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The COllaborative Neonatal NEtwork for the first CPAM Trial, the CONNECT

The main objective is to identify the optimal management for patients with an asymptomatic CPAM, based on functional outcome measures. Furthermore, the primary objective is to stratify asymptomatic CPAM patients into a low and high risk group for…

A phase 3 study of PTC923 in subjects with phenylketonuria

Primary:• To evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria (PKU) as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6 (ie, the average of each respective…

A randomized controlled trial to investigate the (cost)effectiveness of oral immunotherapy with different allergens in young children with an established food allergy.

What is the clinical- and cost-effectiveness of early low-dose oral immunotherapy aimed at long-term tolerance induction in children under the age of 30 months with an established food allergy compared to routine care?What is the effect of early low…

Reduction of blood loss in pediatric osteotomies around the hip
- A randomized placebo-controlled trial with tranexamic acid -

This study has been transitioned to CTIS with ID 2024-516324-34-01 check the CTIS register for the current data. To evaluate (reduction of) intraoperative blood-loss with and without pre-operative TXA administration, in children undergoing a…

Multicentre prospective trial for extracranial malignant germ cell tumours including a randomized comparison of Carboplatin and Cisplatin

This study has been transitioned to CTIS with ID 2023-507582-25-00 check the CTIS register for the current data. PRIMARY OBJECTIVESThe primary objective of MAKEI V is to assess in a randomized comparison whether the efficacy of Carboplatin (600 mg/…

A Randomized, Blinded, Placebo-Controlled, Phase 1 Single Ascending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study in Pediatric SMA Participants Previously Treated with Onasemnogene Abeparvovec (Zolgensma*) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115

This study has been transitioned to CTIS with ID 2023-505643-39-00 check the CTIS register for the current data. Part A: PrimaryTo evaluate the safety and tolerability of single ascending dose of BIIB115 administered via IT bolus injection to…

A PROSPECTIVE, RANDOMIZED, OPEN-LABEL PHASE 2 STUDY TO EVALUATE THE SUPERIORITY OF INOTUZUMAB OZOGAMICIN MONOTHERAPY VERSUS ALLR3 FOR INDUCTION TREATMENT OF CHILDHOOD HIGH RISK FIRST RELAPSE B-CELL PRECURSOR ACUTE LYMPHOBLASTIC LEUKAEMIA

This study has been transitioned to CTIS with ID 2023-509810-13-00 check the CTIS register for the current data. Primary Objectives Efficacy: To demonstrate the superiority of InO monotherapy vs ALLR3 induction in paediatric participants between 1…

The influence of electroencephalographic density spectral array guidance of sevoflurane administration on recovery from general anaesthesia in children between 6 months and 12 years.

To evaluate the influence of DSA monitoring, provided by the NarcotrendTM monitor, on the speed of emergence and recovery from GA.

Active Monitoring versus an Abduction Device for treatment of Infants with Centered Dysplastic Hips, a Comprehensive Cohort Study (TReatment with Active Monitoring (TRAM)-Trial)

The aim of this study is to compare treatment with an abduction device to active monitoring of infants with centered dysplastic hips (Graf type IIa- or IIb or IIc) during the first year of life.

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study to Examine the Efficacy and Safety of ZX008 in Subjects with CDKL5 Deficiency Disorder Followed by an Open-Label Extension

This study has been transitioned to CTIS with ID 2023-506269-78-00 check the CTIS register for the current data. Objectives for Part 1: Primary (Efficacy): To demonstrate that the efficacy of fenfluramine (ZX008) 0.8 mg/kg/day is superior to placebo…