Found 2,070 clinical trials
Pyruvate Kinase Deficiency Global Longitudinal Registry
This study is an observational (ie, noninterventional), longitudinal, multicenter, global registry for patients with pyruvate kinase (PK) deficiency, a rare nonspherocytic 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 …
LOWER: Lomitapide Observational Worldwide Evaluation Registry
This global product exposure registry is a multicentre, long-term, prospective, observational cohort study (exposure registry), designed to evaluate the long term safety and effectiveness of lomitapide.
The True Vie I3 Continuous Glucose Monitoring System in Pediatric Patients With Type 1 Diabetes Mellitus
The purpose of the investigation is to evaluate the performance of the I3 CGM in Pediatric Patients with Type 1 Diabetes Mellitus (T1DM) according to the FDA's special controls for iCGM.
Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
Phenylketonuria (PKU) is an inherited metabolic disorder that impairs the metabolism of the essential amino acid phenylalanine (Phe). Without stringent dietary control, Phe accumulates in the blood and brain of PKU patients, leading to severe cognitive deficits. Achieving metabolic control, defined as blood Phe levels within the range of 120-360 …
Drug Screening System Method Comparison
Prospective, open-label, single-dose, randomized, multi-site performance evaluation of the accuracy and usability Intelligent Fingerprinting Drug Screening System in the hands of intended users: "operators" and "donors."
The Tether™ - Vertebral Body Tethering System Post Approval Study
This study is an opportunity to provide continued reasonable assurance of the safety and probable benefit of The Tether HUD. The study will collect long term safety and efficacy information from patients who have had their idiopathic scoliosis treated via anterior vertebral body tethering (AVBT) with The Tether.
Evaluation of the MuSICCA for Measuring Awareness
Prolonged Disorders Of Consciousness (PDOC) include the Vegetative State (VS) and the Minimally Conscious State (MCS) that persist for longer than four weeks. This research seeks to evaluate the validity (accuracy), reliability, and clinical utility (usefulness to clinicians) of the children's version of the Music Therapy Assessment Tool for Awareness …
Clinical Data Registry of Amblyopia Patients on Luminopia Treatment
Amblyopia is the most prevalent cause of reduced monocular visual acuity in children and young adults, with estimates of prevalence ranging from 1% to 5%. The most common associated amblyogenic risk factors are uncorrected anisometropia, strabismus, or a combination of these. In addition to reduced visual acuity, amblyopic patients may …
Angelman Syndrome Natural History Study
The goal of this study is to conduct a prospective, longitudinal natural history study of children and adults with Angelman Syndrome using investigator-observed and parent-reported outcome measures to obtain data that will be useful for future clinical trials.
A Study to Assess Growth in Children With Idiopathic Short Stature
Study 111-903 will generate baseline growth data in children with ISS by collecting growth measurements and other variables of interest.
