7 results
Primary:*To identify the maximum tolerated dose (MTD) and to investigate the pharmacokinetics (PK) of a single dose of lanreotide PRF in subjects with acromegalySecondary:*To investigate the safety and tolerability of a single dose of lanreotide PRF…
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…
The primary objectives of this study are: * To characterize the effects of 150 mg and 450 mg of AT1001 administered 2 hours before administration of agalsidase on the safety and plasma pharmacokinetics of agalsidase in subjects with Fabry Disease*…
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…
Primary Objective* To describe the antitumour efficacy of LAN monotherapy plus BSC every 28 days, in terms of progression-free survival (PFS), measured by central review using Response Evaluation Criteria in Solid Tumours (RECIST) v1.1 criteria,…
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,…