Haematology

St James’s hospital has the largest Haematology department in Ireland, and Dr Emma Tuohy runs the only national comprehensive care service for patients with rare red cell disorders and in interested in setting up clinical trials in this patient cohort. The CRF manages both investigator led studies and clinical trials.

 

Current Trials & Studies
Study NameStudy TitleStudy TypeSponsorSponsor Type
Atlas-OleAn Open Label, Long Term Saftey and Efficacy Study of Fitusiran in pts with Hemophlia A or B with or without Inhibitory antibodies to factor VIII or IX Clinical trial (NCT03754790) (2018-002880-25) SANOFI GENZYME Commercial: Industry
Pyruvate Kinase DeficiencyAn open label study to evaluate the efficacy and safety of AG-348 in regularly transfused adult subjects with pyruvate kinase (PK) deficiencyClinical trial (NCT03559699) (2017-003803-22)AgiosCommercial: Industry
PK OLEOn open label, multicentre, extension study of AG-348 in adult subjects with pyruvate kinase deficiency previously enrolled in AG-348 studiesClinical trial (NCT03853798) (2018-003459-39) AgiosCommercial: Industry
iPATH (2) An Investigation of Physical Activity, Cardiometabolic Health and Fitness in Healthy Men: Collecton of Normative Data for the Irish Personalised Approach to the Treatment of Haemophilia (iPATH) Study Observational Non-invasive Science Foundation Ireland Non-commercial: Academic
PKD RegistryPyruvate Kinase Deficiency Global Longitudinal RegistryObservational-non invasiveAgiosCommercial: Industry
ACTIVATEExtension study of AG 348 in adult participants with Pyruvate Kinase deficiency previously enrolled in AG348 - 006 or AG348-C-007Clinical trialAgios
Novo Nordisk A multinational, open-label, randomised, controlled trial to investigate efficacy and safety of NNC0365-3769 (Mim8) in adults and adolescents with haemophilia A with or without inhibitors. Clinical trial Novo Nordisk Commercial: Industry
Past Trials & Studies
Study NameStudy TitleStudy TypeSponsorSponsor Type
ACTIVATEA phase 3 open label study to evaluate the efficacy and safety of AG-348 in regularly transfused patients with pyruvate kinase deficiency. Clinical trialAgiosCommercial: Industry