birth-defects Clinical Trials
A listing of birth-defects medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Found 283 clinical trials
Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparametric MRI
This study will evaluate whether cardiac MRI T1 and T2 mapping improves our ability to detect early abnormalities in the heart in patients with Fabry disease and identify patients at increase risk of adverse events.
Prevalence of Abnormalities in Ultrasonography of Joint and Tendons in Patients With Gout
Gout is the most frequent inflammatory arthritis in men after 40 years, with a prevalence of 0.9% in France. This disease is characterized by deposits of sodium urate crystals in joints, tendons and soft tissues, which can be detected by ultrasound examination. To date, there was no study assessing inflammatory, …
Early Detection of Developmental Abnormalities of Preterm Infants at 5 Years of Age
The project is a national, prospective, multicenter, interventional pilot project focused on the follow up of 5 year old children that were born prematurely. The primary aim of the project is to prepare, test, and develop a proposal for a national methodology for the care of children born prematurely. This …
Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: aSingle Centre, Randomized, Placebo-controlled Pilot Trial
The aim of this pilot phase trial is to assess the safety and tolerability, and estimate the efficacy of sirolimus in reducing the incidence of ICH during high-risk periods for rebleeding, compared to placebo. This pilot trial will inform the design of a future definitive clinical trial on sirolimus treatment …
Development of Preclinical Models Derived From Tumors With a Molecular Abnormality of Interest to Test Their Sensitivity to New Anti-tumor Therapies.
This study aims to collect tumor samples from patients carrying a mutation of interest to develop a cell culture technique for "spheroids. The goal is to use these spheroids to model responses to anticancer treatments. Various therapeutic molecules can be tested on these spheroids, enabling the evaluation of the potential …
Weekly Sirolimus Therapy
In current practice, options for venous and lymphatic malformations remain limited. Recently an oral medication, sirolimus, has been found to benefit patients when taken once or twice a day for several months. Unfortunately there are many side effects associated with this medication, some of which can be severe including, neutropenia, …
Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP)
This study is a two periods multi-center Phase II trial, with a 6 months double-blind, placebo-controlled period followed by open label period, to assess the efficacy and safety of alpelisib (BYL719) in pediatric and adult patients with Megalencephaly-CApillary malformation Polymicrogyria syndrome (MCAP)
Gadolinium and Ferumoxytol MRI in Diagnosing Patients With Abnormalities in the Central Nervous System
This phase II trial studies how well gadolinium and ferumoxytol magnetic resonance imaging (MRI) work in diagnosing patients with abnormalities in the central nervous system. Diagnostic procedures, such as gadolinium and ferumoxytol MRI, may help find and diagnose abnormalities in the central nervous system.
Predictors of Range of Motion and Balance Abnormalities in Children Diagnosed with Cerebral Palsy
To better understand the factors that contribute to ROM abnormalities in children diagnosed with CP. To describe the distribution of primary (muscle tone and postural stability)and secondary impairments (ROM and functional muscle strength) across the gross motor spectrum measured by GMFCS scores. To evaluate the effects of primary and secondary …
Cognitive-behavioral Therapy for the Diseases Associated With Esophageal-brain-gut Axis Communication Abnormalities.
Gastroesophageal reflux disease (GERD) is a challenging gastrointestinal disorder, and proton pump inhibitors (PPIs) are typically the first line of treatment. However, up to 40% of GERD patients experience little to no relief or only partial relief of their symptoms after receiving PPI therapy. Among these patients, who do not …
