in the LIGHT4DEAF consortium have developed and will continue to improve a reliable, early molecular diagnosis and protocols for full clinical characterisation of Usher syndrome, which will be valuable for the…
- Trial name
- Light4Deaf (NCT04665726)
- Where
- 4 sites in France
- Volunteers
- 400 people
- Sponsor
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Neurodegenerative Disease, Iron Overload
Ferrochelating Treatment in Patients Affected by Neurodegeneration With Brain Iron Accumulation (NBIA)
This trial is a multicenter, unblinded, single-arm pilot study, lasting one year (plus one year extension Amendment n.3 25 August 2009, plus two years follow-up Amendment n.7) , to evaluate the efficacy and safety of the chelator…
- Trial name
- NCT00907283
- Testing
- Deferiprone
- Where
- 3 sites in Italy
- Volunteers
- 20 people
- Sponsor
- Ente Ospedaliero Ospedali Galliera
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
Spine Degeneration, Fusion of Spine (Disease)
Lumbar Discopathy Decompression with Lateral Interbody Fusion: Radiological and Clinical Outcomes
The aim of the study was to introduce a single-segment lumbar spine decompression and fusion with intervertebral implants through modified by the authors lateral lumbar approach.
- Trial name
- NCT06719349
- Where
- Poland
- Volunteers
- 38 people
- Sponsor
- Podkarpackie Oncological Center
Degenerative Disc Disease in Cervical Spine, Stenosis Cervical Spine
PMCF Study Fortilink-SC
This study is setup to be able to gather information on the safety and performance of the Fortilink-SC Ti cage of Paradigm Spine GmbH, up to one year after the surgery.
- Trial name
- NCT07834749
- Testing
- Cervical interbody fusion surgery (standalone)
- Where
- 2 sites in Spain
- Volunteers
- 50 people
- Sponsor
- BAAT Medical Products B.V.
Retinal Dystrophies, Color Vision Defects
Natural History Study of Inherited Retinal Diseases
This prospective, observational investigation seeks to delineate the interplay between chromatic vision deficits and both functional visual outcomes and anatomical retinal biomarkers in individuals affected by Inherited Retinal…
- Conditions
- Retinal Dystrophies, Color Vision Defects, Vision Disorders, Macular Degeneration
- Trial name
- MojiLVCDT (NCT07085533)
- Where
- China
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Augelux Therapeutics
Macular Edema, Cystoid, Uveitis
Vascular Endothelial Growth Factor (VEGF) in Uveitis
This study will look for the presence in blood of a substance called vascular endothelial growth factor (VEGF) in patients with uveitis (eye inflammation).
- Trial name
- NCT00001738
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- National Eye Institute (NEI)
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult participants with ALS, FTD, or mixed ALS/FTD phenotype with a documented…
- Trial name
- NCT05683860
- Testing
- WVE-004 10 mg Q12W
- Where
- 3 sites in Netherlands, United Kingdom
- Volunteers
- 8 people
- Sponsor
- Wave Life Sciences USA, Inc.