The primary endpoint is the proportion of patients who experience serum free C5-associated BTH (defined as ≥1 new or worsening symptom or sign of IVH with lactate dehydrogenase levels ≥2 x upper limit of normal and free C5 concentrations ≥0.5 μg/mL). Study 401 (NCT04320602) is an ongoing, phase 4, single-arm, multicentre trial designed to investigate the impact of switching treatment from fixed, high-dose IV eculizumab (1200 mg q2w) to weight-based IV ravulizumab q8w in adult (≥18 years of age N = 18) patients with PNH.
However, currently there are no recommendations or clinical trial data regarding the impact of switching these patients to ravulizumab. In such patients, increasing the eculizumab dose can be effective. Approximately 10%–15% of patients with PNH who receive the approved, fixed eculizumab dose (900 mg q2w) experience insufficient inhibition of intravascular haemolysis (IVH), characterised by breakthrough haemolysis (BTH) towards the end of the dosing interval. Ravulizumab provides the same clinical benefit as eculizumab in patients with PNH at a longer dosing interval (every 8 weeks vs every 2 weeks ). The complement C5 inhibitors eculizumab and ravulizumab are the current standards of care in patients with paroxysmal nocturnal haemoglobinuria (PNH), a rare chronic blood disorder. BSH23-OR01 Control of hemolysis in paroxysmal nocturnal hemoglobinuria following switching from High-Dose Eculizumab to Ravulizumab Dr Morag Griffin 1, Dr Shreyans Gandhi 2, Ms Eden Hicks 3, Dr Deepak Jain 3, Dr Richard Kelly 1, Dr Talha Munir 1, Dr Petra Muus 1, Dr Masayo Ogawa 3, Dr Roochi Trikha 2, Mr Ji Yu 3, Dr Austin Kulasekararaj 2 1St James's University Hospital, Leeds, UK, 2King's College Hospital, London, UK, 3Alexion, AstraZeneca Rare Disease, Boston, USA
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