Bala-Cynwyd, Pennsylvania Clinical Trials
A listing of Bala-Cynwyd, Pennsylvania clinical trials actively recruiting patient volunteers.
Found 1,162 clinical trials
Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants
This is an international, multicenter study with two components: Registry A standardized genetic screening and a prospective, standardized, cross-sectional clinical data collection Enrollment is open to all genes on the RD Rare Gene List Natural History Study A prospective, standardized, longitudinal Natural History Study Enrollment opens gene-by-gene, based on funding …
Improving Safety and Quality of Tracheal Intubation Practice in Pediatric ICUs
Advanced airway interventions are common high risk, high stakes events for children in intensive care units (ICU) and emergency departments (ED), with risk for life and health threatening consequences.
Mitochondrial Myopathy Rating Scale
The goal of this observational study is to develop and validate tools to measure disease course in patients with primary mitochondrial myopathy (PMM). The main aims of this study are Development, validation, and optimization of objective outcome measures for mitochondrial myopathy Defining the natural history of mitochondrial myopathy Researchers will …
Outcomes and Health Care Resource Utilization in Pediatric Congenital Heart Disease Patients Undergoing Non-Cardiac Procedures
The incidence of moderate to severe congenital heart disease (CHD) in the United States is estimated to be 6 per 1000 live-born full term infants. Recent advances in pediatric cardiology, surgery and critical care have significantly improved the survival rates of patients with CHD leading to an increase in prevalence …
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
This global patient registry is being conducted to characterize the natural history and real-world clinical management of patients with AHP, and to further characterize the real-world safety and effectiveness of givosiran and other approved AHP therapies.
Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others
This is an observational longitudinal study to determine the natural history and genotype-phenotype correlations of disease causing mutations in Charcot Marie Tooth disease (CMT) type 1B (CMT1B), 2A (CMT2A), 4A (CMT4A), and 4C (CMT4C). The investigators will also be determine the capability of the newly developed CMT Pediatric Scale (CMT …
Long-term Safety of Nipple Sparing Mastectomy in Women With GPV in Breast Cancer
Patients with a germline pathogenic variant (GPV) in high-penetrance breast cancer susceptibility genes who are considering risk reducing mastectomy (RRM) often strongly desire to keep their nipple areola complex but inquire as to whether it is safe to do so. Relative to traditional or skin sparing mastectomy (SSM) techniques, nipple …
Non-Invasive Diagnosis of Pediatric Pulmonary Invasive Mold Infections
This study will establish a non-invasive diagnostic approach and evaluate clinical outcomes for children at high-risk for pulmonary invasive fungal infection (PIFI).
Reducing Patient Memory Recall in the Burning Mouth Patient Population
The goal of this study is to learn about burning mouth syndrome symptoms in real time in patients with burning mouth syndrome. The main questions it aims to answer are: (1) To test the ability of a smartphone app to collect repeated observations of individual data to assess fluctuations in …
Evaluation of Outcome Metrics in Alexander Disease
The purpose of this study is to define the natural history of Alexander Disease, a leukodystrophy that causes neurological dysfunction. Investigators will obtain clinical outcome assessments to measure how the disease affects a patient's gross motor, fine motor, speech and language function, swallowing, and quality of life. Specimens are collected …
