Haemostasis & Thrombosis


The Haemostasis & Thrombosis Department at Children’s Health Ireland at Crumlin provides the national service for all children with bleeding disorders. The unit provides a comprehensive service to patients with a wide range of bleeding and clotting disorders on an inpatient and outpatient basis, from investigation and diagnosis, to long-term management of severe coagulation disorders. Led by Consultant Haematologist, Dr Beatrice Nolan, the team includes medical, nursing, pharmacy, physiotherapy, dentistry, social work and psychology services.

Research is a core activity for the unit. Dr Nolan and her core research team actively participate in both interventional and non-interventional (observational) clinical trials, working closely with a range of multinational pharmaceutical companies. Dr Nolan’s high recruitment rates and commitment to clinical research have placed Ireland at the forefront of haemophilia and haemostasis and thrombosis care globally. Dr Nolan works closely with the 4 centres of haemophilia in Ireland, and the Irish Haemophilia Society, in order to ensure that Irish patients receive the best available treatments.

Dr Nolan also provides anonymised data for academic research studies such as;

  • PedNet – The Paediatric Network for Haemophilia Management (An international network to record and publish data on paediatric haemophilia).
  • EUHASS – European Haemophilia Safety Surveillance (A pharmacovigilance program to monitor the safety of treatments for people with inherited bleeding disorders in Europe).
  • LoVIC-K – The Low Von Willebrand factor Ireland Cohort of Kids (A longitudinal study of Irish children with low Von Willebrand factor).
  • iPATH – An Irish Personalised Approach to the Treatment of Haemophilia (Examining patient perspectives to improve and enhance their care).

 

Current Trials & Studies

Study NameStudy TitleStudy TypeSponsorSponsor Type
LoVIC-KLow Von Willebrand Factor in Ireland Kids Cohort StudyNon-interventional StudyRSCIAcademic - Collaborative Group
iPATHAn Irish Personalised Approach to the Treatment of HaemophiliaNon-interventional StudyRCSI, ShireIndustry Supported
LTE-15174ATLAS-OLE: An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients with Hemophilia A or B, with or without Inhibitory Antibodies to Factor VIII or IXClinical Trial of IMPGenzyme CorporationIndustry supported
ALN-AT3SC-009A Phase 3 study to describe the efficacy and safety of Fitusiran prophylaxis in hemophilia A and B patients previously receiving factor or bypassing agent prophylaxis.Clinical Trial of IMPSanofi GenzymeIndustry supported
Elocta-003 ITI StudyA Non-Controlled, Open-Label, Multicenter, Study of Immune Tolerance Induction Performed with rFVIIIFc within a Timeframe of 60 Weeks in Severe Haemophilia A Patients with Inhibitors who have Failed Previous Immune Tolerance Induction TherapiesClinical Trial of IMPSobi (Swedish Orphan Biovitrum AB)Industry Sponsored
PedNETThe European Paediatric Network for Haemophilia Management and the PedNet Haemophilia RegistryNon-Interventional StudyEuropean Paediatric Network for HaemophiliaAcademic - Collaborative Group
Elocta-004A 5-year chart review study of patients with Haemophilia A with Inhibitors treated with rFVIIIFc (Elocta) for Immune Tolerance Induction.Non-interventional study (Phase IV)SobiIndustry Sponsored
B-MOREA 24-month prospective, non-interventional, international, multicentre study to describe the real-world effectiveness and usage of Alprolix in patients with haemophilia B (B-MORE).ObservationalSobiIndustry Sponsored

Past Trials & Studies

Study NameStudy TitleStudy TypeSponsorSponsor Type
9HB01EXT (BYOND)Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B (B-YOND)Clinical TrialBioverativIndustry Sponsored
Einstein Junior Phase III – 14372BAY 59-7939/14372: Multicenter, open-label, active-controlled, randomized study to evaluate the efficacy and safety of an age-and body weight-adjusted rivaroxaban regimen compared to standard of care in children with acute venous thromboembolismClinical TrialBayer HealthCare AGIndustry Sponsored
LeopoldA multi-center Phase III uncontrolled open-label trial to evaluate safety and efficacy of BAY 81-8973 in children with severe haemophilia A under prophylaxis therapyClinical TrialBayer HealthCare AGIndustry Sponsored
Affinity CSL627_3001A Phase III Open-label, Multicenter, Extension Study to Assess the Safety and Efficacy of Recombinant Coagulation Factor VIII (rVIII-SinglChain, CSL627) in Subjects with Severe Hemophilia AClinical TrialCSL BehringIndustry Sponsored
8HA02PED (Kids A-LONG)Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia AClinical TrialBiogenIndustry Sponsored
9HB02PED (Kids B-LONG)Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia BClinical TrialBiogenIndustry Sponsored
8HA01EXT (ASPIRE)Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia AClinical TrialBiogenIndustry Sponsored
CSLCT-BIO-12-83An Open-label, Multi-centre Post-marketing Study to Assess the Efficacy and Safety of Voncento® in Subjects with Von Willebrand DiseaseClinical TrialCSL BehringIndustry Sponsored
Einstein Junior Phase III – 12892Phase I: Single-dose pilot study of oral rivaroxaban in pediatric subjects with venous thromboembolismClinical TrialBayer HealthCare AGIndustry Sponsored
Einstein Junior Phase I – 17992Single-dose study of oral rivaroxaban dry powder suspension formulation in children treated for thrombosisClinical TrialBayer HealthCare AGIndustry Sponsored
997HA306 (PUPS A)An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Untreated Males With Severe Hemophilia AClinical TrialBioverativ A Sanofi CompanyIndustry Sponsored
998HB303 (PUPS B)An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Untreated Males With Severe Hemophilia AClinical TrialBioverativ A Sanofi CompanyIndustry Sponsored
GAPPGenotyping and Phenotyping of PlateletsNon-Interventional StudyUniversity of BirminghamAcademic - Collaborative Group