References
- HealyBCSchoenfeldDComparison of analysis approaches for phase III clinical trials in amyotrophic lateral sclerosisMuscle Nerve201246450651122987690
- BerryJDCudkowiczMENew considerations in the design of clinical trials for amyotrophic lateral sclerosisClin Investig (Lond)201111013751389
- MillerRGMooreDHALS trial design: expectation and realityAmyotroph Lateral Scler Other Motor Neuron Disord20045suppl 1525415512873
- RutkoveSBClinical measures of disease progression in amyotrophic lateral sclerosisNeurotherapeutics201512238439325582382
- ShefnerJMDesigning clinical trials in amyotrophic lateral sclerosisPhys Med Rehabil Clin N Am2008193495508ix18625412
- GordonPHChengBMontesJDoorishCAlbertSMMitsumotoHOutcome measures for early phase clinical trialsAmyotroph Lateral Scler20078527027317852017
- GordonPHCorciaPLacomblezLDefining survival as an outcome measure in amyotrophic lateral sclerosisArch Neurol200966675876119506136
- SimonNGTurnerMRVucicSQuantifying disease progression in amyotrophic lateral sclerosisAnn Neurol201476564365725223628
- GordonPHMeiningerVHow can we improve clinical trials in amyotrophic lateral sclerosis?Nat Rev Neurol201171165065421947135
- BerryJDMillerRMooreDHThe combined assessment of function and survival (CAFS): a new endpoint for ALS clinical trialsAmyotroph Lateral Scler Frontotemporal Degener201314316216823323713
- CudkowiczMEvan den BergLHShefnerJMEMPOWER InvestigatorsDexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trialLancet Neurol201312111059106724067398
- MeiningerVGengeAvan den BergLHNOG112264 Study GroupSafety and efficacy of ozanezumab in patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled, phase 2 trialLancet Neurol201716320821628139349
- LacomblezLBensimonGLeighPNGuilletPMeiningerVDose-ranging study of riluzole in amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis/Riluzole Study Group IILancet19963479013142514318676624
- RizopoulosDJoint Models for Longitudinal and Time-to-Event Data: With Applications in RBoca RatonCRC Press2012
- Group UK-LSMorrisonKEDhariwalSLithium in patients with amyotrophic lateral sclerosis (LiCALS): a phase 3 multicentre, randomised, double-blind, placebo-controlled trialLancet Neurol201312433934523453347
- AggarwalSPZinmanLSimpsonENortheast and Canadian Amyotrophic Lateral Sclerosis ConsortiaSafety and efficacy of lithium in combination with riluzole for treatment of amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trialLancet Neurol20109548148820363190
- AtassiNBerryJShuiAThe PRO-ACT database: design, initial analyses, and predictive featuresNeurology201483191719172525298304
- HommelGA stagewise rejective multiple test procedure based on a modified Bonferroni testBiometrika1988752383386
- IbrahimJGChuHChenLMBasic concepts and methods for joint models of longitudinal and survival dataJ Clin Oncol201028162796280120439643
- FinkelsteinDMSchoenfeldDACombining mortality and longitudinal measures in clinical trialsStat Med199918111341135410399200
- van EijkRPAEijkemansMJCFergusonTANikolakopoulosSVeldinkJHvan den BergLHMonitoring disease progression with plasma creatinine in amyotrophic lateral sclerosis clinical trialsJ Neurol Neurosurg Psychiatry201789215616129084868
- ChenLMIbrahimJGChuHSample size and power determination in joint modeling of longitudinal and survival dataStat Med201130182295230921590793
- GordonPHChengBSalachasFProgression in ALS is not linear but is curvilinearJ Neurol2010257101713171720532545
- BeghiEChioAInghilleriMA randomized controlled trial of recombinant interferon beta-1a in ALS. Italian Amyotrophic Lateral Sclerosis study groupNeurology200054246947410668716
- BeghiEPupilloEBonitoVItalian ALS Study GroupRandomized double-blind placebo-controlled trial of acetyl-L-carnitine for ALSAmyotroph Lateral Scler Frontotemporal Degener2013145–639740523421600