Clinical Trials About "Rescuing failing heart targeted gene transfer" RSS

11:14 EST 19th November 2017 | BioPortfolio

We list hundreds of Clinical Trials about "Rescuing failing heart targeted gene transfer" on BioPortfolio. We draw our references from global clinical trials data listed on and refresh our database daily.

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Showing "Rescuing failing heart targeted gene transfer" Clinical Trials 1–25 of 9,200+

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SERCA Gene Therapy Trial

The aim of the study is to determine the safety and feasibility of giving an adeno-associated viral vector expressing the sarcoplasmic reticulum calcium ATPase (SERCA2a), driven by the CMV promoter (AAV6-CMV-SERCA2a), to heart failure patients that have received a left ventricular assist device (LVAD) for an accepted clinical indication. It is a randomised, double-blind study of 16 patients that will be randomised to receive either the study drug (AAV6.SERCA2a) or placebo. ...

AC6 Gene Transfer for Congestive Heart Failure

Dr. H. Kirk Hammond and associates are conducting a research study to find out 1) whether gene transfer using an agent called Ad5.hAC6 (adenovirus-5 encoding human adenylyl cyclase type 6) can be given safely to patients with congestive heart failure (CHF) and 2) whether this agent may be of benefit in heart failure. Gene transfer is an idea in which genes are introduced into cells and the cells then produce the specific protein that the gene directs, in this case, a protein kn...

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...

Gene Transfer for Patients With Sickle Cell Disease

The purpose of this study is to determine whether transfer of a fetal hemoglobin gene using a lentivirus vector (gene transfer) into human blood making cells is safe and feasible in patients with sickle cell disease.


Long-Term Follow-Up of Recipient of Gene Transfer Research

The goal of this clinical research study is to collect information about the long-term safety of gene transfer therapy. This study is also designed to create a database of medical information about patients who have received gene transfer therapy, in order for researchers to track and review the long-term safety and any effects (good or bad) of gene transfer therapy.

Efficacy and Safety Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure

The study is divided into 2 parts. In the first part, the safety of the gene transfer agent MYDICAR® will be evaluated. In the second part, the ability of MYDICAR® to improve heart function will be studied.

LTFU for Gene Transfer Subjects With Hemophilia B

Several subjects enrolled in a multi-site, gene transfer clinical study to evaluate the intrahepatic administration of AAV2-hFIX16 vector for the treatment of severe hemophilia B between 2001 and 2009. As the US FDA has established guidelines for the long-term follow-up (LTFU) of subjects receiving investigational gene therapy products, this protocol seeks to characterize the clinical outcome and the type and seriousness of adverse events following the AAV gene transfer. The pr...

EndocardialVascularEndothelialGrowth Factor D(VEGF-D)Gene Therapy for the Treatment of Severe Coronary Heart Disease

The purpose of the study is to evaluate the safety and efficacy of catheter mediated endocardial adenovirus VEGF-D gene therapy in patients with severe coronary heart disease.

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.

Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis

OBJECTIVES: I. Determine the maximum tolerated dose of recombinant adeno-associated virus-CFTR vector in patients with cystic fibrosis. II. Assess the safety of this gene therapy in these patients. III. Assess the in vivo gene transfer of this vector in these patients. IV. Assess the CFTR gene expression and physiologic activity following gene transfer in these patients. V. Assess the clinical impact of CFTR gene expression following gene transfer in thes...

Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector

Researchers are working on ways to treat SCID patients who don't have a matched brother or sister. One of the goals is to avoid the problems that happen with stem cell transplant from parents and unrelated people, such as repeat transplants, incomplete cure of the immune system, exposure to chemotherapy, and graft versus host disease. The idea behind gene transfer is to replace the broken gene by putting a piece of genetic material (DNA) that has the normal gene into the chil...

A Gene Transfer Study for Hemophilia A

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.

Stem Cell Mobilization and VEGF Gene Transfer for Heart Failure

The purpose of this study is to evaluate the safety and bioactivity of intramyocardial gene transfer using VEGF (vascular endothelial growth factor) in patients with ischemic heart failure. The research treatment of this CHF study will involve the use of an intramuscular injection with a catheter inserted in through the groin to transfer a type of DNA (phVEGF165 gene) to the wall of the heart. Genes (which are part of the DNA molecules) carry instructions to allow the cells to ...

Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B

The purpose of this study is to determine the safety of giving a normal factor IX gene to treat individuals who have an abnormal or no factor IX gene. Recruitment will be limited to adults (≥ 18 years) with a confirmed diagnosis of hemophilia B (HB), resulting from a missense mutation in the coagulation factor IX (FIX) gene or a nonsense mutation that has not been associated with an inhibitor.Only subjects who have no evidence of active hepatitis or anti-hFIX antibodies, and ...

Safety Study of hMaxi-K Gene Transfer to Treat Overactive Bladder Syndrome and Detrusor Overactivity

The purpose of this study is to evaluate the safety of a new product that uses human gene transfer (called hMaxi-K) when it is given to patients with overactive bladder. Human gene transfer is a new type of therapy that is the process of placing genetic material (DNA or RNA) into a person. The primary objective of this study is to evaluate safety parameters occurring subsequent to administration of a single intravesical instillation of study drug. Three different dose group...

Canadian Profiling and Targeted Agent Utilization Trial (CAPTUR)

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.

CD19+ CAR T Cells for Lymphoid Malignancies

Sometimes researchers change the DNA (genetic material in cells) of donated T-cells (white blood cells that support the immune system) using a process called "gene transfer." Gene transfer involves drawing blood from a transplant donor (or the patient him/herself), and then separating out the T-cells using a machine. Researchers then perform a gene transfer to change the T-cells' DNA, and then inject the changed T-cells into the body of the patient receiving the transplant. ...

Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector

This is a clinical gene transfer study that aims to verify the safety and efficacy of the use of the EFS-ADA lentiviral vector to introduce the human adenosine deaminase (ADA) gene into the hematopoietic progenitors of patients affected with severe combined immunodeficiency due to ADA deficiency. The EFS-ADA vector expresses the human ADA cDNA under the control of the elongation factor alpha short promoter (EFS). In addition, this protocol will examine the effects of the ADA ge...

Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia

Fanconi anemia (FA) is a disease that affects an individual's bone marrow. It is caused by a defective gene in the bone marrow cells that produce various types of blood cells. Individuals with FA may experience fatigue, bleeding, and increased infections. The purpose of this study is to evaluate the safety and effectiveness of a gene transfer procedure in generating new, healthy cells in individuals with FA.

Evaluation of Safety of Rexin-G Gene Transfer for Advanced Pancreatic Cancer

This is a dose-seeking study that will test the safety of increasing doses of Rexin-G, given intravenously, in patients with advanced or metastatic pancreatic cancer who have failed standard chemotherapy. Rexin-G is a tumor-targeted gene therapy vector that contains a "killer" gene that blocks the action of the human cyclin G1 gene. Cyclin G1 is a cell cycle control element that plays an important role in cancer growth. When injected into a vein, the Rexin-GTM vector seeks ou...

Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency

Individuals with a deficiency of the Alpha 1-antitrypsin (AAT) protein are at risk for developing emphysema and liver damage. Researchers have developed a way to introduce normal AAT genes into muscle cells so that the AAT protein is produced at normal levels. This study will evaluate the safety of the experimental gene transfer procedure in individuals with AAT deficiency.

Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency

The purpose of this study is to evaluate the safety and effectiveness of lentiviral gene transfer treatment at restoring immune function to participants with X-linked severe combined immunodeficiency (XSCID) who are 2 to 40 years of age, and have significant impairment of immunity.

Ventricular Arrhythmias in Uremic Cardiomyopathy

There is a certain gene called sarcoplasmic reticulum gene (SERCA2a), which is found in heart muscle. This gene is also found in blood vessels and skin tissue. When active this gene builds a crucial protein inside the heart muscle called SERCA2a protein. This is responsible for regulating calcium levels inside your heart muscle. When this gene is not activated, studies have shown that it can lead to abnormal electrical currents in the heart that can lead to death. The investiga...

Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B

In this study a modified virus called adeno-associated virus (AAV) will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B (AAV human Factor IX vector). Gene therapy is a very new medical technique being used in a number of clinical studies for diseases such as cancer and cystic fibrosis. At this time, the U.S. Food and Drug Administration has approved no gene transfer products for commercial use. To date, 8 subjects have re...

Wild Type p53 Adenovirus for Oral Premalignancies

Primary Objectives: 1. To determine the maximum tolerated dose and transduction efficiency of adenoviral mediated wild type p53 gene transfer in premalignancies of the upper aerodigestive tract. 2. To determine the efficacy of single agent adenoviral mediated wild type p53 gene transfer in reversing oral premalignancies.

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