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A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of BMN 045 (previously known as PRO045) in subjects with Duchenne muscular dystrophy

Primary objective:To assess the efficacy of BMN 045 after 48 weeks treatment in ambulant subjects with Duchenne muscular dystrophy.Secondary objectives:To assess the safety and tolerability of BMN 045 after 48 weeks of treatment in all study…

A Multi-center, Randomized Parallel Group, Placebo-Controlled Double-Blind Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of Belimumab, a Human Monoclonal Anti-BLyS Antibody, Plus Standard Therapy in Pediatric Patients with Systemic Lupus Erythematosus (SLE) (BEL114055)

Primary: safety and tolerability of belimumab in a pediatric population (5-17 y) with SLE.Secondary: PK, efficacy, quality of life.

30-day, single-arm study of the safety, efficacy and the pharmacokinetic and pharmacodynamic properties of oral rivaroxaban in children with various manifestations of venous thrombosis.

-Assess the incidence of major bleeding and clinically relevant non-major bleeding -Assess the incidence of recurrent venous thromboembolism -Characterize the pharmacokinetic/ pharmacodynamic profile of a 30-day treatment with oral rivaroxaban

Pharmacokinetics and safety of Meropenem in infants below 90 days of age (inclusive) with probable and confirmed meningitis: a European multicenter phase I- II trial

Primairy objectivesTo study the pharmacokinetics (plasma and cerebrospinal fluid) of meropenem in infants <= 90 days of postnatal age with probable or confirmed bacterial meningitis (BM); To describe the safety profile of meropenem in the…

A randomised, controlled, double-blind trial to investigate the effects of a new infant formula on growth, tolerance and safety in healthy term infants.

The primary objective of this study is to investigate equivalence of weight gain from randomisation until the age of 17 weeks in infants receiving the test product compared to infants receiving the control product. Secondary objectives of this study…

An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01)

• To evaluate the safety of up to 3 additional courses of oral QLT091001 administered once daily for 7 days in subjects with LCA or RP caused by RPE65 or LRAT gene mutations who have been treated previously with a single 7-day course of QLT091001 in…

A phase II/III, randomised, double-blind, placebo-controlled, parallel group trial to evaluate safety and efficacy of tiotropium inhalation solution (2.5 µg and 5 µg) administered once daily in the afternoon via Respimat® Inhaler for 12 weeks in patients 1 to 5 years old with persistent asthma

The primary objective of this trial is to evaluate the safety and efficacy of two doses of tiotropium inhalation solution delivered via the Respimat® inhaler once daily in the afternoon in patients (1 to 5 years old) with persistent asthma on top of…

Arm training in boys with Duchenne Muscular Dystrophy

To determine whether 3D arm training with arm-support is effective in delaying the loss of arm functions in daily activities from an early age onwards in boys with DMD.

Single dose open-label PK/PD, safety and tolerability of dabigatran etexilate mesilate given at the end of standard anticoagulant therapy in successive groups of children aged 2 years to less than 12 years and 1 year to less than 2 years.

To provide single dose PK/PD dataTo investigate tolerability and safety of dabigatran etexilate solution in children1 to <12 years of age

Assessment of asymmetrical innervation of the right and left m. puborectalis as a possible cause of idiopathic incontinence, by using concentric needle electrodes in an electromyography investigation of the pelvic floor muscles.

Is it possible to make a discrimination in muscle activity between the right and left puborectalis muscle by using concentric needle electrodes in an electromyography investigation of the pelvic floor muscles?When a asymmetry is found, what can be…

A Phase I/II Study of brentuximab vedotin (SGN-35) in Pediatric Patients With Relapsed or Refractory Systemic Anaplastic Large-Cell Lymphoma or Hodgkin Lymphoma

Primary• To assess the safety profile and determine the pediatric maximum tolerated dose and/or recommended phase 2 dose of brentuximab vedotin• To assess the pharmacokinetics of brentuximab vedotin• To determine the overall response rate (complete…

The effect of intranasal insulin on development and behaviour of children with Phelan-McDermid syndrome

The aim of this project is to validate the hypothesis that intranasal insulin improves development and behaviour in children with Phelan-McDermid syndrome.

A Randomized, controlled, cross-over Study to assess Hypoallergenicity of an extensively hydrolyzed whey protein Infant formula in children with cow's milk allergy using amino-based infant formula as a reference.

The primary study objective is to assess the hypoallergenicity of an extensively hydroloyzed whey protein infant formula in children with cow's milk allergy.The second study objective is to assess the long-term effects on growth and tolerance…