Clinical Trials for Degenerative Nerve Diseases

Currently registered clinical trials for Degenerative Nerve Diseases from ClinicalTrials.gov. 22 recruiting, 50 total studies.

This content is for informational purposes only. Always consult a healthcare professional.

Source: ClinicalTrials.gov — U.S. National Library of Medicine — A database of privately and publicly funded clinical studies conducted around the world (U.S. National Library of Medicine, public domain).

This page lists clinical trials related to Degenerative Nerve Diseases from the ClinicalTrials.gov database.

Trial Summary

  • Total studies: 50
  • Recruiting: 22
  • Active, not recruiting: 11
  • Completed: 5
  • Other: 12

Results are refreshed periodically and may not reflect the most current information available from ClinicalTrials.gov.

Recruiting Trials

StatusRecruiting
PhaseN/A
SponsorNational Human Genome Research Institute (NHGRI)
Enrollment5000
Study TypeOBSERVATIONAL
ConditionsFamilial Mediterranean Fever (FMF); Autoinflammation; Periodic Fever; Fever; Genetic Diseases

This study is designed to explore the genetics and pathophysiology of diseases presenting with intermittent fever, including familial Mediterranean fever, TRAPS, hyper-IgD syndrome, and related diseases.

The following individuals may be eligible for this natural history study: 1) patients with known or suspected familial Mediterranean fever, TRAPS, hyper-IgD syndrome or related disorders; 2) r…

NCT00018889 — Phenotype/Genotype Correlations in Movement Disorders

StatusRecruiting
PhaseN/A
SponsorNational Institute of Neurological Disorders and Stroke (NINDS)
Enrollment2500
Study TypeOBSERVATIONAL
ConditionsMovement Disorder

The goal of this protocol is to identify families with inherited movement disorders and evaluate disease manifestations to establish an accurate clinical diagnosis by using newest technological advances and investigate the underlying molecular mechanisms. Studies of inherited movement disorders in large families with good genealogical records are especially valuable. Patients with diseases of k…

NCT01496625 — National Eye Institute Biorepository for Retinal Diseases

StatusRecruiting
PhaseN/A
SponsorNational Eye Institute (NEI)
Enrollment650
Study TypeOBSERVATIONAL
ConditionsAge-Related Macular Degeneration; Diabetic Retinopathy; Von Hippel-Lindau Syndrome; Retinal Disease; Retinal Vein Occlusion

Background:

- To understand diseases of the retina and the eye, information is needed about people with and without such diseases. Researchers want to study these people and follow them over time. They also want to study body tissues and blood to understand the nature of eye disease. Studying genes, cells, and tissues may help them understand why some people get eye problems and others do not…

NCT03011541 — Stem Cell Ophthalmology Treatment Study II

StatusRecruiting
PhaseN/A
SponsorMD Stem Cells
Enrollment500
Study TypeINTERVENTIONAL
ConditionsRetinal Disease; Age-Related Macular Degeneration; Retinitis Pigmentosa; Stargardt Disease; Optic Neuropathy
Interventions

This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.

NCT02795052 — Neurologic Stem Cell Treatment Study

StatusRecruiting
PhaseN/A
SponsorMD Stem Cells
Enrollment500
Study TypeINTERVENTIONAL
ConditionsNeurologic Disorders; Nervous System Diseases; Neurodegenerative Diseases; Neurological Disorders; Stroke
Interventions

This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3 of the nasal passages in order to determine if such a treatment will provide improvement in neurologic function for patients with certain neurologic conditions. http://mdstemcells.com/nest/

StatusRecruiting
PhaseN/A
SponsorNational Institute of Neurological Disorders and Stroke (NINDS)
Enrollment360
Study TypeOBSERVATIONAL
ConditionsFrontotemporal Dementia; Amyotrophic Lateral Sclerosis; Progressive Supranuclear Palsy

Background:

Neurodegenerative disorders can lead to problems in movement or memory. Some can cause abnormal proteins to build up in brain cells. Researchers want to understand whether these diseases have related causes or risk factors.

Objective:

To test people with movement or thinking and memory problems to see if they are eligible for research studies.

Eligibility:

People ages 18 and ol…

NCT03334292 — Natural History of Wilson Disease

StatusRecruiting
PhaseN/A
SponsorYale University
Enrollment300
Study TypeOBSERVATIONAL
ConditionsWilson Disease

The purpose of the registry/repository is to provide a mechanism to store data and specimens to support the conduct of future research about Wilson disease (WD). The overall aim is to determine the optimal testing for diagnosis and parameters for monitoring treatment of WD that will aid product utilization and development.

NCT03848663 — Visual Remapping to Aid Reading With Field Loss

StatusRecruiting
PhaseN/A
SponsorUniversity of Minnesota
Enrollment108
Study TypeINTERVENTIONAL
ConditionsAge-related Macular Degeneration
Interventions;

Reading performance in patients with Central Vision Loss and age matched controls with artificial scotomas will be measured with and without different kinds of remapping of missing text to different parts of the visual field.

NCT05072483 — Natural History Study of CADASIL

StatusRecruiting
PhaseN/A
SponsorNational Heart, Lung, and Blood Institute (NHLBI)
Enrollment155
Study TypeOBSERVATIONAL
ConditionsCardiovascular Disease; Arterial Stiffness; Germline Mutation in the NOTCH 3 Gene; Pathogenesis of CADASIL; Clinical Phenotype of CADASIL
Interventions

Background:

CADASIL (cerebral autosomal dominant arteriopathy with subcortical infarct and leukoencephalopathy) is a genetic disorder. It causes narrowing of the small blood vessels and can lead to strokes and dementia. Researchers want to monitor people with CADASIL over time.

Objective:

To learn more about how CADASIL affects a person s blood vessels over time.

Eligibility:

Adults ages 1…

NCT06627179 — Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

StatusRecruiting
PhasePhase 2
SponsorLaboratoires Thea
Enrollment81
Study TypeINTERVENTIONAL
ConditionsRetinitis Pigmentosa (RP); Usher Syndrome Type 2; Deaf Blind; Retinal Disease; Eye Diseases, Hereditary
Interventions;

The purpose of this Phase 2b study is to evaluate the safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. This is a multicenter Double-masked, Randomized, Sham-controlled study which will enroll 81 subjects.

NCT06789445 — A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)

StatusRecruiting
PhasePhase 1 / Phase 2
SponsorBlueRock Therapeutics
Enrollment54
Study TypeINTERVENTIONAL
ConditionsPrimary Photoreceptor Disease; Retinitis Pigmentosa (RP); Usher Syndrome; Inherited Retinal Disease (IRD); Rod-Cone Dystrophy
Interventions;

Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 focuses on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 o…

NCT06953583 — A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich’s Ataxia

StatusRecruiting
PhasePhase 3
SponsorBiogen
Enrollment255
Study TypeINTERVENTIONAL
ConditionsFriedreich Ataxia
Interventions;

In this study, researchers will learn more about omaveloxolone, also known as BIIB141 or SKYCLARYS®. Omaveloxolone is already approved for people with Friedreich’s Ataxia (FA) who are 16 years of age or older. However, it is not yet available for younger teens and children. The main goal of this study is to learn how omaveloxolone affects symptoms of FA and its safety in younger participants be…

NCT07180355 — A Study of SGT-212 Gene Therapy in Friedreich’s Ataxia

StatusRecruiting
PhasePhase 1
SponsorSolid Biosciences Inc.
Enrollment10
Study TypeINTERVENTIONAL
ConditionsFriedreich’s Ataxia (FA)
Interventions

This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich’s ataxia (FA). It will be delivered via dual intradentate nucleus (IDN) and intravenous (IV) administration to participants with FA.

All participants will receive SGT-212 and will be enrolled in the study for approximately 5 years.

NCT07348250 — Open-label Study to Evaluate Brain α-Synuclein Deposition Using PET and [18F]MK-0947 in Parkinson’s Disease

StatusRecruiting
PhaseEarly Phase 1
SponsorInvicro
Enrollment22
Study TypeINTERVENTIONAL
ConditionsParkinson’s Disease (PD); Parkinson’s Disease; Parkinson’s Disease (Disorder)
Interventions

This clinical study is being conducted to learn more about a new imaging drug called [18F]MK-0947, which is designed to help doctors see changes in the brain related to Parkinson’s disease (PD). PD is a condition that affects movement, balance, and thinking. The drug works with a type of scan called PET (Positron Emission Tomography) to show areas of the brain where a protein called α-synucle…

NCT07445841 — Protecting the Brain From Post-Stroke Cognitive Impairment and Dementia With Multimodal Exercise Training

StatusRecruiting
PhaseN/A
SponsorMcGill University
Enrollment120
Study TypeINTERVENTIONAL
ConditionsStroke
Interventions;

The rates of cognitive decline and dementia after stroke are disproportionately high. Strategies that can protect the brain early after the stroke event could reduce the future risk of cognitive decline and dementia in these patients. Although physical exercise is usually recommended after stroke, there is very little information about the protective effect of exercise implemented in early stag…

NCT07213700 — InRAD Observational Study

StatusRecruiting
PhaseN/A
SponsorStichting International Registry for Alzheimer’s Disease and other Dementias Foundation
Enrollment50000
Study TypeOBSERVATIONAL
ConditionsAlzheimer’s Disease(AD); Mild Cognitive Impairment (MCI); Subjective Cognitive Decline (SCD); Non-Alzheimer Degenerative Dementia
Interventions

The goal of this international observational study is to evaluate long-term disease outcomes and treatment safety in people with Alzheimer’s disease (PwAD), by collecting real-world data from routine clinical practice across global clinical centers.

The InRAD Registry Observational Study has several aims:

  • To collect medical information for many years from a large group of people with Alzhei…

NCT07660250 — Vagus Nerve Stimulation for Myelopathy

StatusRecruiting
PhaseN/A
SponsorWashington University School of Medicine
Enrollment36
Study TypeINTERVENTIONAL
ConditionsDegenerative Cervical Myelopathy
Interventions;

The objective of this study is to generate preliminary data to establish the feasibility and effectiveness of transauricular vagus nerve stimulation (taVNS) to improve post-operative outcomes of moderate to severe degenerative cervical myelopathy (DCM)

NCT07325487 — Interposed Nucleus aDBS for Ataxia

StatusRecruiting
PhaseN/A
SponsorUniversity of Florida
Enrollment5
Study TypeINTERVENTIONAL
ConditionsSpinocerebellar Ataxia (SCA); Spinocerebellar Ataxia Type 6
Interventions

This is a single-center, open-label study designed to evaluate the feasibility, safety, and preliminary efficacy of cerebellar adaptive deep brain stimulation (aDBS) in adults with spinocerebellar ataxia type 6 (SCA6). A total of 5 participants will be enrolled.

Participants will undergo surgical implantation of deep brain stimulation (DBS) leads targeting the motor interposed nucleus of the c…

NCT07498426 — A Study to Evaluate the Efficacy of NIO752 in Participants With Progressive Supranuclear Palsy

StatusRecruiting
PhasePhase 3
SponsorNovartis Pharmaceuticals
Enrollment300
Study TypeINTERVENTIONAL
ConditionsProgressive Supranuclear Palsy Richardson Syndrome (PSP-RS)
Interventions;

This Phase III study is intended to evaluate the efficacy and safety of NIO752 in participants with Progressive Supranuclear Palsy (PSP). Eligible participants will be randomized to receive either NIO752 or placebo followed by an open-label extension.

NCT07158905 — AV-1980R (Tau Vaccine) in Preclinical Alzheimer’s Disease (TAURUS-1980)

StatusRecruiting
PhasePhase 1
SponsorInstitute for Molecular Medicine
Enrollment48
Study TypeINTERVENTIONAL
ConditionsAlzheimer Disease; Preclinical Alzheimer’s Disease
Interventions; ;

This is a Phase 1, multicenter, randomized, double-blind, placebo-controlled, multiple dose-escalating trial to evaluate the safety, tolerability, and immune response of AV-1980R, an investigational vaccine targeting tau protein, in participants with preclinical Alzheimer’s disease. Up to 48 cognitively unimpaired adults aged 65-80 with biomarker evidence of early Alzheimer’s disease will be en…

NCT07341763 — Brain Stimulation Effects on Orientation and Mobility Skills in Adults With Vision Impairment

StatusRecruiting
PhaseN/A
SponsorUniversity of Waterloo
Enrollment20
Study TypeINTERVENTIONAL
ConditionsRetinitis Pigmentosa (RP); Rod Cone Dystrophy; Visually Impaired Persons; Peripheral Visual Field Defect of Both Eyes; Low Vision, Both Eyes
Interventions;

This pilot clinical trial evaluates whether non-invasive brain stimulation improves the orientation and mobility (O&M) skills of individuals with constricted visual fields in both eyes. The study is composed of three visits. The first visit is meant to confirm eligibility by performing a few clinical tests. Eligible participants will then complete two additional visits, one in which the partic…

NCT06688578 — Testing Effectiveness of a Stochastic Noise Stimulator to Immediately Improve Balance and Gait

StatusRecruiting
PhaseN/A
SponsorMassachusetts Eye and Ear Infirmary
Enrollment120
Study TypeINTERVENTIONAL
ConditionsVestibular Disorder; Aging
Interventions;

The goal of this intervention study is to determine if a new electronic stimulation device, similar to a TENS can improve balance and make walking easier in older individuals with reduced balance function. The main question aims to answer the following:

Can using the device improve walking speed in older individuals?

Participants will be asked to perform a number of tasks while wearing the de…

Active, Not Recruiting

NCT02994719 — Gait Analysis in Neurological Disease

StatusActive, not recruiting
PhaseN/A
SponsorBeth Israel Deaconess Medical Center
Enrollment120
Study TypeOBSERVATIONAL
ConditionsParkinson’s Disease; Parkinsonian Disorders; Atypical Parkinson Disease; Progressive Supranuclear Palsy; Multiple System Atrophy
Interventions;

The purpose of this study is to investigate whether speed-dependent measures of gait (so called gait signatures) can be identified in patients with neurological conditions that affect gait, particularly in subjects with parkinsonian disorders.

NCT02741440 — Natural History of Spinocerebellar Ataxia Type 7 (SCA7)

StatusActive, not recruiting
PhaseN/A
SponsorNational Eye Institute (NEI)
Enrollment22
Study TypeOBSERVATIONAL
ConditionsSpinocerebellar Ataxia

Background:

Spinocerebellar ataxia type 7 (SCA7) is a disease in which people have problems with coordination, balance, speech and vision. It is caused by a change in the ATXN7 gene. A mutation in this ATXN7 gene causes changes in eye cells, which can lead to vision loss. There is no cure for SCA7 but researchers are looking for possible treatments. Researchers need more information about SCA7…

NCT03655223 — Early Check: Expanded Screening in Newborns

StatusActive, not recruiting
PhaseN/A
SponsorRTI International
Enrollment30000
Study TypeOBSERVATIONAL
ConditionsSpinal Muscular Atrophy; Fragile X Syndrome; Fragile X - Premutation; Duchenne Muscular Dystrophy; Hyperinsulinemic Hypoglycemia, Familial 1
Interventions

Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and families who screen positive, and 3) evaluate the Early Check program. The Early Check screening will lead to earlier identification of newborns with rare health condi…

NCT03701399 — Troriluzole in Adult Participants With Spinocerebellar Ataxia

StatusActive, not recruiting
PhasePhase 3
SponsorBiohaven Pharmaceuticals, Inc.
Enrollment299
Study TypeINTERVENTIONAL
ConditionsSpinocerebellar Ataxias; Spinocerebellar Ataxia Type 1; Spinocerebellar Ataxia Type 2; Spinocerebellar Ataxia Type 3; Spinocerebellar Ataxia Type 6
Interventions;

The purpose of this study is to compare the efficacy of Troriluzole (200 mg once daily) versus placebo after 48 weeks of treatment in subjects with spinocerebellar ataxia (SCA).

NCT04516499 — Neurofilament Surveillance Project (NSP)

StatusActive, not recruiting
PhaseN/A
SponsorThe Bluefield Project to Cure Frontotemporal Dementia
Enrollment342
Study TypeOBSERVATIONAL
ConditionsFrontotemporal Dementia; Frontotemporal Lobar Degeneration; FTD-GRN; FTD; FTLD

This is a biomarker study designed to collect and analyze blood specimens from individuals carrying known familial frontotemporal lobar degeneration (f-FTLD) mutations compared to a control group of individuals without known f-FTLD mutations. The NSP is an ancillary study to the ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration" (ALLFTD) study, NCT04363684. More information can be fo…

NCT05184569 — Veri-T: A Trial of Verdiperstat in Patients With svPPA Due to TDP-43 Pathology

StatusActive, not recruiting
PhasePhase 1
SponsorPeter Ljubenkov, MD
Enrollment64
Study TypeINTERVENTIONAL
ConditionsSemantic Dementia
Interventions

The purpose of the study is to test the safety and tolerability of twice daily Verdiperstat in patients with semantic variant primary progressive aphasia (svPPA) due to frontotemporal lobar degeneration with TDP-43 pathology (FTLD-TDP). Three-fourths of the participants will receive Verdiperstat and one-fourth will receive Placebo during the 24-week treatment duration.

NCT05596760 — Promoting Goals-of-Care Discussions for Patients With Memory Problems and Their Caregivers

StatusActive, not recruiting
PhaseN/A
SponsorUniversity of Washington
Enrollment1802
Study TypeINTERVENTIONAL
ConditionsDementia; Dementia, Vascular; Mixed Dementias; Alzheimer Disease; Huntington Disease
Interventions

The goal of this clinical trial is to improve communication among clinicians, patients with memory problems, and their family members. We are testing a way to help clinicians have better conversations to address patients’ goals for their healthcare. To do this, we created a simple, short guide called the “Jumpstart Guide.” The goal of this research study is to show that using this kind of guide…

StatusActive, not recruiting
PhaseN/A
SponsorBayer
Enrollment2500
Study TypeOBSERVATIONAL
ConditionsVisual Impairment; Neovascular Age-related Macular Degeneration (nAMD); Diabetic Macula Edema (DME)
Interventions

This is an observational study in which only data are collected from people who have already been prescribed aflibercept 8 mg by their own doctors.

In this study, data from adults with visual impairment due to neovascular age-related macular degeneration (nAMD) or diabetic macula edema (DME) will be collected and studied.

Visual impairment is any degree of vision loss that affects a person’s …

NCT06151600 — A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J

StatusActive, not recruiting
PhaseN/A
SponsorElpida Therapeutics SPC
Enrollment20
Study TypeOBSERVATIONAL
ConditionsPeripheral Neuropathy; Neuro-Degenerative Disease; Neuromuscular Diseases

This is a multicenter, longitudinal, prospective observational natural history study of subjects with a molecularly confirmed diagnosis of CMT4J. The study will enroll 20 subjects of any age into a uniform protocol for follow-up and evaluations. Subject visits will occur every 12 months + 4 weeks for up to 2 years.

NCT06672445 — Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2

StatusActive, not recruiting
PhasePhase 1
SponsorArrowhead Pharmaceuticals
Enrollment39
Study TypeINTERVENTIONAL
ConditionsSpinocerebellar Ataxia Type 2
Interventions;

Adult participants with spinocerebellar ataxia type 2 (SCA2) who carry ≥33 cytosine, adenine, guanine (CAG) repeats in the ATXN2 gene, and who have met all protocol eligibility criteria will be randomized to receive a single dose of ARO-ATXN2 or placebo and be evaluated for safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) parameters.

NCT07033481 — Clinical Study of Neflamapimod in Patients With Primary Progressive Aphasia

StatusActive, not recruiting
PhasePhase 2
SponsorEIP Pharma Inc
Enrollment20
Study TypeINTERVENTIONAL
ConditionsNonfluent Variant Primary Progressive Aphasia (nfvPPA)
Interventions;

The goal of this exploratory study is to evaluate the effect of neflamapimod in participants with nonfluent variant primary progressive aphasia (nfvPPA). We aim to evaluate the safety, pharmacokinetics and clinical effects of neflamapimod of participants with nfvPPA.

Not Yet Recruiting

NCT07680101 — Intra-Articular Adelmidrol 2%/Hyaluronic Acid 1% With or Without m/umPEA, in Knee Pain Due to Joint Degeneration.

StatusNot yet recruiting
PhaseN/A
SponsorUniversita di Verona
Enrollment80
Study TypeINTERVENTIONAL
ConditionsDegenerative Joint Disease of Knee; Rheumatoid Arthritis
Interventions;

Degenerative knee joint diseases are characterized by chronic pain, inflammation, and functional impairment. In these conditions, mast cell density in the synovial membrane is increased, resulting in an excessive release of biological mediators involved in key mechanisms of neuroinflammation and in hyaluronic acid depolymerisation. Adelmidrol acts as a preventive antioxidant due to its radical …

NCT07641140 — Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of LY-M003 Injection in Adult Patients With Wilson’s Disease

StatusNot yet recruiting
PhasePhase 1 / Phase 2
SponsorLingyi Biotech Co., Ltd.
Enrollment18
Study TypeINTERVENTIONAL
ConditionsWilson’s Disease
Interventions; ;

This is a multicenter, open-label, single-arm, single-dose Phase I/II clinical study. It aims to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacodynamic (PD) and pharmacokinetic (PK) profiles of LY-M003 Injection in patients with Wilson’s Disease (WD).

NCT07638813 — MSC-Exosome Therapy for Frontotemporal Dementia

StatusNot yet recruiting
PhasePhase 1 / Phase 2
SponsorRuijin Hospital
Enrollment33
Study TypeINTERVENTIONAL
ConditionsFrontotemporal Dementia
Interventions; ;

This study is testing a new treatment for Frontotemporal Dementia (FTD) - a progressive brain disease that affects personality, behavior, and language. Currently, there is no cure for FTD and no approved medication that can slow down or stop the disease. Existing treatments only help manage some symptoms temporarily.

The investigational treatment in this study is made from exosomes - tiny part…

NCT07661524 — Passive Versus Active Music Therapy Parkinson’s Disease

StatusNot yet recruiting
PhaseN/A
SponsorUniversity of Alabama at Birmingham
Enrollment28
Study TypeINTERVENTIONAL
ConditionsParkinson Disease (PD)
Interventions;

The purpose of this pilot study is to identify the effects of active versus passive music therapy on functional ability and psychophysiological responses to goal-directed exercise in people with Parkinson’s disease.

NCT07658027 — Vestibular Stimulation in Sleep for Neurorehabilitation Patients

StatusNot yet recruiting
PhaseN/A
SponsorCereneo AG
Enrollment20
Study TypeINTERVENTIONAL
ConditionsMemory Deficit Aquired Due to the Disease, e.g. Stroke, Neurodegeneration, Inflammatory Disease of CNS, Traumatic Brain Injury
Interventions
  1. Why are we conducting this study? You are a patient of the neurorehabilitation clinic with one of the therapy focuses on memory and learning. Neuropsychological training is a standard therapeutic approach in this case. It is also known that better quality of sleep may not only improve the well-being of individuals but also enhance memory and learning. In this study, we investigate how the ro…

NCT07681713 — Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich’s Ataxia (FA)

StatusNot yet recruiting
PhasePhase 3
SponsorPTC Therapeutics
Enrollment120
Study TypeINTERVENTIONAL
ConditionsFriedreich’s Ataxia
Interventions

The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.

NCT07290530 — 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome

StatusNot yet recruiting
PhasePhase 3
SponsorNacuity Pharmaceuticals, Inc.
Enrollment80
Study TypeINTERVENTIONAL
ConditionsRetinitis Pigmentosa (RP); Usher Syndrome
Interventions;

The goal of this clinical trial is to learn if NPI-001 works to prevent progression of retinitis pigmentosa in adults diagnosed with Usher syndrome. It will also provide information about the safety of NPI-001. The main questions it aims to answer are:

Does NPI-001 slow down the loss of photoreceptors? What medical problems do participants have when taking NPI-001? Researchers will compare NPI…

NCT07216924 — Remapping Text in Sentence and Word Tasks to Aid Reading With Central Vision Loss

StatusNot yet recruiting
PhaseN/A
SponsorUniversity of Minnesota
Enrollment100
Study TypeINTERVENTIONAL
ConditionsMacular Degeneration; Central Visual Impairment
Interventions; ;

Reading performance in patients with Central Vision Loss will be measured with and without missing text being remapped to different parts of the visual field in a variety of different reading tasks.

Enrolling by Invitation

NCT00341874 — Genetic Analyses of Nonsyndromic and Syndromic Deafness in Pakistan

StatusEnrolling by invitation
PhaseN/A
SponsorNational Institute on Deafness and Other Communication Disorders (NIDCD)
Enrollment24000
Study TypeOBSERVATIONAL
ConditionsHearing Disorder

Objective: One objective of this study is to genetically map and identify mutated genes for human hereditary hearing loss. A second objective is to study the function of these genes in the auditory system using mouse models. Human hereditary hearing impairment is the result of abnormal ear development, abnormal ear function or both. Although the genes for numerous deafness loci have been mapped…

NCT00950430 — Imaging of Brain Amyloid Plaques in the Aging Population

StatusEnrolling by invitation
PhasePhase 4
SponsorMayo Clinic
Enrollment8000
Study TypeINTERVENTIONAL
ConditionsAlzheimer’s Disease; Dementia With Lewy Bodies; Frontotemporal Dementia; Vascular Dementia
Interventions; ;

This is a prospective, open label, non-therapeutic, diagnostic imaging study. The purpose of this study is to utilize Pittsburgh Compound B positron emission imaging (PiB PET) to ascertain the relationship between change in amyloid burden over time, and concurrent change in clinical status.

NCT05617014 — Alzheimer’s Disease Neuroimaging Initiative 4

StatusEnrolling by invitation
PhaseN/A
SponsorUniversity of Southern California
Enrollment1500
Study TypeOBSERVATIONAL
ConditionsMild Cognitive Impairment; Alzheimer Disease; Dementia
Interventions; ;

The Alzheimer’s Disease Neuroimaging Initiative 4 (ADNI4) is a non-randomized, longitudinal, natural history study designed to validate biomarkers, improve clinical trial design, and advance understanding of Alzheimer’s disease across the full disease spectrum. Building on the success of ADNI1, ADNI-GO, ADNI2, and ADNI3, ADNI4 integrates clinical, cognitive, imaging, genetic, and fluid biomarke…

NCT07222930 — Learning Amyloid and Tau Results

StatusEnrolling by invitation
PhaseN/A
SponsorUniversity of Wisconsin, Madison
Enrollment240
Study TypeOBSERVATIONAL
ConditionsAlzheimer Disease
Interventions;

This is a prospective, single-cohort, single-center, observational study to assess if learning one’s Alzheimer’s disease biomarker test result impacts longitudinal psychosocial, behavioral, and neuropsychological outcomes, and to identify factors that moderate and mediate these outcomes. Participants enrolled in this study are requested to complete surveys at four timepoints after learning thei…

Completed Trials

NCT06989632 — Total Lumbar Disc Prosthesis and Subsequent Work Activity at at Least Five Years After Total Lumbar Disc Replacement

StatusCompleted
PhaseN/A
SponsorUniversity of Valencia
Enrollment130
Study TypeOBSERVATIONAL
ConditionsLumbar Disc Degeneration; Lumbar Disc Disease; Sciatica; Lumbar Disc Herniation; Lumbar Degenerative Disease
Interventions

Lumbosciatica is a very prevalent pathology. When conservative treatments fail, surgery should be considered. The traditional surgical treatment is lumbar arthrodesis. The vast majority of patients who undergo spinal fusion cannot return to their same job and a good number of them never work again. Another form of treatment for lumbosciatica is the implantation of a lumbar disc prosthesis. This…

NCT01925196 — Natural History and Biomarkers of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia Caused by the C9ORF72 Gene Mutation

StatusCompleted
PhaseN/A
SponsorNational Institute of Neurological Disorders and Stroke (NINDS)
Enrollment50
Study TypeOBSERVATIONAL
ConditionsAmyotrophic Lateral Sclerosis; Frontotemporal Lobar Degeneration

Background:

- Some people have a mutation in the C9ORF72 gene that causes amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD). The mutation causes a small piece of DNA to repeat itself thousands of times. The C9ORF gene mutation mostly occurs in families. In those families, some persons have ALS and others have FTD. Occasionally the C9ORF gene mutation occurs in persons witho…

NCT02821780 — CADASIL Disease Discovery

StatusCompleted
PhaseN/A
SponsorNational Heart, Lung, and Blood Institute (NHLBI)
Enrollment20
Study TypeOBSERVATIONAL
ConditionsGermline Mutation in the NOTCH 3 Gene; Cardiovascular Disease; Arterial Stiffness; Pathogenesis of CADASIL; Clinical Phenotype of CADASIL

Cerebral autosomal dominant arteriopathy with subcortical infarct (CADASIL) is a lethal disease caused by a gene mutation that affects arteries in the brain. Symptoms include migraines, strokes, memory loss, and dementia. There are no treatments. Researchers want to study people who have CADASIL to learn more about it.

Objectives:

To learn more about CADASIL by studying people who have it.

E…

NCT04355689 — Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome

StatusCompleted
PhasePhase 1 / Phase 2
SponsorNacuity Pharmaceuticals, Inc.
Enrollment49
Study TypeINTERVENTIONAL
ConditionsUsher Syndromes
Interventions;

This study will examine the safety and efficacy of NPI-001 Tablets as compared to placebo for 24 months in subjects with vision loss due to RP associated with Usher syndrome.

NCT04863859 — Persons With Dementia and Their Extended Family Caregivers

StatusCompleted
PhaseN/A
SponsorKaren Roberto
Enrollment275
Study TypeOBSERVATIONAL
ConditionsDementia; Dementia Alzheimers; Neuro-Degenerative Disease; Dementia of Alzheimer Type; Dementia Severe

Immediate family members shoulder the majority of care responsibilities for persons living with dementia. However, due to various societal changes, elder care responsibilities have expanded to extended family members, including grandchildren, siblings, nieces/nephews, siblings, and step-kin.

The main objective of this study is to understand the caregiving journeys of various extended family me…