2025
Linzagolix with and without hormonal add-back therapy for symptomatic uterine fibroids: PRIMROSE 1 & 2 long-term extension and withdrawal study
Donnez J, Petraglia F, Taylor H, Becker C, Becker S, Herrera F, Bestel E, Hori S, Dolmans M. Linzagolix with and without hormonal add-back therapy for symptomatic uterine fibroids: PRIMROSE 1 & 2 long-term extension and withdrawal study. Fertility And Sterility 2025 PMID: 40543832, DOI: 10.1016/j.fertnstert.2025.06.016.Peer-Reviewed Original ResearchHormonal add-back therapyAdd-back therapyHeavy menstrual bleedingMenstrual blood lossSymptomatic uterine fibroidsCessation of therapyMenstrual bleedingBone mineral densityUterine fibroidsBlood lossWithdrawal studyMineral densityPlacebo-controlled phase 3 trialTreatment of symptomatic uterine fibroidsDecreased menstrual blood lossReducing menstrual blood lossRisk of bone lossReducing heavy menstrual bleedingExtension studySignificantly higher proportion of womenMonths of therapyFibroid-associated symptomsPhase 3 trialAlkaline haematin methodBenefits of treatmentSafety and efficacy with esketamine in treatment-resistant depression: long-term extension study
Zaki N, Chen L, Lane R, Doherty T, Drevets W, Morrison R, Sanacora G, Wilkinson S, Young A, Lacerda A, Paik J, Popova V, Fu D. Safety and efficacy with esketamine in treatment-resistant depression: long-term extension study. The International Journal Of Neuropsychopharmacology 2025, 28: pyaf027. PMID: 40319349, PMCID: PMC12143125, DOI: 10.1093/ijnp/pyaf027.Peer-Reviewed Original ResearchConceptsTreatment-resistant depressionSustained 3Oral antidepressantNon-treatment-resistant major depressive disorderMontgomery-Asberg Depression Rating ScaleTreatment-resistant depression treatmentEsketamine nasal sprayDepression Rating ScaleMajor depressive disorderLong-term extension studySafety of esketamineLong-term safetyRate of relapseMontgomery-AsbergDepressive disorderDepression severityEsketamine dosesExtension studySuicide riskDepressive symptomsEsketamineRating ScaleMaintenance treatmentAdverse eventsLong-term treatmentThe Safety Profile of Pridopidine, a Novel Sigma-1 Receptor Agonist for the Treatment of Huntington’s Disease
Goldberg Y, Navon-Perry L, Cruz-Herranz A, Chen K, Hecker-Barth G, Spiegel K, Cohen Y, Niethammer M, Tan A, Schuring H, Geva M, Hayden M. The Safety Profile of Pridopidine, a Novel Sigma-1 Receptor Agonist for the Treatment of Huntington’s Disease. CNS Drugs 2025, 39: 485-498. PMID: 40055280, PMCID: PMC11982116, DOI: 10.1007/s40263-025-01171-x.Peer-Reviewed Original ResearchSafety profileAdverse eventsAntidopaminergic medicationsOpen-label extension studySigma-1Sigma-1 receptor agonistCytosine-adenine-guaninePlacebo-controlled studySpectrum of disease severityAnalysis of pooled dataTotal functional capacityFirst-in-classRecommended human doseSigma-1 receptorTreatment of Huntington's diseaseUnmet medical needLong-term useDouble-blindReceptor agonistsPridopidineHuman doseSafety signalsEffective treatmentPlaceboExtension study
2024
Glycemic Outcomes Persist for up to 2 Years in Very Young Children with the Omnipod® 5 Automated Insulin Delivery System
DeSalvo D, Bode B, Forlenza G, Laffel L, Buckingham B, Criego A, Schoelwer M, MacLeish S, Sherr J, Hansen D, Ly T. Glycemic Outcomes Persist for up to 2 Years in Very Young Children with the Omnipod® 5 Automated Insulin Delivery System. Diabetes Technology & Therapeutics 2024, 26: 383-393. PMID: 38277156, DOI: 10.1089/dia.2023.0506.Peer-Reviewed Original ResearchType 1 diabetesExtension phaseEpisodes of diabetic ketoacidosisEpisodes of severe hypoglycemiaLong-term safetyLong-term useStandard therapyDiabetic ketoacidosisAutomated Insulin DeliverySevere hypoglycemiaYoung childrenConclusion:</i></b>Continuous glucose monitoring metricsExtension studyMethods:</i></b>MonthsGlycemic outcomesTarget rangeTrialsOmnipodHbA1cInsulin deliveryDelivery systemInsulin delivery systemsEpisodes
2023
Comparison of two assessments of real‐world data and real‐world evidence for regulatory decision‐making
Yuan L, Rahman M, Concato J. Comparison of two assessments of real‐world data and real‐world evidence for regulatory decision‐making. Clinical And Translational Science 2023, 17: e13702. PMID: 38093484, PMCID: PMC10766019, DOI: 10.1111/cts.13702.Peer-Reviewed Original ResearchReal-world evidenceEuropean Medicines AgencyDrug approvalOpen-label extension studyNon-interventional studySingle-arm trialClinical trial implementationUnited States FoodInterventional trialsComparator armDrug AdministrationMedicines AgencyExtension studyStudy designUse of RWEStates FoodPharmacovigilance activitiesTrialsRegulatory agenciesApprovalLack of agreementTrial implementationLiterature reviewTherapeutic contextReal-world dataExtension Phase of a Multi-Center, Randomized, Blinded Clinical Study Evaluating the Efficacy and Safety of a Novel Topical Product for Facial Dyschromia.
Wang J, Fabi S, Robinson D, Bajaj S, Geronemus R, Bell M, Robison T, Widgerow A. Extension Phase of a Multi-Center, Randomized, Blinded Clinical Study Evaluating the Efficacy and Safety of a Novel Topical Product for Facial Dyschromia. Journal Of Drugs In Dermatology 2023, 23: 1266-1270. PMID: 38206144, DOI: 10.36849/jdd.7622.Peer-Reviewed Original ResearchConceptsFacial dyschromiaMulti-center trialClinical studiesBlinded clinical studyTopical productsALS cohortExtension studyHydroquinone 4%Follow-up visitExtension phaseClearance of pigmentAssociated with increased productionMulti-centerDaily sunscreen useReduced clearanceCohortDyschromiaSunscreen useIn vitroWeeksTrialsLong-term basisSignificant reboundSkinEfficacySafety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study
Howard J, Bresch S, Genge A, Hewamadduma C, Hinton J, Hussain Y, Juntas-Morales R, Kaminski H, Maniaol A, Mantegazza R, Masuda M, Sivakumar K, Śmiłowski M, Utsugisawa K, Vu T, Weiss M, Zajda M, Boroojerdi B, Brock M, de la Borderie G, Duda P, Lowcock R, Vanderkelen M, Leite M, Team R, Sembinelli D, Teitelbaum J, Nicolle M, Bernard E, Svahn J, Spinazzi M, Stojkovic T, Demeret S, Weiss N, Le Guennec L, Messai S, Tranchant C, Nadaj-Pakleza A, Chanson J, Suliman M, Zaidi L, Tard C, Lecointe P, Zschüntzsch J, Schmidt J, Glaubitz S, Zeng R, Scholl M, Kowarik M, Ziemann U, Krumbholz M, Martin P, Ruschil C, Dünschede J, Kemmner R, Rumpel N, Berger B, Totzeck A, Hagenacker T, Stolte B, Iorio R, Evoli A, Falso S, Antozzi C, Frangiamore R, Vanoli F, Rinaldi E, Deguchi K, Minami N, Nagane Y, Suzuki Y, Ishida S, Suzuki S, Nakahara J, Nagaoka A, Yoshimura S, Konno S, Tsuya Y, Uzawa A, Kubota T, Takahashi M, Okuno T, Murai H, Gilhus N, Boldingh M, Rønning T, Chyrchel-Paszkiewicz U, Kumor K, Zielinski T, Banaszkiewicz K, Błaż M, Kłósek A, Świderek-Matysiak M, Szczudlik A, Paśko A, Szczechowski L, Banach M, Ilkowski J, Garcia S, Bagan P, Segura A, Sans J, Fernandez N, Vicente E, Armenteros P, Ashraghi M, Cavey A, Haslam L, Emery A, Liow K, Yegiaian S, Barboi A, Vazquez R, Lennon J, Pascuzzi R, Bodkin C, Guingrich S, Comer A, Bromberg M, Janecki T, Saba S, Tellez M, Elsheikh B, Freimer M, Heintzman S, Govindarajan R, Guptill J, Massey J, Juel V, Gonzalez N, Habib A, Mozaffar T, Korb M, Goyal N, Machemehl H, Manousakis G, Allen J, Harper E, Farmakidis C, Saavedra L, Dimachkie M, Pasnoor M, Akhter S, Beydoun S, McIlduff C, Nye J, Roy B, Sheldon B, Nowak R, Barnes B, Rivner M, Suresh N, Shaw J, Harvey B, Lam L, Thomas N, Chopra M, Traub R, Jones S, Wagoner M, Smajic S, Aly R, Katz J, Chen H, Miller R, Jenkins L, Khan S, Khatri B, Sershon L, Pavlakis P, Holzberg S, Li Y, Caristo I, Marquardt R, Hastings D, Rube J, Lisak R, Choudhury A, Ruzhansky K, Sachdev A, Shin S, Bratton J, Fetter M, McKinnon N, McKinnon J, Sissons-Ross L, Sahu A, Distad B. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. The Lancet Neurology 2023, 22: 395-406. PMID: 37059508, DOI: 10.1016/s1474-4422(23)00080-7.Peer-Reviewed Original ResearchConceptsTreatment-emergent adverse eventsGeneralised myasthenia gravisOpen-label extension studyMG-ADL scoresBaseline to weekMyasthenia gravisDouble-blindPlacebo-ControlledIncidence of treatment-emergent adverse eventsMG-ADLModified intention-to-treat populationIntention-to-treat populationExtension studyQuantitative Myasthenia Gravis ScoreComplement C5 inhibitorInjection-site bruisingPhase 3 studyPhase 3 trialPopulation of patientsLong-term safetyPotential treatment optionMyasthenia Gravis ActivitiesClinically Meaningful ImprovementsMyasthenia Gravis Activities of Daily LivingMatching placebo
2022
Reinfection and Risk Behaviors After Treatment of Hepatitis C Virus Infection in Persons Receiving Opioid Agonist Therapy : A Cohort Study.
Grebely J, Dore GJ, Altice FL, Conway B, Litwin AH, Norton BL, Dalgard O, Gane EJ, Shibolet O, Nahass R, Luetkemeyer AF, Peng CY, Iser D, Gendrano IN, Kelly MM, Hwang P, Asante-Appiah E, Haber BA, Barr E, Robertson MN, Platt H. Reinfection and Risk Behaviors After Treatment of Hepatitis C Virus Infection in Persons Receiving Opioid Agonist Therapy : A Cohort Study. Annals Of Internal Medicine 2022, 175: 1221-1229. PMID: 35939812, DOI: 10.7326/m21-4119.Peer-Reviewed Original ResearchConceptsOpioid agonist therapyLong-term extension studyHCV reinfectionDrug useExtension studyAgonist therapySuccessful treatmentHepatitis C virus (HCV) reinfectionHepatitis C virus infectionBenefits of cureC virus infectionChronic HCV infectionRate of reinfectionUrine drug screeningOngoing drug useClinical trial sitesSubsidiary of MerckHCV infectionCohort studyVirus reinfectionDohme Corp.Treatment completionGenotype 1Virus infectionHigh risk33-OR: ADA Presidents' Select Abstract: Glycemic Outcomes over 12 Months in Very Young Children with the Omnipod 5 Automated Insulin Delivery (AID) System
DESALVO D, BODE B, FORLENZA G, LAFFEL L, BUCKINGHAM B, CRIEGO A, SCHOELWER M, MACLEISH S, SHERR J, HANSEN D, LY T. 33-OR: ADA Presidents' Select Abstract: Glycemic Outcomes over 12 Months in Very Young Children with the Omnipod 5 Automated Insulin Delivery (AID) System. Diabetes 2022, 71 DOI: 10.2337/db22-33-or.Peer-Reviewed Original ResearchDiabetic ketoacidosisSevere hypoglycemiaMannKind CorporationGlycemic outcomesExtension studyEpisodes of diabetic ketoacidosisOccurrence of severe hypoglycemiaNational Institute of DiabetesDigestive and Kidney DiseasesCenter for Health ResearchChildren Aged 2Type 1 diabetesStandard therapySafety endpointsPivotal studiesPotential long-term benefitsKidney diseaseStudy groupGlucose targetsLong-term benefitsSpeakers bureauImprove glycemic outcomesAged 2MedtronicGlycemic benefitsNovel Pharmacological Agents for the Treatment of Cocaine Use Disorder
Hadizadeh H, Flores J, Nunes E, Mayerson T, Potenza M, Angarita G. Novel Pharmacological Agents for the Treatment of Cocaine Use Disorder. Current Behavioral Neuroscience Reports 2022, 9: 27-46. DOI: 10.1007/s40473-022-00246-z.Peer-Reviewed Original ResearchCocaine use disorderNovel pharmacological agentsUse disordersCocaine vaccinePharmacological agentsClinical supportNarrative reviewAlpha-adrenergic antagonistKappa-opioid receptorsCorticotropin-releasing factorCentral nervous systemPurpose of reviewClinical evidenceGlutamatergic agentsSerotonergic agentsCholinergic agentsOrexin antagonistsNeurobiological targetsDopamine pathwayArousal pathwayNervous systemExtension studyPromising agentCapacity of cocaineDisorders
2021
Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study
Siddiqi ZA, Nowak RJ, Mozaffar T, O'Brien F, Yountz M, Patti F, Mazia C, Wilken M, Wilken M, Barroso F, Saba J, Rugiero M, Bettini M, Chaves M, Vidal G, Garcia A, De Bleecker J, Van den Abeele G, Koning K, De Mey K, Mercelis R, Mahieu D, Wagemaekers L, Van Damme P, Depreitere A, Schotte C, Smetcoren C, Stevens O, Van Daele S, Vandenbussche N, Vanhee A, Verjans S, Vynckier J, D'Hont A, Tilkin P, Siqueira Carvalho A, Brockhausen I, Feder D, Ambrosio D, César P, Melo A, Ribeiro R, Rocha R, Rosa B, Veiga T, Silva L, Engel M, Geraldo J, Penha Ananias Morita M, Coelho E, Paiva G, Pozo M, Prando N, Torres D, Butinhao C, Duran G, Fialho T, Silva T, Gonçalves L, Pazetto L, Volpe L, Duca L, Friedrich M, Guerreiro A, Mohr H, Martins M, Cruz Pacheco D, Ferreira L, Macagnan A, Pinto G, Cassia Santos A, Oliveira A, Andrade A, Annes M, Silva L, Lino V, Pinto W, Assis N, Carrara F, Miranda C, Souza I, Fernandes P, Phan C, Narayan J, Blackmore D, Mallon A, Roderus R, Watt E, Vohanka S, Bednarik J, Chmelikova M, Cierny M, Toncrova S, Junkerova J, Kurkova B, Reguliova K, Zapletalova O, Pitha J, Novakova I, Tyblova M, Jurajdova I, Wolfova M, Andersen H, Harbo T, Vinge L, Krogh S, Mogensen A, Vissing J, Højgaard J, Witting N, Autzen A, Pedersen J, Erälinna J, Laaksonen M, Oksaranta O, Harrison T, Eriksson J, Rozsa C, Horvath M, Lovas G, Matolcsi J, Szabo G, Jakab G, Szabadosne B, Vecsei L, Dezsi L, Varga E, Konyane M, Antonini G, Di Pasquale A, Garibaldi M, Morino S, Troili F, Fionda L, Di Pasquale A, Garibaldi M, Saccà F, Filla A, Costabile T, Marano E, Fasanaro A, Marsili A, Puorro G, Mantegazza R, Antozzi C, Bonanno S, Camera G, Locatelli A, Maggi L, Pasanisi M, Campanella A, Evoli A, Alboini P, D'Amato V, Iorio R, Inghilleri M, Fionda L, Frasca V, Giacomelli E, Gori M, Lopergolo D, Onesti E, Frasca V, Gabriele M, Uzawa A, Kanai T, Kawaguchi N, Mori M, Kaneko Y, Kanzaki A, Kobayashi E, Murai H, Masaki K, Matsuse D, Matsushita T, Uehara T, Shimpo M, Jingu M, Kikutake K, Nakamura Y, Sano Y, Utsugisawa K, Nagane Y, Kamegamori I, Tsuda T, Fujii Y, Futono K, Ozawa Y, Mizugami A, Saito Y, Samukawa M, Suzuki H, Morikawa M, Kamakura S, Miyawaki E, Shiraishi H, Miyazaki T, Motomura M, Mukaino A, Yoshimura S, Asada S, Yoshida S, Amamoto S, Kobashikawa T, Koga M, Maeda Y, Takada K, Takada M, Tsurumaru M, Yamashita Y, Yoshida S, Suzuki Y, Akiyama T, Narikawa K, Tano O, Tsukita K, Kurihara R, Meguro F, Fukuda Y, Sato M, Okumura M, Funaka S, Kawamura T, Nakamori M, Takahashi M, Taichi N, Hasuike T, Higuchi E, Kobayashi H, Osakada K, Imai T, Tsuda E, Shimohama S, Hayashi T, Hisahara S, Imai T, Kawamata J, Murahara T, Saitoh M, Shimohama S, Suzuki S, Yamamoto D, Ishiyama Y, Ishiyama N, Noshiro M, Takeyama R, Uwasa K, Yasuda I, Kim B, Lee C, Koo Y, Seok H, Kang H, Ra H, Kim B, Cho E, Choi M, Lee H, Min J, Seok J, Lee J, Koh D, Kwon J, Park S, Choi E, Hong Y, Ahn S, Koo D, Lim J, Shin C, Hwang J, Kim M, Kim S, Jeong H, Jung J, Kim Y, Lee H, Shin H, Hwang E, Shin M, Kooi A, Visser M, Gibson T, Casasnovas C, Aguilo M, Homedes‐Pedret C, Palacios N, Porras L, Santamaria V, Lazaro A, Tejedor E, Salcedo P, Fernandez‐Fournier M, Ruiz P, Rivera F, Fernandez‐Fournier M, Sastre M, Carbonell J, Sune P, Figueras M, Gili G, Mazuela G, Illa I, Vicente E, Diaz‐Manera J, Gutiérrez L, Garcia R, Vidal N, Arribas‐Ibar E, Piehl F, Hietala A, Bjarbo L, Sengun I, Meherremova A, Ozcelik P, Balkan B, Tuga C, Ugur M, Erdem‐Ozdamar S, Bekircan‐Kurt C, Acar N, Yilmaz E, Caliskan Y, Orsel G, Efendi H, Aydinlik S, Cavus H, Kutlu A, Becerikli G, Semiz C, Tun O, Terzi M, Dogan B, Onar M, Sen S, Cavdar T, Veske A, Norwood F, Dimitriou A, Gollogly J, Mahdi‐Rogers M, Seddigh A, Sokratous G, Maier G, Sohail F, Jacob S, Sadalage G, Torane P, Brown C, Shah A, Sathasivam S, Arndt H, Davies D, Watling D, Amato A, Cochrane T, Salajegheh M, Roe K, Amato K, Toska S, Wolfe G, Silvestri N, Patrick K, Zakalik K, Katz J, Miller R, Engel M, Forshew D, Bravver E, Brooks B, Sanjak M, Plevka S, Burdette M, Cunningham S, Sanjak M, Kramer M, Nemeth J, Schommer C, Scott T, Juel V, Guptill J, Hobson‐Webb L, Massey J, Beck K, Carnes D, Loor J, Anderson A, Pascuzzi R, Bodkin C, Kincaid J, Snook R, Guingrich S, Micheels A, Chaudhry V, Corse A, Mosmiller B, Kelley A, Ho D, Srinivasan J, Vytopil M, Jara J, Ventura N, Carter C, Donahue C, Herbert C, Scala S, Weiner E, Alam S, McKinnon J, Haar L, McKinnon N, Alcon K, McKenna K, Sattar N, Daniels K, Jeffery D, Freimer M, Hoyle J, Kissel J, Agriesti J, Chelnick S, Mezache L, Pineda C, Muharrem F, Karam C, Khoury J, Marburger T, Kaur H, Dimitrova D, Gilchrist J, Agrawal B, Elsayed M, Kohlrus S, Ardoin A, Darnell T, Golden L, Lokaitis B, Seelbach J, Muppidi S, Goyal N, Sakamuri S, So Y, Paulose S, Pol S, Welsh L, Bhavaraju‐Sanka R, Gonzalez A, Dishman L, Jones F, Gonzalez A, Padilla P, Saklad A, Silva M, Nations S, Trivedi J, Hopkins S, Kazamel M, Alsharabati M, Lu L, Nozaki K, Mumfrey‐Thomas S, Woodall A, Cash T, Goyal N, Roy G, Mathew V, Maqsood F, Minton B, Jones H, Rosenfeld J, Garcia R, Echevarria L, Garcia S, Pulley M, Aranke S, Berger A, Shah J, Shabbir Y, Smith L, Varghese M, Shabbir Y, Gutmann L, Gutmann L, Jerath N, Nance C, Swenson A, Olalde H, Kressin N, Sieren J, Barohn R, Dimachkie M, Glenn M, McVey A, Pasnoor M, Statland J, Wang Y, Liu T, Emmons K, Jenci N, Locheke J, Fondaw A, Johns K, Rico G, Walsh M, Herbelin L, Hafer‐Macko C, Kwan J, Zilliox L, Callison K, Young V, DiSanzo B, Naunton K, Benatar M, Bilsker M, Sharma K, Cooley A, Reyes E, Michon S, Sheldon D, Steele J, Howard J, Karam C, Traub R, Chopra M, Vu T, Katzin L, McClain T, Harvey B, Hart A, Huynh K, Beydoun S, Chilingaryan A, Doan V, Droker B, Gong H, Karimi S, Lin F, McClain T, Polaka K, Shah A, Tran A, Akhter S, Malekniazi A, Tandan R, Hehir M, Waheed W, Lucy S, Weiss M, Distad J, Strom S, Downing S, Kim B, Bertorini T, Arnold T, Henderson K, Pillai R, Liu Y, Wheeler L, Hewlett J, Vanderhook M, Dicapua D, Keung B, Kumar A, Patwa H, Robeson K, Yang I, Nye J, Vu H. Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study. Muscle & Nerve 2021, 64: 662-669. PMID: 34590717, DOI: 10.1002/mus.27422.Peer-Reviewed Original ResearchConceptsRituximab useMyasthenia gravisSubgroup analysisAmerica Post-Intervention StatusMyasthenia Gravis ActivitiesSafety of eculizumabOpen-label extensionMyasthenia Gravis FoundationUnmet treatment needDaily living (IADL) total scorePost-intervention statusEculizumab treatmentWeek 130Rituximab treatmentBaseline characteristicsMost patientsPersistent symptomsSafety profileEffective therapyTreatment needsEculizumabMinimal manifestationsExtension studyREGAIN studyPatients
2019
Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study
Murai H, Uzawa A, Suzuki Y, Imai T, Shiraishi H, Suzuki H, Okumura M, O’Brien F, Wang J, Fujita K, Utsugisawa K, Group I, Mazia C, Wilken M, Barroso F, Saba J, Rugiero M, Bettini M, Chaves M, Vidal G, Garcia A, De Bleecker J, Van den Abeele G, de Koning K, De Mey K, Mercelis R, Mahieu D, Wagemaekers L, Van Damme P, Depreitere A, Schotte C, Smetcoren C, Stevens O, Van Daele S, Vandenbussche N, Vanhee A, Verjans S, Vynckier J, D'Hondt A, Tilkin P, de Siqueira Carvalho A, Brockhausen I, Feder D, Ambrosio D, César P, Melo A, Ribeiro R, Rocha R, Rosa B, Veiga T, da Silva L, Engel M, Geraldo J, da Penha Ananias Morita M, Coelho E, Paiva G, Pozo M, Prando N, Torres D, Butinhao C, Duran G, da Silva T, Gonçalves L, Pazetto L, Fialho T, Volpe L, Duca L, Oliveira A, Andrade A, Annes M, Silva L, Lino V, Pinto W, Assis N, Carrara F, Miranda C, Souza I, Fernandes P, Siddiqi Z, Phan C, Narayan J, Blackmore D, Mallon A, Roderus R, Watt E, Junkerova J, Kurkova B, Reguliova K, Zapletalova O, Pitha J, Novakova I, Tyblova M, Jurajdova I, Wolfova M, Andersen H, Harbo T, Vinge L, Krogh S, Mogensen A, Vissing J, Højgaard J, Witting N, Autzen A, Pedersen J, Eralinna J, Laaksonen M, Oksaranta O, Harrison T, Eriksson J, Rozsa C, Horvath M, Lovas G, Matolcsi J, Szabo G, Jakab G, Szabadosne B, Antonini G, Di Pasquale A, Garibaldi M, Morino S, Troili F, Fionda L, Filla A, Costabile T, Marano E, Saccà F, Fasanaro A, Marsili A, Puorro G, Mantegazza R, Antozzi C, Bonanno S, Camera G, Locatelli A, Maggi L, Pasanisi M, Campanella A, Evoli A, Alboini P, D'Amato V, Iorio R, Kanai T, Kawaguchi N, Mori M, Kaneko Y, Kanzaki A, Kobayashi E, Masaki K, Matsuse D, Matsushita T, Uehara T, Shimpo M, Jingu M, Kikutake K, Nakamura Y, Sano Y, Nagane Y, Kamegamori I, Tsuda T, Fujii Y, Futono K, Ozawa Y, Mizugami A, Saito Y, Morikawa M, Samukawa M, Kamakura S, Miyawaki E, Mitazaki T, Motomura M, Mukaino A, Yoshimura S, Asada S, Yoshida S, Amamoto S, Kobashikawa T, Koga M, Maeda Y, Takada K, Takada M, Tsurumaru M, Yamashita Y, Akiyama T, Narikawa K, Tano O, Tsukita K, Kurihara R, Meguro F, Fukuda Y, Sato M, Funaka S, Kawamura T, Makamori M, Takahashi M, Taichi N, Hasuike T, Higuchi E, Kobayashi H, Osakada K, Tsuda E, Shimohama S, Hayashi T, Hisahara S, Kawamata J, Murahara T, Saitoh M, Suzuki S, Yamamoto D, Ishiyama Y, Ishiyama N, Noshiro M, Takeyama R, Uwasa K, Yasuda I, van der Kooi A, de Visser M, Gibson T, Casasnovas C, Aguilo M, Homedes-Pedret C, Palacios N, Porras L, Santamaria V, Lazaro A, Tejedor E, Salcedo P, Fernandez-Fournier M, Ruiz P, de Rivera F, Sastre M, Gamez J, Sune P, Salvado M, Gili G, Mazuela G, Illa I, Vicente E, Diaz-Manera J, Gutierrez L, Garcia R, Vidal N, Arribas-Ibar E, Piehl F, Hietala A, Bjarbo L, Sengun I, Meherremova A, Ozcelik P, Balkan B, Tuga C, Ugur M, Erdem-Ozdamar S, Bekircan-Kurt C, Acar N, Yilmaz E, Caliskan Y, Orsel G, Efendi H, Aydinlik S, Cavus H, Kutlu A, Becerikli G, Semiz C, Tun O, Terzi M, Dogan B, Onar M, Sen S, Cavdar T, Veske A, Norwood F, Dimitriou A, Gollogly J, Mahdi-Rogers M, Seddigh A, Sokratous G, Maier G, Sohail F, Jacob S, Sadalage G, Torane P, Brown C, Shah A, Sathasivam S, Arndt H, Davies D, Watling D, Amato A, Cochrane T, Salajegheh M, Roe K, Amato K, Toska S, Wolfe G, Silvestri N, Patrick K, Zakalik K, Katz J, Miller R, Engel M, Forshew D, Bravver E, Brooks B, Plevka S, Burdette M, Cunningham S, Sanjak M, Kramer M, Nemeth J, Schommer C, Tierney S, Juel V, Guptill J, Hobson-Webb L, Massey J, Beck K, Carnes D, Loor J, Anderson A, Pascuzzi R, Bodkin C, Kincaid J, Snook R, Guingrich S, Micheels A, Chaudhry V, Corse A, Mosmiller B, Kelley A, Ho D, Srinivasan J, Vytopil M, Jara J, Ventura N, Scala S, Carter C, Donahue C, Herbert C, Weiner E, Alam S, McKinnon J, Haar L, McKinnon N, Alcon K, McKenna K, Sattar N, Daniels K, Jeffery D, Freimer M, Hoyle J, Kissel J, Agriesti J, Chelnick S, Mezache L, Pineda C, Muharrem F, Karam C, Khoury J, Marburger T, Kaur H, Dimitrova D, Gilchrist J, Agrawal B, Elsayed M, Kohlrus S, Andoin A, Darnell T, Golden L, Lokaitis B, Seelback J, Muppidi S, Goyal N, Sakamuri S, So Y, Paulose S, Pol S, Welsh L, Bhavaraju-Sanka R, Gonzales A, Dishman L, Jones F, Gonzalez A, Padilla P, Saklad A, Silva M, Kazamel M, Alsharabati M, Lu L, Nozaki K, Mumfrey-Thomas S, Woodall A, Mozaffar T, Cash T, Goyal N, Roy G, Mathew V, Maqsood F, Minton B, Jones H, Rosenfeld J, Garcia R, Echevarria L, Garcia S, Pulley M, Aranke S, Berger A, Shah J, Shabbir Y, Smith L, Varghese M, Gutmann L, Gutmann L, Jerath N, Nance C, Swenson A, Olalde H, Kressin N, Sieren J, Barohn R, Dimachkie M, Glenn M, McVey A, Pasnoor M, Statland J, Wang Y, Liu T, Emmons K, Jenci N, Locheke J, Fondaw A, Johns K, Rico G, Walsh M, Herbelin L, Hafer-Macko C, Kwan J, Zilliox L, Callison K, Young V, DiSanzo B, Naunton K, Benatar M, Bilsker M, Sharma K, Cooley A, Reyes E, Michon S, Sheldon D, Steele J, Howard J, Karam C, Chopra M, Traub R, Vu T, Katzin L, McClain T, Harvey B, Hart A, Huynh K, Beydoun S, Chilingaryan A, Doan V, Droker B, Gong H, Karimi S, Lin F, McClain T, Pokala K, Shah A, Tran A, Akhter S, Malekniazi A, Tandan R, Hehir M, Waheed W, Lucy S, Weiss M, Distad J, Strom S, Downing S, Kim B, Nowak R, Dicapua D, Keung B, Kumar A, Patwa H, Robeson K, Yang I, Nye J, Vu H. Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study. Journal Of The Neurological Sciences 2019, 407: 116419. PMID: 31698177, DOI: 10.1016/j.jns.2019.08.004.Peer-Reviewed Original ResearchConceptsSafety of eculizumabMyasthenia gravisCaucasian patientsAnti-acetylcholine receptor antibody-positive refractoryTerminal complement inhibitor eculizumabOpen-label extension studyMyasthenia Gravis ActivitiesQuantitative Myasthenia GravisGeneralized myasthenia gravisOpen-label extensionComplement inhibitor eculizumabLong-term efficacyMyasthenia Gravis QualityMyasthenia Gravis CompositeExtension baselineWeek 52Safety profileSubgroup analysisJapanese patientsDaily livingEculizumabGravisExtension studyPatientsREGAIN studyLong-term treatment with recombinant human pentraxin 2 protein in patients with idiopathic pulmonary fibrosis: an open-label extension study
Raghu G, van den Blink B, Hamblin MJ, Brown AW, Golden JA, Ho LA, Wijsenbeek MS, Vasakova M, Pesci A, Antin-Ozerkis DE, Meyer KC, Kreuter M, Moran D, Santin-Janin H, Aubin F, Mulder GJ, Gupta R, Richeldi L. Long-term treatment with recombinant human pentraxin 2 protein in patients with idiopathic pulmonary fibrosis: an open-label extension study. The Lancet Respiratory Medicine 2019, 7: 657-664. PMID: 31122893, DOI: 10.1016/s2213-2600(19)30172-9.Peer-Reviewed Original ResearchConceptsOpen-label extension studyIdiopathic pulmonary fibrosisPRM-151Adverse eventsLong-term treatmentWalking distanceExtension studyIPF exacerbationsWeek 52Week 76Pulmonary fibrosisIPF progressionDouble-blind treatment periodLife-threatening adverse eventsDouble-blind periodExploratory efficacy analysesSerious adverse eventsSevere adverse eventsMin intravenous infusionLong-term safetyRandom intercept mixed modelChest painPlacebo groupMore patientsEfficacy analysis
2018
Long-Term Safety and Efficacy of Elagolix Treatment in Women With Endometriosis-Associated Pain [11OP]
Surrey E, Taylor H, Duan W, Peloso P, Schwefel B, Chwalisz K. Long-Term Safety and Efficacy of Elagolix Treatment in Women With Endometriosis-Associated Pain [11OP]. Obstetrics And Gynecology 2018, 131: 4s. DOI: 10.1097/01.aog.0000533299.54528.7c.Peer-Reviewed Original ResearchNon-menstrual pelvic painEndometriosis-associated painBone mineral densityAdverse eventsPivotal studiesElagolix treatmentLumbar spine BMD Z-scoreExtension studyNon-peptide GnRH antagonistsSevere endometriosis-associated painSpine BMD Z-scoreEfficacy of elagolixCommon adverse eventsPhase 3 studyBMD Z-scoresNew safety concernsLong-term safetyDose-dependent decreaseDyspareunia scoresEndometrial thicknessPelvic painHot flushesGnRH antagonistDose groupMineral densityConfirmed Safety of Deutetrabenazine for Tardive Dyskinesia in a 2-Year Open-Label Extension Study (P4.075)
Fernandez H, Stamler D, Davis M, Factor S, Hauser R, Jimenez-Shahed J, Ondo W, Jarskog L, Woods S, LeDoux M, Shprecher D, Anderson K. Confirmed Safety of Deutetrabenazine for Tardive Dyskinesia in a 2-Year Open-Label Extension Study (P4.075). Neurology 2018, 90 DOI: 10.1212/wnl.90.15_supplement.p4.075.Peer-Reviewed Original ResearchMulticenter extension and switch study evaluating the safety of long-term treatment with abiraterone acetate fine particle formulation (AAFP) in patients with metastatic castration-resistant prostate cancer (mCRPC): The STAAR-E study.
Levin R, Given R, Nemeth P, Bosch B, Chapas-Reed J, Oh W. Multicenter extension and switch study evaluating the safety of long-term treatment with abiraterone acetate fine particle formulation (AAFP) in patients with metastatic castration-resistant prostate cancer (mCRPC): The STAAR-E study. Journal Of Clinical Oncology 2018, 36: 181-181. DOI: 10.1200/jco.2018.36.6_suppl.181.Peer-Reviewed Original ResearchMetastatic castration-resistant prostate cancerAdverse eventsSafety of long-term treatmentCastration-resistant prostate cancerExtension studyClinical laboratory test resultsLong-term treatmentLonger-term treatmentDays of treatmentPSA levelsAbiraterone acetateBilirubin increasePrednisone treatmentMedian ageProstate cancerProgressive diseaseSafety profileSerum testosteroneMethylprednisoloneTreatment durationLiver enzymesPrior treatmentPrednisonePatientsLaboratory test results
2017
61 Long-Term Safety and Efficacy of Elagolix Treatment in Women with Endometriosis-Associated Pain: Primary Results From Two Phase 3 Extension Studies
Surrey E, Taylor H, Giudice L, Singh S, Abrao M, Lessey B, Duan W, Peloso P, Schwefel B, Chwalisz K. 61 Long-Term Safety and Efficacy of Elagolix Treatment in Women with Endometriosis-Associated Pain: Primary Results From Two Phase 3 Extension Studies. Journal Of Minimally Invasive Gynecology 2017, 24: s44-s45. DOI: 10.1016/j.jmig.2017.08.078.Peer-Reviewed Original ResearchLong-term safety and efficacy of elagolix treatment in women with endometriosis-associated pain: primary results from two phase 3 extension studies
Surrey E, Taylor H, Giudice L, Singh S, Abrao M, Lessey B, Duan W, Peloso P, Schwefel B, Chwalisz K. Long-term safety and efficacy of elagolix treatment in women with endometriosis-associated pain: primary results from two phase 3 extension studies. Fertility And Sterility 2017, 108: e95-e96. DOI: 10.1016/j.fertnstert.2017.07.293.Peer-Reviewed Original Research
2016
Characterizing absolute lymphocyte count profiles in dimethyl fumarate–treated patients with MS
Fox R, Chan A, Gold R, Phillips J, Selmaj K, Chang I, Novas M, Rana J, Marantz J, Ambler Z, Ehler E, Havrdova E, Meluzinova E, Rektor I, Talab R, Gold R, Hartung H, Csanyi A, Csiba L, Jakab G, Hintzen R, Polman C, Drozdowski W, Fryze W, Kochanowicz J, Kwiecinski H, Maciejek Z, Schimrigk S, Selmaj K, Szczudlik A, Wajgt A, Belova A, Boiko A, Elchaninov A, Kozlov V, Odinak M, Shvarkov S, Skoromets A, Stolyarov I, Vorobieva O, Zavalishin I, Hillert J, Olsson T, Kappos L, Eraksoy M, Karabudak R, Siva A, Giovannoni G, Hawkins C, Sharief M, Sharrack B, Confavreux C, Ware J, Barnett M, Paine M, Lechner-Scott J, Beran R, Chapman C, Schwartz R, Butzkueven H, Casse R, Macdonell R, Berger T, Fazekas F, Ransmayr G, Vass K, Belachew S, De Deyn P, Decoo D, Dubois B, Medaer R, Seeldrayers P, Sindic C, Gaer L, Vanopdenbosch L, Grgic S, Bar-Or A, Bhan V, Bouchard J, Christie S, Grammond P, Jacques F, Veloso F, Vorobeychik G, Brinar V, Demarin V, Rudež J, Soldo-Butković S, Vurdelja R, Ambler Z, Doležil D, Havrdova E, Kanovsky P, Meluzinova E, Nova'k J, Rektor I, Skoda O, Duchcovská M, Camu W, Antoanela P, Coman I, Confavreux C, Edan G, Gout O, Lebrun-Frenay C, Papeix A, Vermersch P, Bachus-Banaschak K, Berghoff M, Bethke F, Boldt H, Chan A, Diener H, Eisensehr I, Emrich P, Griewing B, Haas J, Heesen C, Hoffmann F, Hufnagel A, Heidenreich F, Kleiter I, Lüer W, Marckmann-Böenke S, Marziniak M, Stangel M, Oschmann P, Rosenkranz T, Schneider H, Siefjediers V, Springub J, Stangel M, Stögbauer F, Bergh F, Tiel-Wilck K, Tinschert K, Grigoriadis N, Karageorgiou C, Kyritsis A, Papadimitriou A, Thomaides T, Vlaikidis N, Salguero Gonzalez L, Chon D, Ordoñez Sarg H, Agrawal C, Behari M, Cu V, Dwivedee S, Kothari S, Kulkarni R, Kushwaha S, Meena A, Mehndiratta M, Chandra Mukherjee S, Pandit L, Shah A, Shukla R, Vijayan K, Achiron A, Flechter S, Milo R, Vaknin-Dembinsky A, Pozzilli C, Daskalovska V, Castro Farfan F, Rangel Guerra R, Leyva I, Oropeza de Alba J, Revilla R, Rivera J, Gavriliuc M, Groppa S, Odainic O, Sanders E, Hupperts R, Mason D, Timmings P, Willoughby E, Czlonkowska A, Dorobek M, Drozdowski W, Fryze W, Hertmanowska H, Kleczkowska M, Kochanowicz J, Kwieciński H, Nowacki P, Podemski R, Potemkowski A, Selmaj K, Stelmasiak Z, Wajgt A, Zielinski T, Daskalovska V, Bajenaru A, Muresanu F, Nica S, Simu M, Drakulić S, Nadj C, Obradovic D, Vojinovic S, Turčáni P, Kahancová E, Kurca E, Lisý L, Krajnak V, Badenhorst F, Green J, Heckmann J, Isaacs M, Kappos L, Linnebank M, Müller S, Buchakchyys'ka N, Goloborodko A, Kobys T, Lebedynets V, Lytvynenko N, Nehrych T, Pasyura I, Ryabichenko T, Voloshina N, Duddy M, Hawkins C, Nicholas R, Palace J, Sharrack B, Silber E, Turner B, Camac A, Chumley W, Crayton H, Dixit S, Edwards K, English J, Freedman S, Gordon N, Gazda S, Goldstick L, Gottschalk P, Grazioli E, Grainger W, Green B, Grelinger B, Gudesblatt M, Gupta A, Hayat G, Hendin B, Henson L, Hentati A, Herrman C, Itkin A, Janus T, Kasper L, Lathi E, Mowry E, Parry G, Perel A, Rao T, Riskind P, Rizvi S, Rossman H, Scott T, Shafer S, Storey J, Thrower B, Rossen M, Tosches W, Trefts L, Turel A, Weinstock-Guttman B, Gold R, Kappos L, Arnold D, Bar-Or A, Giovannoni G, Selmaj K, Dawson K, Gallacher P, Milla C, Yang M, Antel J, Bakris G, Chung R, Kowey P, Polman C, Richert J, Seibert B, Ware J, Brandes D, Brassat D, Cohen B, Diem R, Goldman M, Herndon R, Miller A, Tumani H, Kneebone C, Fedulau A, Likhachev S, Mikhailova E, Naumova H, Decoo D, Sindic C, Gaer L, Grgic S, Sinanovic O, Suljic E, Deleva N, Georgiev D, Haralanov L, Ivanova S, Manchev I, Minchev D, Stamenova P, Tournev I, Vacheva E, Zahariev Z, Howell R, Vindas A, Bar-Or A, Blevins G, Kremenchutzky M, Veloso F, Witt N, Habek M, Rudež J, Soldo-Butković S, Vurdelja R, Doležil D, Havrdova E, Nova'k J, Vaclavik D, Antsov K, Gross-Paju K, Haldre S, Palu A, Toomsoo T, Al Khedr A, Camu W, De Seze J, Debouverie M, Defer G, Labauge P, Moreau T, Rumbach L, Angnstwurm K, Benes H, Berthele A, Boldt H, Christopher A, Derfuß T, Eisensehr I, Emrich P, Feneberg W, Hoffmann F, Hohlfeld R, Hüntemann R, Kallmann B, Kieseier B, Kleiter I, Landefeld H, Lüer W, Masri S, Mäurer M, Nelles G, Oschmann P, Paschen C, Reifschneider G, Sailer M, Schimrigk S, Spiegel-Meixensberger M, Steinbrecher A, Storch-Hagenlocher B, Tackenberg B, Tiel-Wilck K, Karageorgiou C, Papathanasopoulos P, Thomaides T, Vlaikidis N, Arjundas D, Behari M, Shah S, Ghosh A, Ghosh P, Ichaporia N, Khurana D, Kulkarni R, Mehndiratta M, Mehta N, Misra U, Mukherji J, Nellikunja S, Radhakrishnan S, Salem A, Sethi P, Singh G, Lines C, Singh M, Singh Y, Srinivasa R, Vijayan K, Hutchinson M, Sweeney B, Gilad R, Shahien R, Metra M, Paegle A, Daskalovska V, Delgado C, Escamilla J, Estañol B, Lopez N, Macias M, Punzo G, Quiñones S, Renteria M, Santos C, Gavriliuc M, Groppa S, Odainic O, Timmings P, Czlonkowska A, Dorobek M, Drozdowski W, Fryze W, Hertmanowska H, Kaminska A, Kapelusiak-Pielok M, Kleczkowska M, Kochanowicz J, Kwieciński H, Losy J, Nowacki P, Nyka W, Pierzchala K, Podemski R, Potemkowski A, Selmaj K, Stelmasiak Z, Szczudlik A, Tutaj A, Wajgt A, Zielinski T, Chinea A, Balasa R, Ionescu-Dimancea V, Mihancea P, Popescu C, Protosevici L, Drakulić S, Nadj C, Raicevic R, Vojinovic S, Kahancová E, Kurca E, Lisý L, Turčáni P, Arroyo R, Fernández O, Guijarro C, Izquierdo G, Montalbán X, Oreja-Guevara C, Prieto J, Lopez F, Buchakchyys'ka N, Chmyr G, Goloborodko A, Grytsay N, Kobys T, Kushnir G, Lebedynets V, Lytvynenko N, Moskovko S, Nehrych T, Palamar B, Pasyura I, Ryabichenko T, Voloshina N, Apperson M, Applebee A, Asher S, Ayala R, Ayres D, Azizi S, Baker M, Bauer B, Bomprezzi R, Buckler R, Carlini W, Cohan S, Crowell G, Edwards K, Eubank G, Felton W, Fodor P, Foley J, Ford C, Forester M, Fox E, Fox R, Freedman S, Garmany G, Gazda S, Giang D, Glaun B, Gold S, Gottesman M, Gudesblatt M, Herbert J, Herskowitz A, Honeycutt W, Huddlestone J, Hull R, Hunter S, Hutton G, Jacobs D, Janicki M, Khatri B, Kinkel R, Kita M, Krolczyk S, Krupp L, LaGanke C, Levin M, Licht J, Luzzio C, Lynch S, Mattson D, Mikol D, Miller T, Minagar A, Mitchell G, Moses H, Negroski D, Newman S, Pardo G, Patel M, Perel A, Phillips J, Picone M, Rammohan K, Rao T, Rinker J, Sadiq S, Schaeffer J, Sheremata W, Shin R, Shubin R, Silverman S, Smith R, Stein L, Stein M, Steiner D, Steingo B, Sullivan H, Sunter W, Vaishnav A, Vasquez A, Voci J, Warach J, Weinstock-Guttman B, Williams M, Wray S, Fox R, Phillips J, Hutchinson M, Havrdova E, Kita M, Wilson K, Yang M, Dawson K, Antel J, Ware J, Polman C, Kowey P, Chung R, Bakris G, Richert J, Seibert B, Brandes D, Brassat D, Cohen B, Diem R, Goldman M, Herndon R, Miller A, Tumani H, Alfaro-Vidal T, Crespo C, Foster J, Hunter K, Garcia-Gomez A, MacManus D, Miller D, Santana V, Tozer D, Kingshott-Wheeler C, Yousry T. Characterizing absolute lymphocyte count profiles in dimethyl fumarate–treated patients with MS. Neurology Clinical Practice 2016, 6: 220-229. PMID: 27347439, PMCID: PMC4909524, DOI: 10.1212/cpj.0000000000000238.Peer-Reviewed Original ResearchDelayed-release dimethyl fumarateAbsolute lymphocyte countMultiple sclerosisLymphocyte countDelayed-release dimethyl fumarate treatmentDMF discontinuationMean absolute lymphocyte countsEarly identification of patientsRelapsing-remitting multiple sclerosisUS Prescribing InformationTreatment of patientsIdentification of patientsComplete blood countDisease-modifying therapiesWhite blood cellsLymphocyte monitoringProlonged lymphopeniaBlood countClinical trialsPrescribing informationPatientsExtension studyBlood cellsDimethyl fumarateNeuroprotective effects
2012
Open-label extension study of the RNAi therapeutic ALN-VSP02 in cancer patients responding to therapy.
Alsina M, Tabernero J, Shapiro G, Burris H, Infante J, Weiss G, Cervantes-Ruiperez A, Gounder M, Paz-Ares L, Falzone R, Hill J, Cehelsky J, Vaishnaw A, Gollob J, LoRusso P. Open-label extension study of the RNAi therapeutic ALN-VSP02 in cancer patients responding to therapy. Journal Of Clinical Oncology 2012, 30: 3062-3062. DOI: 10.1200/jco.2012.30.15_suppl.3062.Peer-Reviewed Original ResearchStable diseasePartial responseExtension studyAdverse eventsEndometrial cancerCancer patientsCell carcinomaOpen-label extension studyNeck squamous cell carcinomaMore prior therapiesOngoing partial responseSafety/tolerabilityUnconfirmed partial responseEndometrial cancer patientsPhase II trialMonths of treatmentPhase I trialPhase 1 trialFavorable safety profileSquamous cell carcinomaTime of enrollmentPancreatic neuroendocrine tumorsVascular endothelial growth factorYears of treatmentRenal cell carcinoma
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