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Web-based attention bias modification treatment for childhood anxiety disorders: a randomized, double-blind, controlled trial

Specific AimsAs reviewed above, childhood anxiety disorders are highly prevalent and debilitating, and there is an urgent need for improvement of current treatment strategies. An innovative, but scarcely examined, treatment option for childhood…

Response to DDAVP In mild Hemophilia A patients, in Search for dEterminants

The aim of this project is to investigate the association between clinical and genetic factors and DDAVP response in MHA patients and to determine their relative contributions to the DDAVP response.

Treatment of severe acute GVHD after allogeneic hematopoietic stem cell transplantation with steroids versus MSC and steroids.
A prospective double-blind placebo-controlled randomized phase III trial

To improve the response rate to treatment of severe acute GVHD (grade II-IV with gut involvement ) by adding infusion of Mesenchymal Stroma Cells to standard high dose prednisolone.

Unrelated cord blood transplantation after reduced toxicity conditioning with mesenchymal stromal cell co-infusion in patients with severe epidermolysis bullosa

We propose a phase II study (intervention) in patients with severe generalized recessive dystrophic EB receiving reduced toxicity conditioning chemotherapy followe by cord blood transplantation with co-infusion of mesenchymal stromal cell units.…

*IBDome; Identification of rare variants in candidate genes and regulatory elements in familiar and sporadic early-onset IBD (inflammatory bowel disease) patients*

Primary Objective: Identification of rare variants in candidate genes and regulatory elements in pediatric IBD patients. Secondary Objective(s): To determine the functional consequences of identified genetic variants in IBD, we will correlate…

A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL GROUP, MULTI-CENTER TRIAL OF PREGABALIN AS ADJUNCTIVE THERAPY IN PEDIATRIC AND ADULT SUBJECTS WITH PRIMARY GENERALIZED TONIC-CLONIC SEIZURES

Determine efficiency and safety of two different doses of pregabalin as compared to placebo.

A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

primary objective: To evaluate the long-term safety and tolerability of lumacaftor in combination with ivacaftor in subjects with cystic fibrosis (CF), homozygous or heterozygous for the F508del-cystic fibrosis transmembrane conductance regulator (…

Prevent CardioKids

The aim of Prevent CardioKids is to evaluate to what extend disturbed vascular function markers are present in children of different weight categories and whether these are age dependent. Also differences * and relationships between * vascular…

Treatment of severe steroid-refractory acute GvHD with mesenchymal stromal cells.
A phase III randomized double-blind multi-center HOVON study.

Primary objective* To improve the response rate to treatment of severe steroid-refractory acute GvHD grade II-IV (with gut and/or liver involvement) by early addition of MSC to standardized second line treatmentSecondary objectives* To study the…

Prospective data collection on patients with rare bleeding disorders (PRO-RBDD)

The main objective is to improve treatment with clotting factor concentrate of patients affected by RBDs by collecting and analysing data on the patients genotype, laboratory phenotype and clinical severity.Secondary objectives are to gain…

Prospective Open label study of Parenteral vs Enteral iron in Young IBD patients and Effect on physical fitness

To examine if intravenous administration of iron ismore efficacious than oral iron in improvement offitness scores, iron status and reduction of fatigue

A Phase I/II, open-label, dose escalating with 48-week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously knoen as PRO053) in subjects with Duchenne muscular dystrophy

Primary objective: To assess the efficacy of BMN 053 at recommended dosing regimen after 48 weeks treatment in ambulant subjects with Duchenne muscular dystrophy.Secondary objectives: - To assess the safety and tolerability of BMN 053 after single…

Novel Self-management Techniques for Early Expression of Psychopathology:
An Experimental Study Linking Neural and Mental State Reactivity Phenotypes:
SMARTSCAN (Self-Management of Altered environmental Reactivity Treatment SCANning)

By relating valid learning fMRI phenotypes to the earliest expression of psychopathology and response to non-pharmacological self-management treatment, the project will contribute to the new innovative field of practical neuroimaging in the earliest…

Set Shifting in Adolescents with Anorexia Nervosa.

Primary ObjectiveTo assess in 20 adolescents with anorexia nervosa the cognitive flexibility in response to two computerised category learning tasks and compare these results to a age and gender matched control group. Task one will present pictures…

Bronchiolitis obliterans syndrome and other allo-reactive lung syndromes
after allogeinic hematopoietic stem cell transplantation
Is early prediction of bronchiolitis obliterans possible by using biomarkers possible?

In this study we would like to find new biomarkers in BOS after haematopoietic stem cell transplantation (HSCT), that may lead to earlier identification of BOS than a lung function decline.

TimeToStop (TTS) Trial, A randomized controlled trial of cognitive consequences of early versus late antiepileptic drug withdrawal after pediatric epilepsy surgery

Primary Objective: To assess whether early postoperative AED withdrawal improves cognitive function compared to late withdrawal.Secondary objectives: * To confirm safety of earlier AED withdrawal; we will assess eventual seizure freedom, seizure…

Robot-aided system identification (ROBIN) of neural and non-neural contributors to ankle joint impedance in children with CP

Primary objective of this study is to assess the reliability and validity of the neuromuscular parameters of ankle joint impedance estimated by nonlinear SIPE technology in children with CP compared to current clinical manual tests and gait analysis…

A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis

Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.

The value of MRI diagnostics in haemophilic arthropathy: a pilot study

Primary Objective: (diagnostic)• Does MRI detect structural changes of haemophilic arthropathy in knees and ankles undetected by the Pettersson score and HJHS? (Research question 1 a & b)Secondary Objective(s): (prognostic)• Can MRI predict…

The incidence of (para) thyroid disorders ten years after the introduction of a new thyroid protection during 131I-MIBG treatment in children with neuroblastoma

Long-term evaluation of thyroid disorders ( TSH elevation and the occurrence of thyroid nodi/carcinoma) in survivors of childhood NBL who received KI, methimazole and thyroxine as thyroid protection during 131I-MIBG exposure. Investigation of the…