8 results
The primary objective of the PROTI study is to establish whether patients can correctly determine which treatment assignment they received during the randomised phase of the trial.The key secondary objective is to compare the rate of withdrawal from…
To assess the efficacy of idebenone, compared to placebo, in improving or delaying the loss of respiratory function in patients with DMD
- To gather long-term data on the safety and tolerability of idebenone inFriedreich*s Ataxia- To explore the effect of idebenone after longer term administration onneurological symptoms and signs as assessed by the InternationalCooperative Ataxia…
To obtain user and medical staff feed-back on the safety, clinical performance and ergonomic features of the IPS and IBO.
Primary:* To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.Secondary:* To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study,…
Primary objectives:- To evaluate whether the outcome in children, young people and adults with HR-MB is improved over standard therapy i.e. conventional (once a day) radiotherapy (RT) (standard therapy), for those treated with: hyperfractionated-…
Overall program:The overall aim of this project is to improve the outcome of patients diagnosed with ependymoma by improving the staging and the standard of care and to improve our understanding of the underlying biology. The program will evaluate…
PNET 5 MB-LR: Primary objective:-to confirm that the 3-year Event-Free Survival (EFS) rate in children and adolescents with standard-risk medulloblastoma having a low-risk biological profile remains in excess of 80% when patients are treated with 18…