13 results
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…
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,…
Primary: To compare the proportion of patients demonstrating Major Molecular Response (MMR) at 12 months (48 weeks) in the bosutinib arm with that of the imatinib arm in newly diagnosed Philadelphia chromosome positive (Ph+) chronic phase (CP)…
Primary ObjectiveTo assess the capability of the dPCR technique to predict the absence of disease relapses after imatinib discontinuation in CML patients with negative Q-RT-PCR results for longer than 18 months.Secondary Objectives• To estimate…
Primary: To characterize the pharmacokinetics of imatinib in pediatric patients age 1 to less than 4 years via appropriate integrated PBPK and pop PK approaches
The primary objective of this study is to evaluate whether surgery of residual disease in patients iwth advanced GIST responding to Imatinib improves the progression free survival.The secondary objective is to correlate the pharmacokinetics of…
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…
To investigate the efficacy (and the toxicity) of Glivec in systemic sclerosis by examining clinical outcomes (clinical and laboratory findings).
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 objective:To evaluate the effect of AMG 145 administered subcutaneously (SC) once every month (QM) compared with ezetimibe (Part B), on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in hypercholesterolemic…
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.
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…