An Open-Label, phase 3 extension study of REGN3918 in adults with PNH
Research type
Research Study
Full title
AN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF REGN3918 IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
IRAS ID
270370
Contact name
Peter Hillmen
Contact email
Sponsor organisation
Regeneron Pharmaceuticals, Inc.
Eudract number
2019-000130-20
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 6 months, 1 days
Research summary
This study is a phase 3, open-label extension study of REGN3918 (study drug) for adults with Paroxysmal Nocturnal Haemoglobinuria (PNH). PNH is a rare and life threatening blood condition characterised by the destruction of red blood cells (RBC) by the immune system. It is estimated that approximately 1.3 million people are diagnosed each year.\n\nCurrently, eculizumab (marketed under the brand name Soliris®) is the approved treatment of PNH in many countries worldwide, including the UK. \n\nThe main purpose of this study is to evaluate long-term tolerability, safety and effect of REGN3918 in participants who have already completed one of the parent studies.\n\nThe study will include approx. 147 participants in multiple sites worldwide\n\nParticipation will be at least 2 years with a 21-week follow-up period. Participants will be allowed to self-administer the subcutaneous injection at home. \n\nREGN3918 will be administered by subcutaneous (SC, injection into fat underneath the skin) doses on a weekly basis. Other study procedures include physical examinations, blood and urine samples, vital signs and ECG.
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
20/YH/0040
Date of REC Opinion
23 Apr 2020
REC opinion
Further Information Favourable Opinion