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Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II

This study has been transitioned to CTIS with ID 2024-516123-13-00 check the CTIS register for the current data. Part I:Primary Objective: Evaluate the effects of tiratricol on neurodevelopment in young MCT8 deficiency patients, as measured by the…

An open label, single arm study to evaluate single and multiple dose pharmacokinetics, safety and tolerability, and to explore clinical outcomes of treatment with intravenous |(IV) zanamivir in neonates and infants under 6 months of age with confirmed complicated influenza infection

PrimaryTo characterise the single and multiple dose to steady state pharmacokinetics of IV zanamivirin hospitalised neonates and infants under 6 months of age with influenza infection.SecondaryTo evaluate the safety and tolerability of IV zanamivir…

FaR-RMS - An overarching study for children and adults with Frontline and Relapsed RhabdoMyoSarcoma

This study has been transitioned to CTIS with ID 2024-510579-40-00 check the CTIS register for the current data. PRIMARY OBJECTIVES*Phase 1 Dose Finding Studies:-To determine the recommended phase II dose (RP2D) of new systemic therapy regimens. *…

An exploratory, open label, multiple dose, multicentre phase I/II trial evaluating safety and efficacy of prenatal and/or postnatal infusion of allogenic expanded fetal mesenchymal stem cells for the treatment of severe Osteogenesis Imperfecta compared with historical and untreated prospective controls.;In the Netherlands patients will not be treated. Retrospective and prospective control patients will be included

This study has been transitioned to CTIS with ID 2023-504593-38-00 check the CTIS register for the current data. The aim of this trial is to infuse multiple doses of human 1st trimester liver-derived MSC for the treatment of severe OI to determine…

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…

The effect of antenatal acetaminophen administration on breathing effort of premature infants at birth: a pilot study

The primary objective of this study is to evaluate the feasibility of antenatal paracetamol/placebo administration to pregnant women 0.5-2 hours prior to birth.The secondary objective of this study is to compare the effect of antenatal acetaminophen…

Oxytocin and reactivity to infant signals in mothers postpartum depression

In a randomized control trial (RCT) with mothers with postpartum depressive symptoms the following main hypotheses will be tested:• Oxytocin will promote more sensitive caregiving during mother-infant interactions and while interacting with a life-…

Study on Pharmacokinetics of newly developed ANtiretroviral agents in HIV-infected pregNAnt women (PANNA)

This study has been transitioned to CTIS with ID 2024-515487-31-00 check the CTIS register for the current data. The primary objective of this study is to describe the pharmacokinetics of antiretroviral agents, for which no or limited available…

A Phase 1/2 Study of the Oral TRK Inhibitor larotrectinib in Pediatric patients with Advanced Solid or Primary Central Nervous System Tumors

This study has been transitioned to CTIS with ID 2022-502668-20-00 check the CTIS register for the current data. PHASE 1 OBJECTIVES:Primary:To determine the safety of oral larotrectinib , including dose-limiting toxicity (DLT), in pediatric patients…

Treatment study for children and adolescents with Acute Promyelocytic Leukemia

This study has been transitioned to CTIS with ID 2024-518669-83-00 check the CTIS register for the current data. Primary objective:To assess, in an international pediatric study, the efficacy, in terms of event-free survival, of a combination of ATO…

RIC-NEC Phase II Feasibility Randomized Controlled Trial: Remote Ischemic Conditioning in Necrotizing Enterocolitis

Primary Endpoint: Feasibility of including patients in the study, randomization and delivery of the intervention. To assess the primary endpoint, we will examine the probability that: (1) a screened patient is eligible; (2) an eligible patient…

Maternal and fetal/neonatal pharmacokinetics and - dynamics of corticosteroids during pregnancy as treatment for fetal lung maturation

To examine the pharmacokinetics in maternal blood of standard regimen at the Erasmus MC of two doses of 12 mg betamethasone intramuscular with a 24 hours interval for pregnant women suspected of preterm birth with a gestational age between 23+5…

A phase I/II post-cord blood HCT dendritic cell vaccination trial directed against WT1 for pediatric and young adult acute myeloid leukemia: the U-DANCE-anti-AML-trial

This study has been transitioned to CTIS with ID 2024-517922-24-00 check the CTIS register for the current data. Although DC vaccinations have been used in allo-HCT settings, no previous studies have been performed using a CBDC vaccine after CBT.…

Phase I/II, Open-Label, Multicenter Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Durvalumab Monotherapy or Durvalumab in Combination with Tremelimumab in Pediatric Patients with Advanced Solid Tumors and Hematological Malignancies

Dose-finding:-To determine the adult equivalent exposure/MTD/recommended Phase II pediatric dose of durvalumab monotherapy and durvalumab in combination with tremelimumab-To determine the safety profile of durvalumab monotherapy, or durvalumab in…

A Phase IIb, prospective, intra-patient randomised controlled, multicentre study to evaluate the safety and efficacy of an autologous bio-engineered dermo-epidermal skin substitute (EHSG-KF) for the treatment of partial deep dermal and full thickness burns in children in comparison to autologous split-thickness skin grafts (STSG)

To evaluate the efficacy and safety of EHSG-KF in comparison to meshed STSG in children with partial deep dermal and full thickness burns. Primary Objective: To evaluate the efficacy of EHSG-KF in comparison to meshed STSG based on:• Ratio of…

A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ORGN001 (formerly ALXN1101) in Pediatric Patients with Molybdenum Cofactor Deficiency (MoCD) Type A Currently Treated with Recombinant Escherichia coli-derived Cyclic Pyranopterin Monophosphate (rcPMP)

Primary:• To evaluate the safety of ORGN001 (formerly ALXN1101) over the first 6 months of treatmentSecondary:• To characterize the pharmacokinetics (PK) of increasing doses of ORGN001 (formerly ALXN1101) • To evaluate the effect of ORGN001 (…

Non-interventional study to investigate the transfer of vaccination induced maternal antibodies in infants with fetal growth restriction.

The aim of this study is to investigate whether the transfer of antibodies from mother to infant is less effective in infants with intrauterine growth restriction (FGR) compared to term-born infants without intrauterine growth restriction or who are…