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.
| Study Name | Study Title | Study Type | Sponsor | Sponsor Type |
|---|---|---|---|---|
| PK OLE | On open label, multicentre, extension study of AG-348 in adult subjects with pyruvate kinase deficiency previously enrolled in AG-348 studies | Clinical trial (NCT03853798) (2018-003459-39) | Agios | Commercial: Industry |
| PKD Registry | Pyruvate Kinase Deficiency Global Longitudinal Registry | Observational-non invasive | Agios | Commercial: Industry |
| Frontier OLE | Open-label, long-term safety and efficacy study of Mim8 in participants with haemophilia A with or without inhibitors | Observational-non invasive | Novo Nordisk | Commercial : Industry |
| Study Name | Study Title | Study Type | Sponsor | Sponsor Type |
|---|---|---|---|---|
| Pyruvate Kinase Deficiency | An open label study to evaluate the efficacy and safety of AG-348 in regularly transfused adult subjects with pyruvate kinase (PK) deficiency | Clinical trial (NCT03559699) (2017-003803-22) | Agios | Commercial: Industry |