- active not recruitingPHASE4
Pediatric Long-Term Follow-up and Rollover Study
This study looks at the long-term well-being of children and young people who have been treated with specific medications (dabrafenib and/or trametinib) for certain brain cancers in previous clinical trials. It aims to understand the lasting effects of these treatments.
United States · Argentina · Australia - recruiting
FARD (RaDiCo Cohort) (RaDiCo-FARD)
This study looks at the daily impact of rare skin conditions on patients and families in the UK. Researchers want to understand how these conditions affect people's lives, including their health, feelings, social life, and finances, by asking patients and families directly.
France - active not recruitingPHASE1, PHASE2
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
This study is testing a new liquid form of the medicine selumetinib for children aged 1 to 6 with a type of tumour linked to Neurofibromatosis Type 1 (NF1). It aims to find the right dose and check if it's safe and helps shrink the tumours.
United States · Germany · Italy - active not recruitingPHASE3
A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma
This study compares a new drug, selumetinib, with a standard treatment (carboplatin/vincristine) for children and young people aged 2-21 with a type of brain tumour called low-grade glioma, linked to Neurofibromatosis Type 1. It aims to see if the new drug is as effective and especially if it improves vision.
United States · Canada · Puerto Rico - active not recruiting
PASS of Paediatric Patients Initiating Selumetinib
This study looks at the safety of selumetinib for children aged 3-17 with Neurofibromatosis Type 1 (NF1) and certain nerve tumours (plexiform neurofibromas). It helps doctors understand the medicine's long-term effects in real-world use.
Austria · France · Germany - authorisedTherapeutic exploratory (Phase II)
Trametinib in neurofibromatosis type 1 related symptomatic plexiform neurofibromas
This study is looking into a new tablet called Mekinist for people with Neurofibromatosis Type 1 (NF1) and painful nerve tumours. It aims to see if Mekinist can shrink these tumours and improve quality of life.
Netherlands