Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinu
Research type
Research Study
Full title
Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose Eculizumab
IRAS ID
1003015
Contact name
Morag Griffin
Contact email
Eudract number
2019-003440-74
Research summary
Paroxysmal nocturnal haemoglobinuria (PNH) is a condition caused by an abnormality\nthat causes destruction of red blood cells and release into the urine.\nThis is a Phase 4, single-arm, open-label study to evaluate the efficacy and safety of\nravulizumab in adult participants with PNH who switch from eculizumab 1200 mg every\n2 weeks (above the recommended dose) to ravulizumab (at the recommended dose).\nALXN1210 is an experimental drug, however, it has been approved in the United States,\nEuropean Union, Canada, Brazil, Australia, and Japan for the treatment of PNH. The\npurpose of this study is to determine if ALXN1210 (every 8 weeks) is safe and effective for\nthe treatment of PNH in adult patients who are currently receiving 1200mg treatment of\nSoliris® (also known as eculizumab, which is a drug similar to ALXN1210).\nThe study will consist of a Screening Period of approximately 3 months and a Treatment\nPeriod of 351 days. All eligible participants will receive ravulizumab according to\napproved dose regimen during the Treatment Period. Between 2 and 5 study sites will be\nopened in the United Kingdom and possibly in other countries to enrol 20 participants\nwith PNH.
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
20/YH/0105
Date of REC Opinion
14 May 2020
REC opinion
Further Information Favourable Opinion