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. We have a multidisciplinary team in place; led by Consultant Haematologist Dr. Beatrice Nolan it includes medical, nursing, physiotherapy, dentistry, social work and psychology services.
Research is a core activity for the unit. We actively participate in clinical trials for new treatments in the area of coagulation disorders, helping to put Ireland at the forefront of haemophilia and haemostasis and thrombosis care globally.
We are also involved in several academic research studies and are the paediatric centre for the iPATH study, a significant new collaboration aiming to deliver new insights into haemophilia biology that can be directly translated to the delivery of enhanced clinical care for people with haemophilia in both Ireland and worldwide. iPATH, an Irish Personalised Approach to the Treatment of Haemophilia, is a strategic partnership involving basic scientists, RCSI, Trinity College Dublin (TCD) and Shire, Irish patients with haemophilia and clinicians in St James’s Hospital as well as Children’s Health Ireland at Crumlin. The partnership is jointly funded by RSCI, Science Foundation Ireland and Shire.
| Study Name | Study Title | Study Type | Sponsor | Sponsor Type |
|---|---|---|---|---|
| LoVIC-K | Low Von Willebrand Factor in Ireland Kids Cohort Study | Non-interventional Study | RSCI, HRB | Academic - Collaborative Group |
| iPATH | An Irish Personalised Approach to the Treatment of Haemophilia | Non-interventional Study | RCSI, Shire, SFI | Industry Supported |
| LTE-15174 | ATLAS-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 IX | Clinical Trial of IMP | Genzyme Corporation | Industry supported |
| ALN-AT3SC-009 | A 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 IMP | Sanofi Genzyme | Industry supported |
| Elocta-003 ITI Study | A 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 Therapies | Clinical Trial of IMP | Sobi (Swedish Orphan Biovitrum AB) | Industry 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 A | Clinical Trial | Bioverativ A Sanofi Company | Industry 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 A | Clinical Trial | Bioverativ A Sanofi Company | Industry Sponsored |
| GAPP | Genotyping and Phenotyping of Platelets | Non-Interventional Study | University of Birmingham | Academic - Collaborative Group |
| PedNET | The European Paediatric Network for Haemophilia Management and the PedNet Haemophilia Registry | Non-Interventional Study | European Paediatric Network for Haemophilia | Academic - Collaborative Group |
| Study Name | Study Title | Study Type | Sponsor | Sponsor 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 Trial | Bioverativ | Industry Sponsored |
| Einstein Junior Phase III – 14372 | BAY 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 thromboembolism | Clinical Trial | Bayer HealthCare AG | Industry Sponsored |
| Leopold | A 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 therapy | Clinical Trial | Bayer HealthCare AG | Industry Sponsored |
| Affinity CSL627_3001 | A 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 A | Clinical Trial | CSL Behring | Industry 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 A | Clinical Trial | Biogen | Industry Sponsored |
| 9HB02PED (Kids B-LONG) | Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B | Clinical Trial | Biogen | Industry Sponsored |
| 8HA01EXT (ASPIRE) | Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A | Clinical Trial | Biogen | Industry Sponsored |
| CSLCT-BIO-12-83 | An Open-label, Multi-centre Post-marketing Study to Assess the Efficacy and Safety of Voncento® in Subjects with Von Willebrand Disease | Clinical Trial | CSL Behring | Industry Sponsored |
| Einstein Junior Phase III – 12892 | Phase I: Single-dose pilot study of oral rivaroxaban in pediatric subjects with venous thromboembolism | Clinical Trial | Bayer HealthCare AG | Industry Sponsored |
| Einstein Junior Phase I – 17992 | Single-dose study of oral rivaroxaban dry powder suspension formulation in children treated for thrombosis | Clinical Trial | Bayer HealthCare AG | Industry Sponsored |
