APL3007-PNH-102
Research type
Research Study
Full title
Safety and Tolerability of APL-3007 Administered as a Single Dose in Addition to Background Therapy with a C5 Inhibitor in Adults with Paroxysmal Nocturnal Hemoglobinuria
IRAS ID
1011778
Contact name
Georgia Christoforou
Contact email
Sponsor organisation
Apellis Pharmaceuticals, Inc
Research summary
The primary purpose of the study is to find out if the drug APL-3007 is safe in treating adult participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have been treated with a stable dose of ravulizumab-cwvz for at least the previous 6 months and remain anemic (with a Hb <10.5 g/dL). The drug is given via subcutaneous injection (beneath the skin) and will be given as 2 separate injections of 2mL and 1.5mL. Approximately 6 adults from 1 study center across the UK will take part in the study and it will last for up to 7 months and involve about 12 visits.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
25/NE/0060
Date of REC Opinion
7 May 2025
REC opinion
Further Information Favourable Opinion