Clinical Trials About "Prevail licenses REGENXBIO AAV9 gene delivery technology neurodegenerative" RSS

11:44 EST 20th November 2019 | BioPortfolio

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Showing "Prevail licenses REGENXBIO AAV9 gene delivery technology neurodegenerative" Clinical Trials 1–25 of 9,100+

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Gene Transfer Study of AAV9-CLN3 for Treatment NCL Type 3

Open-label, single dose, dose-escalation clinical trial AAV9-CLN3 via intrathecal injection in NCL type 3 subjects

Gene Therapy for Male Patients With Danon Disease Using RP-A501; AAV9.LAMP2B

This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene (investigational product (IP), RP-A501) in male patients with Danon Disease (DD).


Intravenous Gene Transfer With an AAV9 Vector Expressing Human <=-Galactosidase in Type II GM1 Gangliosidosis

Background: GM1 gangliosidosis is a disorder that destroys nerve cells. It is fatal. There is no treatment. People with GM1 are deficient in a certain enzyme. A gene therapy may help the body make this enzyme. This could improve GM1 symptoms. Objective: To test if a gene therapy helps type II GM1 gangliosidosis symptoms. Eligibility: People ages 2 12 with type II GM1 gangliosidosis Design: Participants will be screened with their medical history a...

Batten CLN6 Gene Therapy

The proposed clinical trial is the first human, open-label, single dose study of self-complementary AAV9 carrying the CLN6 gene delivered one-time intrathecally inserted by a lumbar puncture into Batten CLN6 Disease subjects. One cohort of patients with Batten CLN6 Disease, with confirmed diagnosis, will undergo gene transfer. A minimum of six patients will be enrolled into the cohort.

A Study of Certain Prevail Implants Used for the Preservation of Crestal Bone in Short Fixed Bridge Cases.

This prospective, randomized study will evaluate the performance of the Osseotite Certain Prevail implant when compared to that of the non-lateralized version of the implant. Study (null) hypothesis: the crestal bone changes that take place after placement and loading of the Osseotite lateralized implants will be the same as for a similar but non-lateralized Osseotite Certain implant.

A Study of the Performance of Certain Prevail Implants Used in Short Fixed Cases

This prospective, randomized study evaluates the performance and crestal bone changes difference between the Osseotite Certain Prevail vs the non-lateralized design of the implant. Study (null) hypothesis: the crestal bone changes that take place after placement and loading of the Osseotite lateralized implants will be the same as for similar but non-lateralized Osseotite Certain implants.

Evaluation of Safety and Tolerability of Libella Gene Therapy for Alzheimer's Disease: AAV- hTERT

Using gene therapy to express active telomerase (hTERT) in human cells has the potential to treat many neurodegenerative diseases related to aging, including Alzheimer's disease (AD). This study will entail treating subjects with hTERT delivered via transduction using AAV. The goal is to extend the telomeres to prevent, delay, or even reverse the development of the pathology of AD. This will have a direct consequence on cognitive function and quality of life in patients with n...

18F-S16 PET/CT in Healthy Volunteers and Patients With Neurodegenerative Dementia

This is an open-label dynamic whole-body PET/CT (positron emission tomography/computed tomography) study for investigation of radiation dosimetry, plasma pharmacokinetics, biodistribution, safety and diagnostic performance of 18F-S16 in healthy volunteers and patients with neurodegenerative dementia.

A Study of Osseotite Certain Prevail Implants Used to Support Short Fixed Bridges With Immediate Occlusal Loading.

This prospective, multicenter study evaluates the performance of the Osseotite Prevail implant when to support a short fixed bridge with immediate occlusal loading, providing the patient with the use of a temporary prosthesis until the final prosthesis is made. Study (null) hypothesis: This is an observational study in which no concurrent control group will be observed. Results of the study will be compared with the performance of other Osseotite implants from published Osseo...

Gene Therapy for the Treatment of Brain Tumors

Malignant brain tumors are responsible for a significant amount of deaths in children and adults. Even with advances in surgery, radiation therapy, and chemotherapy, many patients diagnosed with a malignant brain tumor survive only months to weeks. In an attempt to improve the prognosis for these patients, researchers have developed a new approach to brain tumor therapy. This approach makes use of DNA technology to transfer genes sensitive to therapy into the cells of the tu...

DaTSCAN Imaging in Aging and Neurodegenerative Disease

The investigators propose using DaTscan in patients with REM sleep behavior disorder (RBD), mild cognitive impairment (MCI), Parkinson's disease (PD), dementia with Lewy bodies (DLB), Alzheimer's disease (AD), and other neurodegenerative syndromes and disorders, to test several hypotheses - some confirmatory, and some novel. Such use will provide new data on the potential clinical and research utility of DaTscan in neurodegenerative diseases. The findings on DaTscan will be cor...

The Ontario Neurodegenerative Disease Research Initiative

The Ontario Neurodegenerative Disease Research Initiative (ONDRI) is a province-wide collaboration studying dementia and how to improve the diagnosis and treatment of neurodegenerative diseases including: - Alzheimer's disease (AD) - Parkinson's disease (PD) - amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) - frontotemporal lobar degeneration (FTD) - vascular cognitive impairment, resulting from stroke (VCI)

Concurrent Single Gene and 24 Chromosome Aneuploidy Preimplantation Genetic Diagnosis (PGD)

Gene Security Network has developed a novel technology called Parental SupportTM (PS) which is used for Preimplantation Genetic Screening/Diagnosis (PGS/D) during in vitro fertilization (IVF). This technology allows IVF physicians to identify embryos, prior to transfer to the uterus, which have the best chance of developing into healthy children. The purpose of this study is to validate clinical use of PS to detect specific genetic mutation(s) known to cause severe inheritable ...

Biomarkers in Neurodegenerative Diseases

The general purpose of this observational study is to examine biomarkers associated with the pathology of neurodegenerative diseases to potentially develop novel therapeutic approaches.

Peer Mentorship to Reduce Suicide Attempts Among High-Risk Adults

Amid consistently worsening suicide rates, in 2012 the U.S.Surgeon General's National Strategy for Suicide Prevention proposed to "change the narrative" about suicide prevention to include a focus on promoting hope and belongingness. Despite hopelessness and thwarted belongingness being among the most replicated risk factors for suicide, many widely implemented suicide prevention efforts instead emphasize the identification of acute suicide risk and referral to mental health tr...

Lentiviral Gene Therapy for MLD

This is a Phase I/II clinical trial of gene transfer for treating Metachromatic leukodystrophy (MLD) using a safety and efficiency improved self-inactivating lentiviral vector TYF-ARSA to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.

Study of Neurodegenerative Diseases Induced Stem Cells in Patients and Healthy Family Controls.

Neurological and neurodegenerative diseases have a major impact in families and in the national health service due to the lack in many cases of effective and long-lasting therapies. The lack of these therapeutic strategies is due in large part to the difficulty of modeling these pathologies in vitro. In fact, the impossibility of being able to cultivate human neurons in vitro has forced the use of animal cell models that do not adequately recapitulate the complexity of these hu...

Safety and Efficacy of Targeted Gene Transfer in Colorectal Cancer Metastatic to Liver

This is a Phase I safety study of a gene transfer drug for colorectal cancer that has spread to the liver. The main purpose of this study is to determine if it is safe to give this new intervention to persons with cancer, but we will also look for indications that the drug is effective. Although the findings in animals that have cancer are encouraging, this is the first time humans will receive this experimental gene transfer drug. A gene called cyclin G1 has been shown to pla...

Assessment of the Ability to Distinguish Odors in Glaucoma Patients

Glaucoma is one of the leading causes of blindness worldwide. The key feature of glaucoma is damage to the optic nerve head with loss of retinal ganglion cells and local tissue remodeling. Neuronal cell death in glaucoma occurs by apoptotic mechanisms1. Apoptosis of neurons also plays a major role in neurodegenerative diseases, such as Alzheimer's or Parkinson's disease. An association between glaucoma and these disorders has been described 2-4. Furthermore, it has been reporte...

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

The aim of this study is to treat the signs and symptoms of late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal inherited disease in the brain. This will be accomplished by using delivery of a gene (method called gene transfer) to administer to the brain an experimental drug called AAV2CUhCLN2, a gene transfer vector.

Longitudinal Study of Neurodegenerative Disorders

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

Extension Study of Pimavanserin in Adult Subjects With Neuropsychiatric Symptoms Related to Neurodegenerative Disease

The purpose of this study is to evaluate the long-term safety and tolerability of pimavanserin in adult and elderly subjects with neuropsychiatric symptoms related to neurodegenerative disease exposed to open-label pimavanserin for up to 52 weeks.

Gene Expression In Pregnancies Complicated by Preeclampsia

The purpose of this study is to figure out some of the differences in bodily function between women who have preeclampsia and those who do not. - This may eventually lead to an understanding of its cause. At this time, there is no known way to prevent preeclampsia, and the cause is not known. - The only treatment is delivery of the baby, even if it is premature, in order to decrease the risk to the mother.

PartoSure to Predict Spontaneous Preterm Delivery in Pregnancies After Assisted Reproductive Technology (ART)

To calculate sensitivity (SN), specificity (SP), positive predictive value (PPV) and negative predictive value (NPV) of PartoSure TTD test in predicting delivery within 7 or 14 days from testing and to compare it with cervical length (CL) measurement by transvaginal ultrasound (15 mm cut-off)

Study of Intra-articular Delivery of tgAAC94 in Inflammatory Arthritis Subjects

The 13G01 clinical trial is a Phase I/II dose escalation study designed to be conducted in adults with inflammatory arthritis who have persistent moderate or severe swelling in one or more joints, without a disease severe enough to warrant a change in regimen for the next three months. The study will permit subjects who are concurrently on anti-tumor necrosis factor (TNF)-alpha antagonists. For subjects on disease modifying antirheumatic drugs (DMARDs), a stable regimen...

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