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Virtual reality (VR) to reduce anxiety in children in the plaster room

Primary Objective: The primary objective of this study is to evaluate the difference in anxiety levels in children in the plaster room with or without distraction with VR goggles.Primary outcome parameter:Child*s anxiety score right after the…

EFFICACY, SAFETY, TOLERABILITY AND PHARMACOKINETICS OF TOFACITINIB FOR TREATMENT OF SYSTEMIC JUVENILE IDIOPATHIC ARTHRITIS (sJIA) WITH ACTIVE SYSTEMIC FEATURES IN CHILDREN AND ADOLESCENT SUBJECTS

Primary:* To assess the sustained efficacy of tofacitinib versus placebo in sJIA patients, as measured by time to sJIA flare in the double-blind randomized withdrawal phase.Secondary:* To assess efficacy of tofacitinib versus placebo in sJIA…

An open-label, randomized clinical trial on teicoplanin infection prophylaxis in pediatric patients with acute myeloid leukemia

This study has been transitioned to CTIS with ID 2023-506546-23-00 check the CTIS register for the current data. Objectives safety run-in: The primary objective is:• To assess the safety of i.v. teicoplanin prophylaxis three times per week with a…

A Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease

To determine if emixustat hydrochloride (emixustat) reduces the rate of progression of macular atrophy (MA) compared to placebo in subjects with Stargardt disease (STGD)1. To evaluate the safety and tolerability of emixustat compared to placebo2. To…

A phase 3, multicenter, randomized, double-blind, placebo-controlled study of AG-881 in subjects with residual or recurrent grade 2 glioma with and IDH1 or IDH2 mutation

This study has been transitioned to CTIS with ID 2024-512961-15-00 check the CTIS register for the current data. Primary:• The primary objective of the study is to demonstrate the efficacy of vorasidenib based on radiographic progression-free…

PErsonalized PRognosis for children with traumatic brain injury (PEPR Study)

We aim to develop an innovative personalized prognostic model for outcome of children with TBI, using a unique combination of demographic, pre-injury and clinical predictors. The value of innovative MRI techniques and promising machine learning…

PK/PD of corticosteroids in graft-versus-host disease after hematopoietic cell transplantation in children

To assess the PK/PD relationship of prednisolone as either prophylaxis or therapy of GVHD in children after HCT.

A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF CROVALIMAB VERSUS ECULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS

This study has been transitioned to CTIS with ID 2023-506526-37-00 check the CTIS register for the current data. To evaluate the safety and tolerability of crovalimab compared witheculizumab

BRAVOO study
Better understanding of reactive arthritis in children to prevent overtreatment and unnecessary consumption of care

The aim of this study is to determine the known and unknown pathogens of reactive arthritis in children between six months to 18 years old. We will use a protocolized approach of diagnostic testing in order to develop an evidence based clinical…

A Phase 1 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate
the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of a
Single Dose of DCR-PHXC in Patients with Primary Hyperoxaluria Type 3

Primary objective:To evaluate the safety and tolerability of a single dose of DCR-PHXC in patients with PH3Secondary objectives:To characterize the plasma PK of a single dose of DCR-PHXC in patients with PH3To assess the efficacy of a single dose of…

International Study for Treatment of High Risk Childhood Relapsed ALL 2010 - A randomized Phase II Study Conducted by the Resistant Disease Committee of the International BFM Study Group.

This study has been transitioned to CTIS with ID 2024-513070-21-00 check the CTIS register for the current data. Primary objectives: Improvement of CR2 rates after induction with ALL R3 with bortezomib versus withoutbortezomib in HR relapsed ALL…

Target to B! - Towards patient-tailored immunotherapy to effectively treat B-cell associated disease

To characterize the antibody and the immune cell compartment in B-cell-related disease in different states of disease activity. Study objectives fall within two main groups: 1) to compare antibodies to self or non-self-antigens before and after…

Self-control Beats Anger: Research into a training for children with learning difficulties and challenging behavior

The first aim is to test the effectiveness of the school-based targeted prevention program for youth with mild intellectual disabilities and behavior problems (e.g., aggressive and oppositional defiant behavior) at reducing behaviour problems. The…

Diagnostic accuracy of neuroblastoma patient imaging with [18F]mFBG PET-CT compared to [123I]mIBG scanning.

The primary objective is to compare18F-MFBG PET-CT imaging for neuroblastoma patients with the current standard of imaging, 123I-MIBG SPECT, using the SIOPEN score for skeletal lesions and the number of detected soft tissue lesions as endpoints.

Outcome measures in children with Angelman syndrome

The primary objective of this study is to investigate the feasibility of several promising outcome measures in children with AS, which may later serve as potential outcome measures for treatment studies and other research. The study*s secondary…

Effectiveness of blood flow restriction training after bone-patellar tendon-bone anterior cruciate ligament reconstruction: a randomized placebo-controlled trial

The purpose of this study is to evaluate the effect of low-load BFR therapy on quadriceps and hamstring strength 8-, 14-, 26-, 39- and 52-weeks after BPTB ACL reconstruction compared to placebo BFR therapy and conventional HLR training.

Do effects of methylphenidate decline after long-term use? A double-blind, placebo-controlled cross-over study of effects of methylphenidate on cognitive functioning and real world behavior in treatment naïve children compared to effects after 9 months of treatment in clinical practice

The main objective of this study is to compare the acute effects of methylphenidate on behavioral and cognitive functioning in 6 to 12 year old treatment naïve children compared to the effects after nine months of treatment with methylphenidate in…

REgistry of Pediatric Sjögren syndrome in Umcg- LongiTudinal (REPSULT)

Our hypothesis is that pedSS has a different presentation and pathophysiology than adult SS, with more genetic involvement. To test this hypothesis, we define the following specific aims: define disease presentation of children with SS-like…

Study of expression of and immunity against neoantigens in patients with constitutional mismatch repair deficiency (CMMR-D) syndrome

In this explorative study, we aim to:• Analyze neoantigens expressed on different tumors of CMMR-D patients, to identify possible target antigens for DC vaccination studies.• Investigate the presence of pre-existing immune responses against…

A Multicenter, Randomized, Open-label, Phase 3 Trial Comparing selpercatinib (LOXO-292) to Physicians Choice of Cabozantinib or Vandetanib in Patients with Progressive, Advanced, Kinase Inhibitor Naïve, RET-Mutant Medullary Thyroid Cancer (LIBRETTO-531)

Primary* To compare TFFS of patients with progressive, advanced, kinase inhibitor naïve, RET-mutant MTC treated with LOXO-292 versus cabozantinib or vandetanib.Secondary* To compare other efficacy outcomes, based on RECIST 1.1 criteria, observed in…