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The optimal treatment strategy for complex appendicitis in the pediatric population

Subgroup 1 (complex appendicitis without mass and/or abscess formation): LA is associated with reduced superficial site infections, shorter length of stay, less costs, less pain, and better hr-QoL, compared with OA without compromising overall…

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 two-cohort, open-label, single arm, multicenter study to evaluate efficacy, safety and
tolerability, pharmacokinetics and pharmacodynamics, of emapalumab in children and
adults with macrophage activation syndrome (MAS) in Still*s disease (including systemic
juvenile idiopathic arthritis and Adult onset Still*s disease) or with MAS in Systemic lupus
erythematous

This study has been transitioned to CTIS with ID 2024-516153-52-00 check the CTIS register for the current data. Primary objective: To demonstrate efficacy of emapalumab in the treatment of patients in:• Cohort 1: Macrophage activation syndrome (MAS…

A Phase I/II study of Brigatinib in pediatric and young adult patients with ALK+ Anaplastic Large Cell Lymphoma, Inflammatory Myofibroblastic Tumors or other solid tumors

This study has been transitioned to CTIS with ID 2024-513412-10-00 check the CTIS register for the current data. Phase 1: • To determine the MTD/RP2D regimen of brigatinib monotherapy when administered in pediatric and AYA patients with ALK+ ALCL or…

TEMOkids study (ORP-TMZ-I- b): A Population pharmacokinetic, acceptability and safety study for KIMOZO, a paediatric oral suspension of temozolomide

Primary objective:• Evaluate PK parameters of the oral suspension of temozolomide in the paediatric population aged 1 year and over.Secondary objectives:• Evaluate the safety of the oral suspension of temozolomide,• Evaluate the acceptability of the…

An explorative intervention study to investigate the feasibility and safety of oral immunotherapy with different allergens in young children diagnosed with food allergy.

Primary: Is OIT with standard food products safe to perform in children aged 9 to 24 months with a proven food allergy for hen's egg, peanut, cow's milk, cashewnut, hazelnut, walnut and/or one of the more rare allergens (as soy, pits and…

Dermal Substitution in Paediatric Burns: A Prospective Case Series

The aim of this study is to investigate the scar quality in a paediatric burn population treated with Glyaderm up to 12 months after surgery.

Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia…

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/…

Table salt for the treatment of umbilical granuloma

The aim of this observational study is to evaluate the treatment of umbilical granulomas with table salt.

Effectiveness of L-serine dietary supplementation in children with a GRIN2B loss-of-function mutation: n-of-1 series

Assess the potential efficay of L-serine dietary supplementation in patients children with a GRIN2B LoS mutation.

Immune responses Induced by Vaccination Against COVID-19 in Dutch healthy subjects

Monitoring & evaluation of immune responses induced by COVID-19 vaccines in the general population in the Netherlands.

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…

A phase 2, open-label, single-arm, cohort study to evaluate the safety, efficacy, and pharmacokinetics of sparsentan treatment in pediatric subjects with selected proteinuric glomerular diseases (EPPIK)

This study has been transitioned to CTIS with ID 2023-505497-14-00 check the CTIS register for the current data. Primary Objectives:• Evaluate the safety and tolerability of sparsentan oral suspension (Population 1 and Population 2) and tablets (…

An Open-label, Multinational, Multicenter, Intravenous
Infusion Study of the Efficacy, Safety, Pharmacokinetics,
and Pharmacodynamics of avalglucosidase alfa in
Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)

This study has been transitioned to CTIS with ID 2024-513859-33-00 check the CTIS register for the current data. The overall objective is to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in male and…

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…

International collaborative treatment protocol for infants under one year with KMT2A-rearranged acute lymphoblastic leukemia or mixed phenotype acute leukemia

This study has been transitioned to CTIS with ID 2022-502503-30-00 check the CTIS register for the current data. The primary objective is to improve the outcome (in terms of event-free survival (EFS) as the primary endpoint) of newly diagnosed KMT2A…

A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects with Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy) (GALax-C)

This first-in-human study is planned to include both a dose ranging phase and a confirmatory phase, with the intention that all data collected throughout the study may support registration. The dose ranging phase will be conducted in cohorts of 3-4…

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.

Phase 2 Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sotatercept (MK-7962) in Children from 1 to Less Than 18 Years of Age With PAH on Standard of Care

This study has been transitioned to CTIS with ID 2023-504861-22-00 check the CTIS register for the current data. This study will assess the safety and tolerability of sotatercept in pediatric participants with PAH WHO Group 1 who receive PAH…