75 results
To evaluate the efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA
To evaluate the effect of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) on glucose tolerance in CF subjects with impaired glucose tolerance (IGT) or CF related diabetes (CFRD)
To evaluate the long-term safety and tolerability of elexacaftor(ELX)/tezacaftor (TEZ)/ivacaftor (IVA)
The primary objective is reducing treatment-related morbidity and mortality without adversely impacting DFS in Ph+ ALL patients, classified as Standard Risk (SR) based on low minimal residual disease (MRD) at week 10-12 of therapy.Because there is…
To evaluate the efficacy of VX-121/TEZ/D-IVA in CF subjects who are homozygous forF508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, orhave at least 1 other TCR CFTR mutation and no F508del mutation
Primary objective:To assess, in an international pediatric study, the efficacy, in terms of event-free survival, of a combination of ATO and ATRA in newly diagnosed SR APL children and adolescents and to explore the safety and efficacy of a…
The aim of this study is to investigate the efficacy of subcutaneous IFX in the treatment of moderate to severe Crohn*s disease with and without concomitant immunosuppression, as measured by the proportion of patients in corticosteroid-free clinical…
To study whether polytherapy (methotrexate plus sulfasalazine plus hydroxychloroquine) results in more patients with inactive disease and therefore less patients who need treatment with a TNF inhibitor after 6 months of treatment compared to primary…
Primary objective:- To confirm in a multicenter setting an improvement in EFS to 95% at 2 years of DA-EPOCH-R in patients with newly diagnosed high risk Burkitt lymphoma as compared to an expected EFS of 72% at 2 years for the control arm R-CODOX-M/…
Primary Objectives: * to establish - which is the best treatment, MTX or baricitinib, to ensure rapid symptom relief of recent onset UA, based on clinical and patient reported outcomes from baseline to 3 months. Secondary Objective: * to establish…
Primary:• To compare event-free survival (EFS) of subjects receiving blinatumomab alternating with low-intensity chemotherapy to EFS of subjects receiving standard of care (SOC) chemotherapy• To compare overall survival (OS) of blinatumomab…
To investigate wether the treatment of children and adolescents with AML can be improvedby means of:1) improved risk-group adapted treatment2) reduced treatment toxicity through shortened consolidation therapy.
Primary Objective: To determine whether in recently diagnosed PMR patients concomitant treatment to glucocorticoids with MTX 25 mg/week compared to a placebo will lead to a higher proportion of GC-free remission at 52 weeks. Secondary Objectives: To…
The primary objective of ALLTogether is to improve survival and quality of survival in children and young adults with acute lymphoblastic leukaemia
To compare the effectiveness of weekly subcutaneously administered MTX for maintaining relapse-free sustained steroid/EN-free 1-year remission compared with:- daily oral AZA/6MP in low risk paediatric CD- subcutaneously administered adalimumab in…