biostudies-literatureRandell RLNICHD NIH HHSNIAID NIH HHSNHLBI NIH HHSFDA HHSNIMHD NIH HHSU.S. Food and Drug AdministrationNIAMS NIH HHSNIGMS NIH HHSe000494https://www.ebi.ac.uk/biostudies/studies/S-EPMC8108689biostudies-literatureUnknown8(1)<h4>Introduction</h4>Direct-to-family clinical trials efficiently provide data while reducing the participation burden for children and their families. Although these trials can offer significant advantages over traditional clinical trials, the process of designing and implementing direct-to-family studies is poorly defined, especially in children with rheumatic disease. This paper provides lessons learnt from the design and implementation of a self-controlled, direct-to-family pilot trial aimed to evaluate the effects of a medication management device on adherence to hydroxychloroquine in paediatric SLE.<h4>Methods</h4>Several design features accommodate a direct-to-family approach. Participants meeting eligibility criteria from across the USA were identified a priori through a disease reLupus science & medicineDelivering clinical trials at home: protocol, design and implementation of a direct-to-family paediatric lupus trial.PMC8108689R01 FD006099HHSN272201500006CHHSN272201300017CU19 AR069522K24 AI143971U18-FD006298HHSN275201000003CK23 AR075874U24 MD016258R61 HL147833R13 HD102136HHSN275201000003IU18 FD006298HHSN272201300017IR33 HL147833T32 GM086330Wampler Muskardin TSran RMisajon ETaneja ADover SClairman HOrandi ARafko JRuth NMcCurdy Stokes PDalrymple ARiordan MWoolnough LZeft RNigrovic PFalcon MBlakley MWalker JGotschlich ERitter MMcCurdy DKitcharoensakkul MGillispie-Taylor MVerbsky JTaber SNassi LHahn TLevy DLapin BPagano EWeiser PDedeoglu FGerhold KMiller MYu QManos COberle EPfeiffer TShelly ABenham HShenoi SMagni Manzoni SPepmueller PKienzle BHenrickson MStringer EPhillippi KPaller ARamirez CWaite HWhite ACorrell CMorgan Dewitt ETory HSavani SChang-Hoftman ASantiago LMcMullen-Jackson CZeft AKlein KZhu AHickey KPuplava BDrew JQuinlan-Waters MSchmeling HParedes JRamsey SHong SModica RReed ASeay TDu QHeiart JEdelheit BHance MDavis TDe Benedetti FRothman DTse SGoh IDavis CRodriquez MDavis ANelson BGriffin TOnel KAllenspach ESadun RNeely JTapani SGuittar PDe Guzman MGilbert MSule SWagner JBinstadt BCurry MSanni AFleming CBaszis KKlausmeier TBenseler SPonder LFailing CRandell RLFields KOlson JCurington TLai JShalen JSumner JKemp AJohnson NHoffart CBeltz ECohen-Wolkowiez MZartoshti AHiraki LDowling JGriswold JWeiss JChalom EMartyniuk ASchanberg LJanow GSchollaert-Fitch KFritz KAgbayani RBrothers MDriest KWeiss PCanna SNativ SMcGuire SSingler LLasky AMerritt ASyed RMcConnell KDuarte KStevens AStevens BAnderson EAkoghlanian SChundru KHarris JMercado RSrinivasalu HDean JVolpe NCooper JYomogida KUriguen SHillyer SLaxer RPardeo MRosenkranz MNanda KCooper SConlon HKaufman KSmitherman EArdoin SStewart KFox MNewhall LBigley TBohnsack JBarillas-Arias LInsalaco AMcHale IFox ECassidy ENicholas JSabbagh SCartwright VRadhakrishna SRakestraw AKramer SConfair AHager MFederici SStern SLingis MLo MSinger NVan Mater HVilar GWalters HVannoy LGuevara MKimura YAiello JThomas BFavier LKhalsa UProtopapas SWaqar LNicolai RChang JMason TGoldsmith DGoheer HMalla BMitchell MRonis TSchneider RQuach JSchlefman ABullock DBlack CLing NRichmond AMruk VKingsbury DImundo LWahezi DSoep JSzymanski AFroese SHollander MHobday PCerracchio LMellins ESilverman EWatson MBaker ELang BSchmidt KCo DLvovich SNahal BEckert MSchulert GWise AMohan SSletten JMendoza ETesher MFreeman MGrom AMaller JValcarcel TSpence SHarel LKompelien BYee MPratt SNasah NToib DHuiYuen JAlpizar MVehe RDonaldson BBecker MMannion MGiverc EEde KBeukelman TRamsay ESchikler KSaad NSon MSivaraman VShivers CRemmel KSmith CSmith AVora SSmith JNatter MJustiniano ARabinovich CLovell DCooper AOliver MPan NCunningham AFerrucho RStapp HParsons AGracia CRingold SVarghese CWright TJames KScalzi LMeidan EWilliams CTaylor JFerguson ISpiegel LMcAllister LPatel JHuber AWoo JMcCormick EToruner COsei-Onomah SReyes ASyverson GRojas KDickson TLin Cwith the CARRA Registry investigatorsRaisian JHersh AHartigan EStevenson RGeorge NFeldman BSpitznagle JTwilt MZhao YDingle JArmendariz SGuzman MLentini LTerry MVazzana KShergill KBuckley MMoussa TStoll MFadrhonc SHuggins JLapidus SObispo BRiebschleger MFerguson PHarry OMichalowski LShirley JSundel RMilojevic DMiettunen PLatham DHsu JKremer CMuscal ELuca NHornik CPBalevic SJBasiaga MHay ALieberman SRubinstein THill PFogel LHiskey MBrunner HDvergsten JHammelev ETate LTing TFair DChira PBoneparth ARothermel HFitzgerald MBracaglia CRodela ESutter MAbel NKracker JGottlieb BGrevich SNocton JPanupattanapong SLi SDorsey EJones JAlejandro CJones KBowman STambralli AFuhlbrigge RJones SMirizio EHausmann JLerman MBarbar-Smiley FCarper PJackson AHalyabar OMarques LFlynn OCostanzo DVasquez NFennell JHuttenlocher AJackson SAmarilyo GGodiwala CFranco LWaterfield MMoore KPooni RMoss CZhang YDempsey VO'Neil KWatts AReid HBukulmez HLim LYeung RDe Ranieri DJared STiffany KBalevic SPires Marafon DO'Brien BSacco EHanson AMadison CToth MOkeke OConnor RRiskalla MO'Brien TTaxter ANadler RStokes JBallinger SHayward KMcCarthy KKlein-Gitelman MCannon LDeSantis EMadison JMurphy ABell-Brunson HHenderson LZic CFinkel TTal RTanner TLee PRepp AKessler EMoorthy LVelez JInman CVeiga KOz RLawson EEdens CHuntington JFerreira BGotte AJustice CPentakota KHolland MTipp AAdams AKim SAdams MYen ESandborg CBallenger LCorvalan RHekl KJung LLee TTarvin SWershba EBrown AHorwitz MGerstbacher DScharnhorst SWu EBrooks EStingl CNolan BErvin BSeper CSchanberg LEChauhan VIbarra MElder Mvon Scheven EHaro SMathus SLowman DMalloy MRoth-Wojcicki EBernal WPrahalad SEberhard AAbulaban KAmbler WDuverger ECron RRouster-Stevens KSteigerwald KBoland JGraham TWagner-Weiner LKaran NBalboni IRosolowski BSarkissian AYalcindag AZapata RBalmuri NHung CDurkee DCameron BChinn TMcMonagle ATorok KKosikowski AThatayatikom ACuriel-Duran LJoshi SNg LAlperin RRandell RSterba Rakovchik YTarshish GDelnay NOrlando MEdgerly YSheets RRivas-Chacon RJaquith JSchmitt ETubwell VMorgan SRobinson AKovalick LFuller JLam JShishov MfalseDelivering clinical trials at home: protocol, design and implementation of a direct-to-family paediatric lupus trial.<h4>Introduction</h4>Direct-to-family clinical trials efficiently provide data while reducing the participation burden for children and their families. Although these trials can offer significant advantages over traditional clinical trials, the process of designing and implementing direct-to-family studies is poorly defined, especially in children with rheumatic disease. This paper provides lessons learnt from the design and implementation of a self-controlled, direct-to-family pilot trial aimed to evaluate the effects of a medication management device on adherence to hydroxychloroquine in paediatric SLE.<h4>Methods</h4>Several design features accommodate a direct-to-family approach. Participants meeting eligibility criteria from across the USA were identified a priori through a disease re2021-01-01T00:00:00Z2021 May2026-06-11T06:22:57.507Z2022-02-10T11:43:58.798ZS-EPMC81086893396308410.1136/lupus-2021-000494