5 Clinical Trials for Various Conditions
This is a health system-level research study of physicians and care providers. The purpose of this study is to assess the clinical evaluation and management (drug, procedures, counseling, and other) of a subset of common patient care indications.
The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and childhood myotonic dystrophy type 1, and develop biomarkers for the condition.
This is a natural history study to improve the types of assessments and biological samples that will be used in clinical drug trials in both congenital myotonic dystrophy and childhood myotonic dystrophy.
Real-world evidence of the clinical course of patient symptoms following initiation of sacubitril/valsartan via PROs with a patient-centered study design will provide important evidence of potentially beneficial outcomes associated with the use of this therapy.
The primary objective was to demonstrate the effect of teriflunomide (HMR1726) (14 milligram per day \[mg/day\] and 7 mg/day), in comparison to placebo, for reducing conversion of participants presenting with their first clinical episode consistent with multiple sclerosis (MS) to clinically definite multiple sclerosis (CDMS). The secondary objectives were: * To demonstrate the effect of teriflunomide, in comparison to placebo, on: * Reducing conversion to definite multiple sclerosis (DMS) * Reducing annualized relapse rate (ARR) * Reducing disease activity/progression as measured by Magnetic Resonance Imaging (MRI) * Reducing accumulation of disability for at least 12 weeks as measured by the Expanded Disability Status Scale (EDSS) * Proportion of disability-free participants as assessed by the EDSS * Reducing participant-reported fatigue * To evaluate the safety and tolerability of teriflunomide * To evaluate the pharmacokinetics (PK) of teriflunomide * Optional pharmacogenomic testing aimed at assessing the association between the main enzyme systems of teriflunomide metabolism and hepatic safety, and other potential associations between gene variations and clinical outcomes