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A randomized, multicenter, open-label, Phase 2 study with a safety run-in part to evaluate safety,
pharmacodynamics and efficacy of azacitidine compared to no anticancer treatment in children
and young adults with acute myeloid leukemia in molecular relapse after first complete
remission

Primary Objectives:Safety Run-in PartTo establish a safe and tolerable dose of azacitidine to be used in the randomized part of thestudy.Randomized PartTo evaluate the effect of azacitidine treatment in AML subjects at molecular relapse after…

The future of coeliac disease: immunology, environment and possibilities for new treatments.

The primary objective of CDfuture is to provide duodenal biopsies of patients with and without CD, and venous blood samples, to continue CD research as described above.

A Phase I Open-Label Multicentre Study to Assess the Pharmacokinetics and Safety of Naloxegol in Paediatric Patients Ages >= 6 Months to < 18 Years Receiving Treatment with Opioids

To assess the pharmacokinetics and safety of naloxegol in paediatric patients ages &gt; 6 months to &lt; 18 years receiving treatment with opioids.

An open-label canakinumab (ACZ885) dose reduction or dose interval prolongation efficacy and safety study in patients with Systemic Juvenile Idiopathic Arthritis (SJIA)

Primary:To evaluate if the proportion of patients in clinical remission on canakinumab 4mg/kg (+/- concomitant NSAID only) who are able toremain on a reduced canakinumab dose (2mg/kg every 4 weeks) or prolonged canakinumab dose interval (4mg/kg…

Effect of sapropterin on variations of blood phenylalanine and tyrosine over 24 hours and from day to day in patients with phenylketonuria

Primary objective- To measure the effect of sapropterin on diurnal and day to day variations of blood phenylalanine concentrations.Secondary objective- To measure the effect of sapropterin on diurnal and day to day variations of blood tyrosine…

The 'Livassured' Combined Sensor system to detect Epileptic Noctural SEizures (LICSENSE)

The primary objective of this trial are to assess the clinical usefulness (in terms of sensitivity and positive predictive value of 1. Combined heart rate and movement detection2. And the added value of video and audio detection in a population of…

Effect of transverse versus longitudinal incisions and suprapatellar approach on anterior knee pain after tibial nailing; a multicenter randomized trial

The aim of this prospective, multicenter, trial is 1) to describe the effect of a suprapatellar approach on anterior knee pain, and 2) to assess the effect of a transverse versus a longitudinal transpatellar incision on anterior knee pain.

Study into the effectiveness of behavioral parent training for children with autism spectrum disorder and behavior problems

We aim to determine the effects of a face to face and a blended parent training program (i.e., two formats of Behavioral Parent Training Groningen; BPTG) on behavior problems for children 4 through 12 years old with ASD and behavior problems.…

Allogeneic Stem Cell Transplantation of NiCord, Umbilical Cord Blood-derived Ex Vivo Expanded Stem and Progenitor Cells, in Adolescents and Adult Patients with Hematological Malignancies

The overall study objective is to evaluate the safety and efficacy of NiCord®: single ex-vivo expanded cord blood unit transplantation in patients with hematological malignancies following myeloablative therapy.

A Phase 3 Efficacy and Safety Study of Ataluren
(PTC124 ®) in Patients with Nonsense Mutation Cystic
Fibrosis

To evaluate the ability of ataluren to improve pulmonary function relative to placeboTo determine the effect of ataluren on:1. Pulmonary symptoms2. General well-being3. Health-related Quality Life (HRQL)

Observational multi-center, prospective study to develop a novel multi-parametric diagnostic model for hospitalized patients and patients on the Emergency Department presenting with lower respiratory tract infections and/ or sepsis.

To develop a novel multi-parametric diagnostic model for the management of patients with LRTI and/or sepsis that will be based on novel pathogen- and host-related factors.

The PEPaNIC long-term outcome study: Neurocognitive developmental deficit
after critical illness in children: role of modifiable epigenetic changes

We hypothesise that, in comparison with the current practice of aggressive nutritional support, tolerating caloric deficits early during critical illness in critically ill children has *carry-over* effects, through epigenetic changes, that improve…

Evaluation of the CAP-value for quantitating liver steatosis in obese children using 1H-MRS as reference standard

To investigate the correlation between the CAP-value measured with the FibroScan® device and liver fat percentage measured with 1H-MRS in a well-characterized cohort of obese children at risk of having fatty liver disease.

Reducing symptoms of depression and anxiety in adolescents with inflammatory bowel disease in order to improve quality of life and the clinical course of disease

The primary objective is to determine the efficacy of a disease specific cognitive behavioral therapy program in adolescents with IBD and increased symptoms of depression or anxiety on reducing symptoms of depression. Secondary objectives are to…

Clinical validation of a dried blood spot (DBS) method for the analysis of immunosuppressive and antifungal drugs in pediatric patients (part of the PROTECT study).

PrimairyTo clinically validate a finger prick DBS method compared to conventional venous sampling for the analysis of 5 immunosuppressive and 4 azole antifungal drugs in the pediatric population. Secondairy• Feasibility of the novel finger prick DBS…

A randomized, double-blind, placebo controlled study of canakinumab in patients with Hereditary Periodic Fevers (TRAPS, HIDS, or crFMF), with subsequent randomized withdrawal/ dosing frequency reduction and open-label long term treatment epochs

Primary: The primary objective of the randomized treatment epoch and for the overall study is to demonstrate that subcutaneous canakinumab administered every 4 weeks is superior to placebo in achieving a clinically meaningful reduction of disease…

A randomized, double-blind, double-dummy, parallel group, multicenter study of once daily Fluticasone Furoate/Vilanterol 100/25 mcg Inhalation Powder, twice daily Fluticasone Propionate/Salmeterol 250/50 mcg Inhalation Powder, and twice daily Fluticasone Propionate 250 mcg Inhalation Powder in the treatment of persistent asthma in adults and adolescents already adequately controlled on twice-daily inhaled corticosteroid and long-acting beta2 agonist (study 201378)

Primary: To demonstrate non-inferiority of RELVAR 100/25 once-daily to SERETIDE 250/50 twice-daily in adult and adolescent subjects 12 years of age and older with persistent asthma,adequately controlled on twice-daily ICS/LABA. Secondary: Adverse…

Fatigability in Spinal Muscular Atrophy

To determine the reproducibility and validity of specific outcome measures for skeletal and respiratory fatigability in subjects with SMA.

Thyroid hormone analog therapy of patients with severe psychomotor retardation caused by mutations in the MCT8 thyroid hormone transporter: The Triac Trial.

1. Normalization of the abnormal serum TH parameters and thereby improving the clinical condition of the AHDS patients2. Observation of changes in cognitive and motor function.

Listening effort in the European population: A new innovative program of research and training

The aim of the study is to strengthen the basis for a new application of pupillometry (i.e., the measurement of pupil dilation) within the field of Audiology. An innovative method to quantify listening effort (pupillometry) will be further developed…