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A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy

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…

A placebo-controlled discontinuation trial of off-label used risperidone in people with intellectual disability

The primary objective of this study is: To study the effect of controlled discontinuation of long-term used risperidone, for the treatment of challenging behavior, on behaviour and health. Our hypothesis is that long-term use of risperidone for…

Music Under Surgery In Children

The main objective of this study is to investigate whether a music intervention as complementary treatment is effective in reducing anxiety and distress around surgery. Secondary objectives are effects of music on pain, on preoperative anxiety, on…

An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.

The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.

Paediatric distal radius torus fracture treatment comparison in a prospective randomized control trial: mitella versus plaster cast

If the study shows that the treatment with a sling gives an earlier return to normal function en does not give a clinically significant difference in pain experience compared to cast therapy than the traditional treatment can be adjusted. We aim at…

A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Biodistribution, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.

2-STEP: A single-centre, phase II study to evaluate the safety, tolerability and pharmacokinetics of 2-Iminobiotin (2-IB) in neonates with gestational age of *36 weeks with moderate to severe perinatal asphyxia treated with therapeutic hypothermia

To explore the safety, tolerability and the pharmacokinetic profile of 2-IB when given on top of therapeutic hypothermia.

URIKA, continuous ultrasound monitoring of urinary bladder filling in children with dysfunctional voiding: a pilot study.

To determine the accuracy of the URIKA urinary bladder sensor in measuring the distance between the anterior - and posterior wall of the bladder and to determine the range in bladder diameters at which the child should be alarmed to void.

Physical and psychological sequelae of childhood cancer from diagnosis till 5-8 years after diagnosis

The current study is a continuation of the cohort 5-8 years post-diagnosis and aims to evaluate:1.The course of the nutritional status, and the related factors and consequences of malnutrition in survivors of childhood cancer until 5-8 years post-…

Differentiating simple from complex appendicitis in children before surgery: The Complex Appendicitis Tool (CAT)

1. Immunology and Pathology:To determine the immune response of patients with simple and complex appendicitis in order to identify markers which distinguish between simple and complex appendicitis.2.Clinical and Imaging:To analyze the differences in…

Spinal Muscular Atrophy, SMN protein and Genetics; A population based study on spinal muscular atrophy in the Netherlands to explore the prevalence of SMA type 1-4 and their severity in the Netherlands by registrating all patients in a so called SMA-database, to elucidate the genetic factors determining disease severity and to determine the value of SMN protein concentrations as a surrogate marker for clinical trials.

The main objective of this prospective population-based study of patients with proximal SMA in the Netherlands is to register SMA patients in the Netherlands in a socalled SMA-database to study prevalence and severity of SMA types 1-4, to perform…

ACNS0332: Efficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients - A Groupwide Phase III Study

The goal of this study is to determine whether radiosensitization with carboplatin or the addition of Isotretinoin to maintenance therapy improves cure rates for children with other than average risk medulloblastoma/PNET.

Autism: from phenotype to genotype and back

1. To identify genetic variants associated with an increased risk of ASD using different genetic models (dominant, recessive, interaction).2. To determine the relationship between different genetic variants and the associated neuropsychiatric ASD…

First international Inter-Group Study for classical Hodgkin's Lymphoma in Children and Adolescents

The objective of the study is to diminish the amount of chemotherapy and/ or radiotherapy for children with Hodgkin lymphoma who are likely to receive too much treatment with current treatment protocols.

Effect of in- and outpatient brace adaptation protocols on brace compliance in adolescent patients with idiopathic scoliosis

The objective of this study is to evaluate whether an in- or outpatient initiation of the brace treatment has an influences short and long term brace compliance.

A Phase 2, multicenter, open-label study to evaluate the pharmacokinetics, pharmacodynamics,
safety and activity of azacitidine and to compare azacitidine to historical controls in pediatric
subjects with newly diagnosed advanced myelodysplastic syndrome or juvenile myelomonocytic
leukemia before hematopoietic stem cell transplantation

The primary objective is to assess the treatment effect on response rate (MDS: either completeremission [CR], partial remission [PR], or marrow CR; JMML: either clinical completeremission [cCR] or clinical partial remission [cPR]); at Cycle 3 Day 28…

Brain development between ages 8 and 25:
A longitudinal study

A fundamental question in the current research proposal concerns how changes in cognitive and social-emotional behavior are related to functional and anatomical brain development. The current proposal has the objective to (a) understand the neural…

The LINC-study: LInking EDCs in maternal Nutrition and Child health

To relate exposure markers of EDCs to health outcomes in children - in particular obesity and neurobehavioral disorders -, effect biomarkers, and other parameters via multiple regression and multivariate analysis, while taking into account relevant…

A Multicentre, Long-term Safety, Efficacy and Pharmacokinetics Study of Lubiprostone in Paediatric Subjects Aged *6 to <18 years with Functional Constipation

To assess the long-term safety, efficacy, and pharmacokinetics of oral lubiprostone 12 or 24 mcg capsules dosed twice daily (BID) when administered orally for 36 weeks in paediatric subjects with functional constipation. Evaluation of lubiprostone…

European genetics study of hearing impairment in patients with cancer during childhood treated with cisplatin.

The aim of this study is to validate known, and identify novel allelic variants which may play a role in the risk of cisplatin-induced ototoxicity in a substantial international cohort of children diagnosed with cancer.