162 results
* To estimate the prevalence of hyposalivation and xerostomia in pediatric cancer survivors treated with different cancer regimens (e.g., H&N RT, TBI, CT, or those who developed cGVHD following allogeneic stem cell transplantation).* To…
to assess the pulmonary toxicity in patients treated with cyclophosphamide compared to a control group
The objectives of the trial are to assess whether ABT-414 alone or in combination with TMZ improves overall survival (OS), PFS, tumor response, quality of life, NDFS and steroid use compared to standard treatment with lomustine single agent or TMZ…
DDAVP treatment combined with FVIII clotting factor concentrates in patients with mild hemophilia A.
Primary objectives:To assess the proportion of non-severe hemophilia A patients within FVIII target levels with the DDAVP and FVIII concentrate combination treatment in the first 72 hours after the start of combination treatment, without adding off-…
Primary objective- To compare the efficacy of liraglutide versus placebo on weight loss in obese paediatric subjects with PWS at 16 weeks and versus no treatment at 52 weeks.Secondary objectives - To compare the efficacy of liraglutide versus…
The primary objective is to investigate the neural correlates of the integration of multisensory information in adolescents with ASD.
Primary Objective: Identification of genetic variants causative of CHD. Expression of candidate genes in a zebrafish model. Overall goal is to gain insight in cardiac development by expanding our knowledge of genetic components in de novo and…
Primary objective:The primary objective of this study is to assess the time to painful VOC resolution, measured from the first dose of sevuparine given to achievement of crises resolution, as compared to placebo. Secondary objectives: The secondary…
The iBerry Study, phase 2, has two related main study objectives:1) Describing the long-term prognosis of sub-threshold symptoms in terms of course (transition to several full-blown psychiatric disorders, chronicity, recurrence, comorbidity) and…
In the MILESTONE study, we will test and evaluate a model of managed transition which contains a planned, purposeful and needsbasedassessment of those who reach the transition boundary at CAMHS. The Transition Readiness and Appropriateness Measure…
The aim of this study is to compare gut-directed hypnotherapy with standard medical treatment in children and adolescents with chronic idiopathic nausea or nausea due to functional dyspepsia. We will examine the effect of these treatments on…
To assess the prevalence of hypothyroidism and hyperthyroidism in the entire cohort of childhood cancer survivors and the therapy related risk factors. Specifically:Part I: 1. To systematically review existing studies on the association between…
The primary objective is to demonstrate the efficacy of AR101, a pharmaceutical-grade peanut allergen formulation, through reduction in clinical reactivity to limited amounts of peanut allergen in peanut-allergic children (ages 4-17 years, inclusive…
-Evaluate the safety and efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in male subjects <= 17 years of age with cerebral adrenoleukodystrophy (CALD)
To investigate whether an individualized dosing regimen for Thymoglobulin leads to a better immune reconstitution after HCT (definition as in primary endpoint), as compared to historically non-individualized treated patients receiving Thymoglobulin…
To determine the contribution of musculoskeletal pain to activity limitations corrected for the impact biomechanical factors (joint biomechanics, proprioception, physical fitness) and psychological comorbidity. The secondary objective is to identify…
To characterize the natural history and clinical management of AHP patients with porphyria attacks.
Safety ObjectiveThe safety objective for this study is as follows:* To evaluate the safety and tolerability of atezolizumab in pediatric and young adult patients, focusing on the nature, frequency, and severity of serious and non-serious adverse…
Primary objective of this study is to improve identification of epileptic HFOs in EEG and MEG, by comparing them to HFOs in corticography. Secondary objectives are to study the predictive value of non-invasively recorded HFOs for disease activity…
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with the study drug. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a…