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We list hundreds of Clinical Trials about "Pharma Supports 10th Annual Rare Disease Joins Global" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.
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This study is an observational (ie, non-interventional), longitudinal, multicenter, global registry for patients with pyruvate kinase (PK) deficiency, a rare non-spherocytic hemolytic anemia. This Registry will be open for enrollment for 7 years and all enrolled participants will be followed prospectively for a minimum of 2 years, and up to 9 years. Data will be collected from participating Registry Physicians, participants, and, where appropriate, parents/guardians who...
SPECTArare is a program aiming at establishing a quality assured platform for collecting clinicopathologically annotated biological material from patients with primary rare tumors (6 new cases/100,000/year) to support biospecimen-based translational research and biomarker discovery.
Minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), and Membranous nephropathy (MN), generate an enormous individual and societal financial burden, accounting for approximately 12% of prevalent end stage renal disease (ESRD) cases (2005) at an annual cost in the US of more than $3 billion. However, the clinical classification of these diseases is widely believed to be inadequate by the scientific community. Given the poor understanding of MCD/FSGS and MN b...
NYU Langone Health calls patients to remind them to schedule their annual well-health appointments (including annual well visit, mammograms, etc.). The proposed study will test different iterations of the call script with the goal of increasing call completion rate and the number of scheduled appointments within an appropriate timeframe.
Heart failure (HF) is a major health problem worldwide but there is no global HF study that documents demographics, socioeconomic and clinical factors, diagnostic and management patterns, etiology, biomarkers, co-morbidities, treatments, quality of life, barriers to care and outcomes in all parts of the world. Such knowledge is essential in the prevention and treatment of this global disease. The Global Congestive Heart Failure Registry (G-CHF) is a global registry of approxima...
Posner-Schlossman syndrome, or glaucomatocyclitic crisis, is a condition characterized by recurrent, acute attacks of mild, nongranulomatous, anterior uveitis accompanied by markedly elevated intraocular pressure. Most of patients are the 20 to 50-year-old young adults, with unilateral eye involvement. PSS was originally deemed benign, but is now recognized as a relatively rare cause of chronic secondary glaucoma,especially in patients with recurrent episodes. This relatively r...
Our knowledge on the rare type of pulmonary hypertension which can not be explained by left heart disease, respiratory disease or congenital heart disease is very limited. Investigators aim to setup a national registration study for the rare type of pulmonary hypertension, to understand the natural history, survival, progression, genetic and environmental contributions to disease.
The purpose of this study is to determine whether orthopedic spinal supports are effective in the treatment of low back pain.
The objective of this trial is to study the administration of the Menomune vaccine given intradermally and low-dose subcutaneously versus standard subcutaneously. This study will describe the immunogenicity of Menomune® - A/C/Y/W-135 administered subcutaneously (standard dose) versus intradermally over a dose range (1/10th, 2/10th, and 3/10th of standard dose) and a low dose (2/10th of standard dose) subcutaneously. The secondary objective is to describe the safety of the sub...
To show an increase in annual growth rate 3 years after Visit 2. Annual growth rate in standard deviation (SD) after 3 years will be compared to growth rate before the start of GH treatment.
Despite advances in the management of and changes in clinical practice, little is known about the epidemiology, patterns of care and outcomes of rare peritoneal surface malignancies patients in France. In order to better understand the characteristics of rare peritoneal surface malignancies and to evaluate treatment strategies, the RENAPE registry aims at the collection of data from patients presenting with a rare peritoneal surface malignancy in France. Data will be ent...
The project seeks to study the use of an navigator-based intervention called "PICU Supports." The study will test the impact of PICU Supports during and after PICU discharge on parent outcomes (satisfaction with decision making, decision regret, anxiety, depression, post-traumatic stress, health-related quality of life, and complicated bereavement) and on parent and healthcare team member assessments of communication and team collaboration.
Peritoneal mesothelioma is a rare disease representing one third of all mesothelioma and nothing is known about molecular characteristics of this disease. As main cancers, genetic heterogeneity is probable. This genomic profiling associates Comparative Genomic Hybridization (CGH) array, BAP1 sequencing and gene expression in order to discover a biomarker that could be used in the treatment of this rare disease. Corresponding histopathological and immunohistochemical report as a...
This study will help the investigator understand the pathogenesis of different rare genetic conditions and to establish database of rare genetic databases. This would ultimately help to provide more accurate diagnosis through advanced genomic diagnostic testing and databases established from this study. This knowledge would in turn help in the clinical management of other affected family members and other individuals affected with similar conditions. Understanding of pathogenes...
This is an observational, exposure-registration and follow-up study, to be conducted in the United States (US). The AMEVIVE® Pregnancy Exposure Registry is designed to monitor pregnant subjects and fetuses exposed to AMEVIVE® in order to detect any potential increase in the risk of major birth defects. The AMEVIVE® Pregnancy Exposure Registry is sponsored by Astellas Pharma Global Development and will be managed by Kendle International. The Registry will be monitored by a...
The standard or usual treatment for this disease may be chemotherapy or other types of treatment to slow the spread of the disease and relieve some symptoms of this cancer.
To demonstrate the relative bioavailability of Sandoz Inc. and UCB Pharma, Inc (Keppra) 750 mg Levetiracetam tablets in healthy adult volunteers under fasting conditions.
The DiRiP study will enroll patients (n = 3500) with unclear rare diseases and suspected genetic reasons. In group 1 (n = 500) subjects are clinically characterized in the context of outpatient/ inpatient standard care at the UKT or cooperating location, NGS analyzes and other omics analyzes (transcriptomics, proteomics, metabolomics), functional cell biology studies will be performed. In group 2 diagnostics is already performed.
The Global Program for the Elimination of Lymphatic Filariasis (GPELF) has been in operation sing the year 2000, with the aim of eliminating the disease by the year 2020, following 5-6 rounds of effective annual Mass Drug Administration (MDA). The treatment regimen is Ivermectin (IVM) in combination with Diethylcarbamazine (DEC) or Albendazole (ALB). In Ghana, MDA has been undertaken since 2001. While the disease has been eliminated in many areas, transmission has persisted in ...
The purpose of this study is to investigate clinical feasibility of annual non-contrast magnetic resonance imaging for surveillance of hepatocellular carcinoma in high-risk group, in comparison with biannual ultrasonography.
The Global Postural Re-Education is a global approach for the treatment of musculoskeletal disease. Using specific progressive posture for stretching particular shortened chain muscle the method wants to equilibrate the never-ending war between static and dynamic muscles by guiding the patient in a particular breathing pattern to maintain a correct posture and feeling it.
The investigators will investigate the usefulness of biannual ultrasonography versus annual non-contrast magnetic resonance imaging for surveillance of hepatocellular carcinoma in single arm patients.
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 ...
Fabry Disease (FD) is a rare, X-linked lysosomal storage disorder leading to left ventricular hypertrophy, myocardial fibrosis, arrhythmia and heart failure. Cardiac involvement is the leading cause of death in FD. Treatment with enzyme replacement therapy is expensive, may be poorly targeted and there are difficulties in early detection and disease monitoring. T1 mapping signal change is a potential remarkable biomarker for FD. Fabry400 is a multicentre study aiming to unders...
This study allows to evaluate the strength and duration of immune responses between annual receipt of standard inactivated vaccine and alternative potent vaccines, including annual receipt of adjuvanted inactivated vaccine, annual receipt of high-dose inactivated vaccine, annual receipt of recombinant HA vaccine, and the alternate combinations of the former three vaccines over four years, for identifying improved vaccination strategies for influenza vaccination in older adults ...