Haemophilia Studies
The Haemophilia Department at St James’s hospital, has a long established research program over the last 7 years. The CRF in conjunction with the National Coagulation Centre runs a number of both investigator-led and industry centred drug trials in Haemophilia. In fact this area of medicine has seen dramatic changes over the last number of years with the introduction of gene therapies within a research setting. We are the only centre in Ireland to have conducted gene therapy studies in haemophilia.
| Study Name | Study Title | Study Type | Sponsor | Sponsor Type |
|---|---|---|---|---|
| Freedom NCC | A phase 3b open-label, multicenter study evaluating physical activity and joint health in previously treated patients ≥12 years of age with severe haemophilia A treated with intravenous recombinant coagulation factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; efanesoctocog alfa) for 24 months | Clinical Trial (NCT05817812) | SOBI | Commercial: Industry |
| AAV5-Padua | Phase III, open-label, single dose, multi-center multinational trials investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene (AAV5-hFIXco-Padua,AMT-061) administered to adult subjects with severe or moderately severe haemophilia B | Clinical Trial (NCT03569891) (2017-004305-40) | UniQure | Commercial: Industry |
| Atlas-Ole | An Open Label, Long Term Safety 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 |
| Frontier 2 | 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 (NCT05053139) (2020-001048-24) | Novo Nordisk | Commercial: Industry |
| Study Name | Study Title | Study Type | Sponsor | Sponsor Type |
|---|---|---|---|---|
| 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 |
| iPATH | The Irish Personalized Approach to the treatment of Haemophilia (iPATH): An investigation of the relationship between physical health, physical activity and bleeding phenotype in adults with haemophilia | Observational-non invasive | National (Government agency) | Non-commercial: Academic |
| BH30071- Haemophilia A | A randomized, multicenter, open-label, phase III clinical trial to evaluate the efficacy, safety, and pharmacokinetics of prophylactic emicizumab versus no prophylaxis in hemophilia a patients without inhibitors | Clinical Trial (NCT02847637) | Roche | Commercial: Industry |
| FIX-GT | A Phase I/II, open label, multicentre, ascending, single dose, safety study of a novel adeno-associated viral vector (FLT180a) in patients with Haemophilia B | Clinical Trial (NCT03369444) | Commercial: Industry | |
| Hope-B | Phase III trial of AMT-061 in subjects with severe or moderately severe hemophilia B. | Clinical Trial | ||
| PERSONAL | PERSONALising Factor VIII prophylaxis regimens: Efficacy of standard versus pharmacokinetically based regimens in adult patients with severe Haemophilia A (PERSONAL trial) | Clinical Trial (NCT02314325) | Commercial: Industry | |
| Dabigatran Reversal | A phase 3 case series clinical study of the reversal of the anticoagulant effects of dabigatran by intravenous administration of 5.0g idarucizumab BI 655075 in patients treated with dabigatran etexilate who have uncontrolled bleeding or require emergency surgery or procedures | Clinical Trial | Commercial: Industry | |
| BAX326 Continuation | BAX326 (recombinant Factor IX): Evaluation of Safety, Immunogenicity, and Hemostatic Efficacy in Previously Treated Patients with Sever (FIX level < 1 %) or moderately severe (FIX level 1-2%) Hemophilia B- A Continuation Study | Clinical Trial (NCT01286779) | Commercial: Industry |