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We list hundreds of Clinical Trials about "JPM19 Spark looks scale manufacturing gene therapy matures" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.
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Advanced cancer patients have a poor quality of life and a high rate of unplanned hospital admissions. Palliative care has been shown to improve patient outcomes such as quality of life and symptom burden. Our long-term aim is to study the effect of Supportive and Palliative Care Review Kit (SPaRK) - a novel model of early palliative care in the acute hospital inpatient setting. To this end, we propose a pilot study that will provide the necessary information for the planning o...
Supportive care Prioritization, Assessment and Recommendations for Kids (SPARK) is a web-based application which builds upon the Symptom Screening in Pediatrics Tool (SSPedi) and consists of two components: (1) a symptom screening component centered on SSPedi; and (2) a supportive care clinical practice guideline (CPG) component. This proposal is a randomized controlled trial that compares daily completion of SSPedi via SPARK for 5 days with symptom feedback to healthcare provi...
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.
Introduction There is a rising need for palliative care services in Singapore due to a rapidly ageing population and an increasing incidence of cancer. Current existing resources are inadequate - novel models of care are needed to expand access to palliative care without requiring significantly more specialist palliative care manpower. Oncologist-driven referrals to a palliative care consultation service is the norm worldwide, including Singapore. This results in variab...
Children with cancer and hematopoietic stem cell transplant (HSCT) recipients suffer from severe and bothersome symptoms because treatments are intense. So, the investigators developed SPARK (Supportive care Prioritization, Assessment and Recommendations for Kids), a website devoted to helping children track symptoms and providing a way to let doctors and nurses know which symptoms are bothersome. This study will examine the likelihood that children will complete SPARK once dai...
The purpose of this research is to compare four different heart rate monitors (Apple Watch Nike III,Fitbit Iconic, Garmin Vivosmart HR, Tom Tom Spark 3) to an Electrocardiograph (ECG) machine to determine the accuracy of the devices. Over the last two decades, there has been a proliferation of commercially available heart rate monitors. Elite athletes often use heart rate measurements to monitor training and fitness levels. In response, fitness companies have offered a variety ...
Despite recommends that school-based interventions use a comprehensive approach for health promotions; most fitness programs exclusively emphasize physical activity. This study compared the comprehensive Translational Health in Nutrition and Kinesiology (THINK) program to a traditional YMCA program (The Sports, Play, and Active Recreation for Kids (SPARK) fitness curriculum) on measures of physical fitness, body composition, and executive cognitive function outcomes among 105 e...
The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe hemophilia A receiving prophylactic FVIII replacement therapy as standard of care. The data collected from this study may assist in providing baseline information for comparison to the Spark's investigational hemophilia A gene therapy in future Phase 3 studies.
RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system. This is a safety and dose ranging study to determine whether RGX-111 is safe and tolerated by patients with MPS I.
This is a Phase I/II clinical trial of gene therapy for treating X-linked adrenoleukodystrophy using a high-safety, high-efficiency, self-inactivating lentiviral vector TYF-ABCD1 to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.
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.
OBJECTIVES: I. Develop an approach for treating patients with homozygous familial hypercholesterolemia using gene therapy with autologous hepatocytes transduced with a normal low-density lipoprotein receptor gene.
RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a safety and dose ranging study to determine whether RGX-121 is safe and tolerated by patients with MPS II.
This study answers a U.S. Food and Drug Administration requirement for evaluation in people of a manufacturing lot of vaccine. Subjects receive one dose of one lot of vaccine. The antibodies in the blood measure the immunogenicity of the vaccine, and typical vaccine safety information is also collected.
This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s). The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.
In this self-control experiment, anesthesiologists without experiment of bronchoscopy working in anesthesiology department in Peking Union Medical College Hospital will receive a training and evaluation procedure in simulation and clinical practice about clinical airway management. The objects was (1) to investigate the improvements of manufacturing bronchoscopy in simulation and clinical practice before and after training on simulation, (2) to record the study curve and effort...
The purpose of this study is to assess the safety,tolerability and immunogenicity of manufacturing scale 13-valent pneumococcal conjugate(13vPnC) vaccine compared to pilot scale 13vPnC in healthy infants when given with routine paediatric vaccines
RATIONALE: Inserting the gene for adenovirus p53 into a person's tumor may improve the body's ability to fight cancer. PURPOSE: Phase I trial to study the effectiveness of gene therapy in treating patients who have recurrent malignant gliomas.
RATIONALE: Inserting the gene for p53 into a person's cancer cells may improve the body's ability to fight cancer. Radiation therapy uses high-energy x-rays to damage tumor cells. PURPOSE: Phase I trial to study the effectiveness of gene therapy plus radiation therapy in treating patients who have non-small cell lung cancer.
The aim of this study is to determine possible molecular changes on large scale gene expression profiling after treatment with Metvix photodynamic therapy (PDT) of actinic keratoses (AK) and cancerised field in renal transplant recipients.
This is a Phase I/II clinical trial of gene therapy for X-linked severe combined immunodeficiency (SCID-X1) using a self lentiviral vector to replace the defective genes with good genes. The primary objectives are to evaluate the safety and efficacy of the ex vivo gene therapy clinical protocol.
RATIONALE: Inserting the p53 gene into a person's cancer cells may improve the body's ability to fight cancer or make the cancer more sensitive to chemotherapy. Combining chemotherapy with gene therapy may kill more tumor cells. PURPOSE: Phase I trial to study the effectiveness of gene therapy plus chemotherapy in treating patients who have breast cancer.
This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.
RATIONALE: Inserting the p53 gene into a person's bladder cancer cells may improve the body's ability to fight cancer. PURPOSE: Phase I trial to study the effectiveness of gene therapy in treating patients with advanced bladder cancer.
Patients with diabetes may develop chronic wounds that respond poorly to treatment. Gene therapy with the platelet-derived growth factor-B gene has been shown to help with the healing of chronic wounds. This study will evaluate a new way to deliver the gene to the wound tissue.