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We list hundreds of Clinical Trials about "Frontier Pharma Ophthalmology Therapeutics Gene Therapies FirstinClass Targets" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.
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The IRIS™ Registry (Intelligent Research in Sight) is the nation's first comprehensive eye disease clinical registry. The American Academy of Ophthalmology is developing it as part of the profession's shared goal of continual improvement in the delivery of eye care.The IRIS Registry will be a centralized system for ophthalmology practices to promote practice innovations and achieve clinical excellence.
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.
Cancer drugs which target the effects of abnormal gene changes are called 'targeted therapies'. This study, called PM.1 or CAPTUR, will include some targeted therapies that are currently available. The purpose of this study is to find out what are the effects on a patient and their cancer when they are given a targeted therapy drug that is specific to an abnormal gene change in their cancer.
The purpose of this research is to determine if Ibis™, a digital therapeutics solution developed by Senscio Systems, reduces the emergency room visits and hospitalizations of patients with COPD.
The "dry eye syndrome" DES is a highly prevalent ocular disease, in particular in the elderly population. One mainstay of therapy for patients suffering from DES is the use of topically administered lubricants. However, despite many efforts, no "ideal" formulation has yet been found. Recently, Croma Pharma has introduced chitosan-N-acetylcysteine eye drops, designed for treatment of symptoms related to DES. Chemically, chitosan is a polycationic biopolymer with favourabl...
Assess the effect on coronary atheroma of serial infusions of autologous selectively delipidated HDL/preβ enriched plasma following use of HDL Therapeutics PDS-2™ System
In this study, DNA sequencing, computational biology modeling, and ex vivo drug sensitivity assays will be utilized to define clinically relevant gene mutations and identify potential therapeutics for patients with acute myeloid leukemia (AML).
Over the last 10 years, technological advances in molecular biology enabled a more accurate genomic characterization of tumors. For each tumor location, this led to the identification of subgroups with similar molecular characteristics. This identification allowed the development of targeted therapies and thus to improve the patient prognosis. This molecular characterization has also revealed the tumor heterogeneity. It may be the cause of treatment resistance and therefore of ...
The objective of our study will be to analyze the interplay between particular VDR variants and its ligand in cells obtained from intervertebral disc and cartilage endplate and their action on cell proliferation and phenotype maintenance. The identification of novel molecular targets in the vitamin D pathway, potentially promoting pathological alterations of the tissues involved in spinal disc diseases, will allow to develop innovative and more personalized preventive and pharm...
To demonstrate the relative bioavailability of Sandoz Inc. and UCB Pharma, Inc (Keppra) 750 mg Levetiracetam tablets in healthy adult volunteers under fasting conditions.
Gene and cell therapies have a real value potential (example: potential long-term effect, curative potential, and single administration) but also drawbacks (example: treatment perceived as complex and risky generating fear and anxiety in subjects and their family; high cost) that require significant educational efforts and planning for optimal market access. It is therefore important to understand subject and physician perceptions of T-Cell Receptor (TCR) therapies. The aim of ...
RATIONALE: Biological therapies, such as anakinra, may interfere with the growth of the tumor cells and slow the growth of metastatic tumors that express the interleukin-1 gene. PURPOSE: This phase I trial is studying the side effects and best dose of anakinra in treating patients with metastatic cancer that expresses the interleukin-1 gene.
Endolymphatic Sac Tumors in a Population of Patients With Von Hippel-Lindau Disease:The Natural History and Pathobiology, and a Prospective Non-Randomized Clinical Trial of Hearing Preservation Surgery in Patients With Early Stage Endolymphatic Sac Tumors
The von Hippel Lindau (VHL) gene has recently been identified as the genetic defect resulting in a syndrome of multiple neoplasias. Patients with VHL disease develop retinal angiomata, renal cysts and/or carcinomas, CNS hemangioblastomas as well as pancreatic cysts and pheochromocytomas. Investigators have shown the gene to be a tumor suppressor type proto-oncogene located at chromosomal locus 3p26. The gene includes three exons whose gene product targets a cellular transcri...
The purpose of this study is to evaluate the safety, effectiveness, and tolerability, of the Pharma-Pen(TM) intramuscular (IM) and subcutaneous (SC) auto-injector systems used by untrained users to self-administer Influenza vaccines as compared to standard IM injections administered by a licensed healthcare professional (e.g. a nurse). Participants will be 120 healthy men and women aged 18 - 50 years who have not received influenza vaccine for at least four years and who have ...
Comparative randomized, single dose, two-way crossover open-label study to determine the bioequivalence of Cefadroxil from Duricef 1 gm Film Coated tablets(Smthkline Beecham Egypt,LLC affiliated co. to GalaxoSmithKline ) and Biodroxil 1 gm Film Coated tablets (Kahira Pharm &Chem .Ind. Co . for Novartis Pharma ) after a single oral dose administration of each to healthy adults under fasting conditions.
The purpose of this study is to assess the clinical anti-proliferative activity of STI571 (Glivec®, Novartis, Pharma) in patients with HES defined as: 1. Idiopathic Hypereosinophilic Syndrome (secondary HES), defined as a peripheral blood eosinophilia greater than 1,500 cells/µL for longer than 6 months, absence of other apparent aetiologies for eosinophilia and with or without signs and symptoms of organ involvement, irrespective to expression of any of ima...
Asthma and COPD have a significant impact on public health, affecting about 8 million people in France and generating health care costs of 5.5 billion euros, almost 50% of which are dedicated to long-term treatments are essentially inhaled therapies. The good control of the disease depends on the patient's compliance, but also on the proper use of the devices used for the administration of inhaled drugs under penalty of the degradation of the control of the disease with major ...
The P-glycoprotein (P-gp) is a membranous transporter that modulates the intracellular concentrations of many drugs and plays thus a major role in the efficacy of the therapeutics that act within the lymphocytes, such as antiretroviral drugs. We aim at studying the evolution of this transporter's expression and activity on lymphocytes in relation with the human development from newborns to adults. We also aim at studying the influence of HIV and antiretroviral treatments on thi...
Primary mucosal melanomas (MPM) are rarer than cutaneous melanomas, but also more severe. They are usually refractory to conventional approaches, regularly excluded from major therapeutic programs and not sensitive to new targeted therapies orphans. " The identification of therapeutic targets and accessibility to existing, developing or future targeted therapies improves the survival of patients with MPM. The principal goal is to describe, using a large panel of genes, the pre...
This study will look for new types of gene changes that may be related to cancer in some patients. Some gene changes (mutations) are passed on from parents to offspring (child). Other gene changes are new and are seen for the first time in a child. They are not seen in the parent. Some of these gene changes may cause cancers in the offspring. We will look for gene changes by studying patients with cancer their parents and family members without cancer. In this study, we ...
The purpose of this first-in-human study of CX-072 is to characterize the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and antitumor activity of CX-072 administered intravenously (IV) as a single agent or in combination with ipilimumab or vemurafenib in adult subjects with advanced or recurrent solid tumors or lymphomas. PROCLAIM-001: PRObody CLinical Assessment In Man CX-072 clinical trial 001 CX-072 is a Probody™ therapeutic directed against PD-L1 (pr...
The prevention and treatment of diseases via artificial intelligence represents an ultimate goal in computational medicine. Application scenarios of the current medical algorithms are too simple to be generally applied to real-world complex clinical settings. Here, the investigators use "deep learning" and "visionome technique", an novel annotation method for artificial intelligence in medical, to create an automatic detection and classification system for four key clinical sce...
Comparative randomized, single dose, three way three sequence two treatment partial replicate crossover open-label study to determine the bioequivalence of Sofosbuvir from Sofodelevier 400 mg F.C.T (Al-debeiky pharma, Egypt) versus Sovaldi 400 mg Tablets (Gilead Sciences, Canada)
Polypoidal choriodal vasculopathy (PCV) is an ophthalmologic disease, characterized by vascular abnormalities of the walls of small choroidal vessels, reproducing the specific aspect of polyps (cluster aspect). PCV is one of the "boundary-forms" of age related macular degeneration. These vasculopathies can be idiopathic. Following the radiotherapy treatments of active and occult-typed neovessels in Age-Related Macular Degeneration (ARMD), 10% of the patients would presen...
RATIONALE: Inserting the p53 gene into a person's tumor may improve the body's ability to fight liver cancer. PURPOSE: Phase I trial to study the effectiveness of gene therapy with the p53 gene in treating patients who have cancer of the liver that cannot be surgically removed.