2024
The PAX LC Trial: A Decentralized, Phase 2, Randomized, Double-blind Study of Nirmatrelvir/Ritonavir Compared with Placebo/Ritonavir for Long COVID
Krumholz H, Sawano M, Bhattacharjee B, Caraballo C, Khera R, Li S, Herrin J, Coppi A, Holub J, Henriquez Y, Johnson M, Goddard T, Rocco E, Hummel A, Al Mouslmani M, Putrino D, Carr K, Carvajal-Gonzalez S, Charnas L, De Jesus M, Ziegler F, Iwasaki A. The PAX LC Trial: A Decentralized, Phase 2, Randomized, Double-blind Study of Nirmatrelvir/Ritonavir Compared with Placebo/Ritonavir for Long COVID. The American Journal Of Medicine 2024 PMID: 38735354, DOI: 10.1016/j.amjmed.2024.04.030.Peer-Reviewed Original ResearchLC trialPROMIS-29Participants' homesTargeting viral persistencePlacebo-controlled trialDouble-blind studyElectronic health recordsCore Outcome MeasuresLong COVIDEQ-5D-5LRepeated measures analysisEvidence-based treatmentsPhase 2Double-blindParticipant-centred approachStudy drugPrimary endpointSecondary endpointsCommunity-dwellingHealth recordsHealthcare utilizationContiguous US statesViral persistencePatient groupDrug treatment
2017
Postapproval studies of drugs initially approved by the FDA on the basis of limited evidence: systematic review
Pease AM, Krumholz HM, Downing NS, Aminawung JA, Shah ND, Ross JS. Postapproval studies of drugs initially approved by the FDA on the basis of limited evidence: systematic review. The BMJ 2017, 357: j1680. PMID: 28468750, PMCID: PMC5421452, DOI: 10.1136/bmj.j1680.Peer-Reviewed Original ResearchConceptsSingle pivotal trialPivotal trialsPostapproval studiesSurrogate markerPrimary endpointNovel drugsClinical outcomesClinical studiesLimited evidenceSystematic reviewDouble-blind studyMedian total numberClinical evidenceSuperior efficacyBlind studyDrug AdministrationOriginal FDATrial approvalDiseaseTrialsDrugsFDAEndpointEfficacyMarkers