The precision interventions for severe and/or exacerbation-prone asthma (PrecISE) adaptive platform trial: statistical considerations.
Anastasia IvanovaElliot IsraelLisa M LaVangeMichael C PetersLoren C DenlingerWendy C MooreLeonard B BacharierM Alison MarquisNathan M GotmanMichael R KosorokChalmer TomlinsonDavid T MaugerSteve N GeorasRosalind J WrightPatricia NoelGary L RosnerPraveen AkuthotaDean BillheimerEugene R BleeckerJuan Carlos CardetMario CastroEmily A DiMangoSerpil C ErzurumJohn V FahyMerritt L FajtBenjamin M GastonFernando HolguinSonia JainNicholas J KenyonJerry A KrishnanMonica KraftRajesh KumarMark C LiuNgoc P LyJames N MoyWanda PhipatanakulKristie RossLewis J SmithStanley J SzeflerW Gerald TeagueMichael E WechslerSally E WenzelSteven R WhitePublished in: Journal of biopharmaceutical statistics (2020)
The Precision Interventions for Severe and/or Exacerbation-prone Asthma (PrecISE) study is an adaptive platform trial designed to investigate novel interventions to severe asthma. The study is conducted under a master protocol and utilizes a crossover design with each participant receiving up to five interventions and at least one placebo. Treatment assignments are based on the patients' biomarker profiles and precision health methods are incorporated into the interim and final analyses. We describe key elements of the PrecISE study including the multistage adaptive enrichment strategy, early stopping of an intervention for futility, power calculations, and the primary analysis strategy.
Keyphrases
- chronic obstructive pulmonary disease
- physical activity
- randomized controlled trial
- healthcare
- study protocol
- public health
- end stage renal disease
- high throughput
- phase iii
- ejection fraction
- newly diagnosed
- early onset
- peritoneal dialysis
- double blind
- risk assessment
- air pollution
- social media
- density functional theory