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A recent trial by the ANZIC's group has indicated that a 7.8 point change in physical QOL domain scores is considered clinically relevant based on pilot trial data.
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While VPA appears safe and well-tolerated in this initial pilot trial, these data suggest that weight gain and carnitine depletion are likely to be significant confounding factors in clinical trials.
A pilot trial of data extraction will also be conducted to check the adequacy of the form and optimise it if necessary.
This pilot trial provides data to support the feasibility of the study in terms of recruitment and consent, the intervention and completion rates.
Although these results need to be validated in a large clinical trial, this pilot trial provides important data for clinical trial design of hypothermia treatment in neonatal hypoxic-ischemic injury.
For this pilot trial, participant-level data will not be publicly posted.
The pilot trial also collected data on resource use and health-related quality of life.
Pilot trials using the same design and methods as the subsequent main trial are ethically and financially advantageous especially when pilot and main trial data can be pooled.
This pilot trial provides feasibility data for undertaking a larger, powered randomized controlled trial, and provides justification for the importance of environment in relaxation interventions.
The sample size was also assumed to be adequate for testing the differences in patient-related change in laboratory parameters in this pilot trial (no preliminary data were available for calculations).
Parameters used in sample size calculations were estimated in our pilot trial and historical data from metabolic patients in our clinics, and we assumed complete randomization of the study subjects to each treatment arms.
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CEO of Professional Science Editing for Scientists @ prosciediting.com