Clinical Trials About "Pharma Supports 10th Annual Rare Disease Joins Global" RSS

04:31 EST 17th December 2018 | BioPortfolio

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Showing "Pharma Supports 10th Annual Rare Disease Joins Global" Clinical Trials 1–25 of 18,000+


SPECTArare: Screening Patients With Rare Tumors for Efficient Clinical Trial Access

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.

Pyruvate Kinase Deficiency Global Longitudinal Registry

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...

Steady State Global Bioequivalence Study of Amphotericin B Liposome for Injection 50 mg/ Vial in Fed Condition

The primary objective is to determine clinical bioequivalence of Amphotericin B liposome for injection of Auromedics Pharma LLC, USA and AmBisome (Amphotericin B) liposome for injection of Astellas Pharma US, Inc., in patients with Visceral Leishmaniasis under fed condition

Nephrotic Syndrome Study Network

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...

Rifaximin to Modify the Disease Course in Sickle Cell Disease

In this single-arm, one-stage Phase II study, we hypothesize that gut decontamination with rifaximin will reduce the frequency of hospital admission due to painful crisis in patients with SCD. We will accrue 20 SCD patients who had at least two hospital admissions in the previous 12 months. These patients will receive rifaximin 550 mg twice a day for a total of 12 months. This following clinical parameters will be measured: 1. Changes in the annual rate of hospital admissions d...

Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children

The incidence of rare diseases is extremely low, the disease is numerous, the symptoms are serious, and the detection technology is complicated. Countries have different definitions of rare diseases. The definition of rare diseases in China is defined as: diseases with a prevalence of less than 1 in 500 000 or newborns with an incidence of less than 1/10 000 are rare diseases. Due to the low incidence of rare diseases and the accumulation of multiple organs and systems in most ...

Using Rapid Cycle Randomized Controlled Trials to Increase Patient Engagement in the Annual Well Visits Program

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.

Global Congestive Heart Failure Registry

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...

Patient Registry of Posner-Schlossman Syndrome

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...

Registration Study for Rare Type of Pulmonary Hypertension

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.

A Randomized Prospective Study to Evaluate the Effectiveness of Orthopedic Spinal Supports in the Treatment of Low Back Pain

The purpose of this study is to determine whether orthopedic spinal supports are effective in the treatment of low back pain.

Safety and Immunogenicity of Intradermal Versus Subcutaneous Doses of Menomune®

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...

Growth Retardation In Children With Special Pathological Conditions Or Disease

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.

French National Registry of Rare Peritoneal Surface Malignancies

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...

Improving Communication in the PICU: The Navigate Study

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.

Rivastigmine Bioequivalence Trial With Multiple Application of Transdermal Patches (9.5mg/24h)

The present clinical trial will be conducted in order to compare the bioavailability of rivastigmine and to assess bioequivalence at steady-state of the Test product RID-TDS 9.5 mg/24 h (Luye Pharma AG, Germany) and the marketed Reference product Exelon® 9.5 mg/24 h transdermales Pflaster (Novartis Pharma GmbH, Germany) after multiple patch application. Each of both treatments will last for 11 days with a washout period of 14 days between the treatments.

Genomic Analysis of Peritoneal Mesothelioma by CGH Arrays

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...

AMEVIVE® Pregnancy Registry

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...

To Demonstrate the Relative Bioavailability of Sandoz Inc. and UCB Pharma, Inc (Keppra) 750 mg Levetiracetam Tablets In Healthy Adults Volunteers Under Fasting Conditions

To demonstrate the relative bioavailability of Sandoz Inc. and UCB Pharma, Inc (Keppra) 750 mg Levetiracetam tablets in healthy adult volunteers under fasting conditions.

Finding Genes for Rare Diseases

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...

Durvalumab and Tremelimumab in Patients With Advanced Rare Tumours

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.

Twice Yearly Treatment for the Control of LF

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 ...

Diagnostic Research in Patients With Rare Diseases - Solving the Unsolved Rare Diseases

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.

Comparison of Biannual Ultrasonography and Annual Unenhanced Magnetic Resonance Imaging for HCC Surveillance

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.

Annual MRI Versus Biannual US for Surveillance of Hepatocellular Carcinoma in Liver Cirrhosis

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.

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