2023
Topical Isotretinoin (TMB-001) Treatment for 12 Weeks Did Not Result in Clinically Relevant Laboratory Abnormalities in Participants with Congenital Ichthyosis in the Phase 2b CONTROL Study
Marathe K, Teng J, Guenthner S, Bunick C, Kempers S, Eads K, Castelo-Soccio L, Mendelsohn A, Raiz J, Murrell D. Topical Isotretinoin (TMB-001) Treatment for 12 Weeks Did Not Result in Clinically Relevant Laboratory Abnormalities in Participants with Congenital Ichthyosis in the Phase 2b CONTROL Study. Dermatology And Therapy 2023, 13: 1255-1264. PMID: 37170057, PMCID: PMC10264299, DOI: 10.1007/s13555-023-00923-1.Peer-Reviewed Original ResearchSignificant laboratory changesLaboratory changesCongenital ichthyosisLaboratory parameter changesRelevant laboratory abnormalitiesPhase 2b studyWeeks of treatmentYears of ageSingle reportCI subtypesIsotretinoin treatmentStudy discontinuationSystemic isotretinoinUrinalysis abnormalitiesLaboratory abnormalitiesOral retinoidsTreatment cohortsVehicle treatmentDrug withdrawalLaboratory valuesLeukocyte esteraseUrea nitrogenControl studyWeeksNeutropenia
2022
The CONTROL study: A randomized, double-blind vehicle-controlled phase 2b study of novel topical isotretinoin formulation demonstrates improvement in recessive X-linked and autosomal recessive lamellar congenital ichthyosis
Teng JMC, Bunick CG, Guenthner S, Murrell DF, Marathe K, Kempers S, Eads K, Mendelsohn AM, Raiz J, Tavakkol A, Castelo-Soccio L. The CONTROL study: A randomized, double-blind vehicle-controlled phase 2b study of novel topical isotretinoin formulation demonstrates improvement in recessive X-linked and autosomal recessive lamellar congenital ichthyosis. Journal Of The American Academy Of Dermatology 2022, 87: 1455-1458. PMID: 35872261, DOI: 10.1016/j.jaad.2022.07.028.Peer-Reviewed Original Research
2020
First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies
Pipe S, Recht M, Key N, Leebeek F, Castaman G, Lattimore S, Van Der Valk P, Peerlinck K, Coppens M, O'Connell N, Pasi J, Kampmann P, Meijer K, von Drygalski A, Young G, Hermans C, Astermark J, Klamroth R, Lemons R, Visweshwar N, Crary S, Kazmi R, Symington E, Escobar M, Gomez E, Kruse-Jarres R, Kotowski A, Quon D, Wang M, Wheeler A, Sawyer E, Verweij S, Colletta V, Bajma N, Gut R, Miesbach W. First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies. Blood 2020, 136: lba-6. DOI: 10.1182/blood-2020-143560.Peer-Reviewed Original ResearchAdeno-associated virus serotype 5Entity's Board of DirectorsEtranacogene dezaparvovecInvestigational gene therapyCSL BehringHemophilia BAdverse eventsPersonal feesGene therapyFrequent treatment-related adverse eventsClinical studiesPre-existing neutralizing antibodiesTreatment-related adverse eventsLead-InGene therapy clinical studiesPre-existing NAbsAnnualized bleeding rateGene therapy trialsInfusion-related reactionsPhase 2b studyPhase 3 studyLead-in periodUse of corticosteroidsInfluenza-like illnessLiver-specific promoter
2019
Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B
Von Drygalski A, Giermasz A, Castaman G, Key N, Lattimore S, Leebeek F, Miesbach W, Recht M, Long A, Gut R, Sawyer E, Pipe S. Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B. Blood Advances 2019, 3: 3241-3247. PMID: 31698454, PMCID: PMC6855101, DOI: 10.1182/bloodadvances.2019000811.Peer-Reviewed Original ResearchEtranacogene dezaparvovecFactor IX replacementFactor IX activityAMT-061Factor IXBleeding cessationHemophilia BFactor IX replacement therapyPhase 2b studySecondary end pointsLevels of liver enzymesCessation of bleedingClinically relevant increasePhase 3 trialLiver-specific promoterSevere hemophilia BSevere hemophilia B.AAV5 vectorReplacement therapyInflammatory markersSingle doseClinically significant elevationsBleeding frequencyNeutralizing antibodiesBefore treatment
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