Multicentre trial

Antidrug antibody responses were similar in both groups. Interpretation: We consider that this study provides evidence of clinically meaningful improvement with avalglucosidase alfa therapy over alglucosidase alfa in respiratory function, ambulation, and functional endurance, with no new safety signals reported.

An open-label extended-treatment period is ongoing to confirm the long-term safety and efficacy of avalglucosidase alfa, with the aim for this therapy to become the new standard treatment in late-onset Pompe disease.

Funding: Sanofi Genzyme. Associated data ClinicalTrials. However, the numerical reduction in time to clinical improvement in those treated earlier requires confirmation in larger studies. Abstract Background: No specific antiviral drug has been proven effective for treatment of patients with severe coronavirus disease COVID Associated data ClinicalTrials.

Problems and solutions Many single centre trials often recruit too few patients to be scientifically viable. A trial with only a small number of participants carries a considerable risk of failing to demonstrate a treatment difference when one is really present i.

Often a single source of participants may be insufficient to make a clinical trial of viable size. This problem may be clear-cut from the beginning but on other occasions it may linger on with too few participants and finally peter out as enthusiasm wanes. Thus, it is important to recognise early on whether a single centre trial is feasible and scrutinise the ethics, organisation and relevance of it carefully.

Multicentre trials Advantages Multicentre trials are carried out for two main reasons. Firstly, a multicentre trial is an accepted way of evaluating a new technology more efficiently; under some circumstances, it may present the only practical means of accruing sufficient subjects to satisfy the trial objective within a reasonable time frame.

Multicentre trials of this nature may, in principle, be carried out at any stage of clinical development. They may have several centres with a large number of subjects per centre or, in the case of a rare disease, they may have a large number of centres with very few subjects per centre.