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Effect of an immune-supportive diet on gut permeability and allergic symptoms in children with peanut and/or nut allergy.

Therefore, the purpose of our study is to study, in peanut and nut allergic children:1. the relationship between gut permeability and threshold levels to peanut or nuts;2. the effect of an immune-supportive diet on gut permeability, coexisting…

PHASE 1/2 STUDY TO EVALUATE PALBOCICLIB (IBRANCE® ) IN
COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE AND/OR IN COMBINATION WITH TOPOTECAN AND CYCLOPHOSPHAMIDE IN
PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID
TUMORS

Primary: To compare the efficacy of palbociclib in combination with TMZ and IRN vs TMZ and IRN chemotherapy alone in the treatment of children, adolescents, and young adults with recurrent or refractory EWS.Secundary: To further compare the efficacy…

Investigator led, double-masked, multicenter, randomized clinical trial for the comparison of Atropine 0.5% versus Atropine 0.05% eye drops for the prevention of myopia progression in Dutch children.

This study has been transitioned to CTIS with ID 2024-516379-34-00 check the CTIS register for the current data. Objectives:To compare the efficacy of Atropine 0.05% to Atropine 0.5% treatment in European children with progressive myopia, and to…

An Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Multiple Doses of ELA026 in Participants with Secondary Hemophagocytic Lymphohistiocytosis (sHLH).

Primary Objectives:• To determine the safety of ELA026 administered IV and SC to participants with sHLH.• To identify the RP3D and schedule for ELA026.Secondary Objectives:• To characterize the pharmacokinetic (PK) profile of ELA026 administered IV…

Phase II trial of nivolumab for pediatric and adult relapsing/refractory ALK+ anaplastic large cell lymphoma, for evaluation of response in patients with progressive disease (Cohort 1) or as consolidative immunotherapy in patients in complete remission after relapse (Cohort 2)

Primary objectivesCohort 1 Estimate the efficacy of nivolumab treatment in patients with relapsed/refractory ALK+ ALCL in terms of best objective response rate within thefirst 24 weeksCohort 2 Estimate the efficacy of nivolumab treatment as…

Pharmacokinetic-guided dosing of emicizumab in congenital haemophilia A patients - The DosEmi study

This study has been transitioned to CTIS with ID 2024-515528-35-00 check the CTIS register for the current data. Primary objectiveTo determine whether individualized pharmacokinetic (PK)-guided dosing of emicizumab is non-inferior to conventional…

An open-label, first-in-human, dose-escalation/expansion study of SAR443579 administered as single agent by intravenous infusion in adult and pediatric participants with relapsed or refractory acute myeloid leukemia (R/R AML), B-cell acute lymphoblastic leukemia (B-ALL), high risk-myelodysplasia (HR-MDS) or blastic plasmacytoid dendritic cell neoplasm (BPDCN)

This study has been transitioned to CTIS with ID 2023-508357-58-00 check the CTIS register for the current data. The aim of the escalation portion of this study, in which SAR443579 is administered for the first time in humans, is to establish the…

A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants with Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency

This study has been transitioned to CTIS with ID 2022-502492-32-00 check the CTIS register for the current data. PrimaryPart I: Evaluate the safety and tolerability of mRNA-3705 administered via IV infusion to participants with isolated MMA due to…

A study comparing the effect and safety of once weekly dosing of somapacitan with daily Norditropin® as well as evaluating long-term safety of somapacitan in a basket study design in children with short stature either born small for gestational age or with Turner syndrome, Noonan syndrome, or idiopathic short stature

This study has been transitioned to CTIS with ID 2023-506927-27-00 check the CTIS register for the current data. Primary objectiveThe primary objective of this study is to confirm non-inferiority of once-weekly somapacitan compared with once-daily…

Phase IIIb, open-label, single-arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101 administered intrathecally (1.2 x 1014 vector genomes) to participants 2 < 18 years of age with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen (Spinraza®) or risdiplam (Evrysdi®)

The purpose of this study is to characterize the safety and tolerability of OAV101 IT in participants who have discontinued treatment with nusinersen (Spinraza®) or risdiplam (Evrysdi®). The data from this study will expand on the data generated…

An open-label, single-center, exploratory study of the safety and efficacy of avalglucosidase alfa in patients with non-classic Pompe disease aged >= 5 years.

This study has been transitioned to CTIS with ID 2024-518215-18-00 check the CTIS register for the current data. To explore safety, tolerability and efficacy of avalglucosidase alfa in previously treated alglucosidase alfa late-onset Pompe disease…

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.