11 results
The aim of the trial is to obtain data on the optimal dose, feasibility and pharmacokinetics of bumetanide when given as an add-on treatment for seizures in full term babies with hypoxic ischemic encephalopathy (HIE). Bumetanide will be given in a…
The primary objective of the proposed study is to investigate whether bumetanide therapy indeed reduces autistic symptomatology. Important secondary goals of this project are to determine whether bumetanide will improve specific behavioral,…
The primary objective is to demonstrate the superiority of bumetanide (0.5mg BID) oral liquid formulation compared to placebo in the improvement of ASD core symptoms after 6 months of treatment in ASD children and adolescents aged from 7 to less…
The primary objective of the study is to test the effectiveness of bumetanide across the whole spectrum of sensory processing disorders within ASD, ADHD and/or epilepsy. We also want to determine which subgroups (high/low IQ, comorbidities or not)…
Primary aim: to confirm that treatment with bumetanide improves daily life functioning and reduces behavioral symptoms related to hyperexcitability in children and adolescents with TSC.Secondary aim: to identify neurophysiological and cognitive…
The primary objective is to demonstrate the superiority of bumetanide (0.5mg BID) oral liquid formulation compared to placebo in the improvement of ASD core symptoms after 6 months of treatment in ASD children aged from 2 to less than 7 years old.
Phase 2:Primary ObjectivesTo determine the recommended Phase3 dose of mitapivat by evaluating the effect of 2 dose levels of mitapivat versus placebo on:• Anemia in subjects with sickle cell disease (SCD)• SafetySecondary ObjectivesTo evaluate the…
The primary objective is to demonstrate the superiority of ziltivekimab 15 mg s.c. once-monthly in reducing the risk of MACE (as defined by the primary endpoint) compared to placebo, both added to standard of care, in participants with established…
In this study we will look at the blood levels of the study compound ziltivekimab. We will investigate how quickly and to what extent different compositions of ziltivekimab are absorbed, transported, and eliminated from the body. Ziltivekimab is not…
To study whether ziltivekimab therapy reduces arterial wall inflammation as assessed by imaging, and reduces the systemic inflammatory tone as assessed by circulating monocytes, inflammatory biomarkers and proteomics.
1. Provide post-trial access to bumetanide treatment for NDD participants.2. To test how bumetanide cohort data compare to the existing RCT data in terms of treatment effectiveness by using randomization tests. 3. To further develop EEG biomarker…