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Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

Primary-Assess the efficacy of apitegromab compared with placebo using the HFMSE in patients 2 through 12 years oldKey secondary-Assess the efficacy of apitegromab compared with placebo based on the number of patients with clinical improvement in…

A Phase 3, 52-week, open-label, single arm study to investigate the efficacy and safety of mepolizumab SC in participants aged 6 to 17 years with hypereosinophilic syndrome.

This study has been transitioned to CTIS with ID 2023-510110-36-00 check the CTIS register for the current data. Primary objective: To evaluate the efficacy of mepolizumab SC given every 4 weeks in participants aged 6 to 17 years with HESSecondary…

Motor development and physical fitness after preterm birth in children at school age.

To explore motor development, the level of physical activity and participation in daily (school) life in preterm born children at the age of 10-12, who were seen earlier at the follow-up outpatient clinic in Maxima MC at the age of 5.5 and 8.

A Proof-of-Concept Study to Explore the Potential Efficacy of Deferiprone in Patients With
Pelizaeus-Merzbacher disease (PMD)

This study has been transitioned to CTIS with ID 2024-511968-81-00 check the CTIS register for the current data. Studying the effect of deferiprone on the motor function of patients with Pelizaeus-Merzbacher disease.

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…

Personalized tacrolimus treatment for pediatric kidney transplant recipients by using a dosing algorithm and a once-daily tacrolimus formulation.

This study has been transitioned to CTIS with ID 2024-511585-36-00 check the CTIS register for the current data. The key objective is to personalize tacrolimus treatment for children with a renal transplant by using dosing algorithms to calculate…

Combined use of induced pluripotent stem cell derived cardiomyocytes and 3D-heart tissue to develop Therapies for pediatric heart failure in Tetratology of Fallot.

The main objective of the study is to dissect the genetic and stress-based mechanisms on the development of pediatric heart failure in congenital heart disease (ConHD) with the use of induced pluripotent stem cell-derived cardiomycoytes (iPSC-CMs).…

A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes

This study has been transitioned to CTIS with ID 2024-515637-14-00 check the CTIS register for the current data. To evaluate the long-term safety and tolerability of elexacaftor(ELX)/tezacaftor (TEZ)/ivacaftor (IVA)

A clinical study to investigate Interferon gamma (IFN*) signature in patients post HSCT and in patients with impaired HSC proliferation pre-transplant

HSCT cohort:* -To investigate the relationship between IFNγ levels and IFNγ activity by measuring CXCL9 levels and the risk of graft failure* -To investigate the relationship between IFNγ levels and IFNγ activity by measuring CXCL9 levels and the…

A phase II trial of [177]Lutetium-DOTATATE in children with primary refractory or relapsed high-risk neuroblastoma

This study has been transitioned to CTIS with ID 2023-503684-42-00 check the CTIS register for the current data. Main Study Objective• To confirm the dose and assess response to single agent 177Lutetium-DOTATATE treatmentin patients with relapsed or…

Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series

This study has been transitioned to CTIS with ID 2024-514012-28-00 check the CTIS register for the current data. Assess the neurological efficacy of ambroxol in adults and children with GD3.

Testing an increased visit interval scHeme UsIng web-based Self-evaluation (THUIS-study)

Our objective is to safely increase the visit interval to 6 months in JIA patients with stable disease. The skipped 3-month visit isreplaced by a self-evaluation via e-mail by EQ-5D-5L-Y and JAMAR questionnaires that will be send via Castor EDC.…

International cooperative prospective study for children and adolescents with standard risk ALK-positive anaplastic large cell lymphoma (ALCL) estimating the efficacy of Vinblastine.

This study has been transitioned to CTIS with ID 2022-501454-11-00 check the CTIS register for the current data. PRIMARY OBJECTIVE:- To show that it is possible to cure at least 75% of patients belonging to the SR group with Vinblastine-monotherapy…

Advanced Early Detection of airway pathogens in children and adults with Cystic Fibrosis during routine follow-up and exacerbation.

Primary Objective: To investigate the yield of two methods of exhaled breath analysis (PCR based and VOC based) in the detection of Pseudomonas aeruginosa colonization or infection in young patients with cystic fibrosis (CF).Secondary Objective(s):…

Respiratory sEMG in dyspneic children before and after treatment with Salbutamol.

This study aims to validate the efficacy of a wireless and small EMG sensor as a tool for monitoring asthmatic children. We will analyse differences in diaphragmatic muscle activity as measured with this EMG sensor before and after administration of…

A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension (RESILIENT)

This study has been transitioned to CTIS with ID 2024-511852-42-00 check the CTIS register for the current data. Primary Objective:To evaluate the efficacy of taldefgrobep alfa in participants who are already taking a stable dose of nusinersen or…

An Integrated Pharmacokinetic and Safety Open-label Basket Trial of Daprodustat for the Treatment of Anemia Associated with Chronic Kidney Disease in Male and Female Children and Adolescents Aged 3 Months to Under 18 Years Requiring or Not Requiring Dialysis.

Primary objectives: - Describe the safety of daprodustat, overall (all ages) and in each age group.Secondary objectives:- Describe changes in other parameters relevant to safety, overall and in each age group.- Describe the effect of daprodustat on…

A virtual, decentralised observational follow-up study investigating feeding patterns in infancy and the associated parent-reported allergic manifestations, allergies and infections in childhood.

Given the worldwide-observed rapid increase in the prevalence of allergic diseases, it is likely that environmental risk factors, including geographic area and lifestyle factors play a substantial role in the development of allergies and other…

Genetic Risk factors for Multi-system Inflammatory Syndrome in Children and Pediatric Post COVID condition (GRIP)

Primary objective: To identify rare, high impact genetic variants in immunological genes and pathways in children with a history of MIS-C or pediatric post-COVID condition. Secondary objectives: To analyze the clinical characteristics and long-term…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial: Multiple Independent Sub-studies of Setmelanotide in Patients with POMC/PCSK1, LEPR, NCOA1 (SRC1) or SH2B1 Gene Variants in the Melanocortin-4 Receptor Pathway

This study has been transitioned to CTIS with ID 2023-507634-24-00 check the CTIS register for the current data. To evaluate the efficacy of setmelanotide on changes in body weight.