CWoW - ABP 959 in PNH
Research type
Research Study
Full title
A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
IRAS ID
235842
Contact name
Austin Kulasekararaj
Contact email
Sponsor organisation
Amgen Inc.
Eudract number
2017-001418-27
Duration of Study in the UK
2 years, 0 months, 17 days
Research summary
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, life-threatening bone marrow disorder that is associated with a significant increase in mortality, development of arterial and venous thrombosis (blood clots), organ damage, and rapid deterioration in quality of life. The clinical symptoms of PNH include abdominal pain, chest pain, dyspnoea (breathlessness), and haemoglobinuria (excretion of haemoglobin into urine); and life-threatening complications include thromboembolism (obstruction of a blood vessel by a blood clot), pulmonary hypertension (high blood pressure in blood vessels of the lungs), and impaired renal (kidney) function.
Eculizumab, the active ingredient in Soliris® (Alexion Pharmaceuticals), is a monoclonal antibody that is licensed for use in patients with PNH. ABP 959 is being developed as a biosimilar to Soliris® (eculizumab), for the treatment of PNH and other indications. The active ingredient of ABP 959 is an anti-C5 monoclonal antibody with the same amino acid sequence as eculizumab, and has the same pharmaceutical form, dosage strength, route of administration, and dosing regimen as eculizumab.
The current study is designed to demonstrate that there is no clinically meaningful difference between ABP 959 and eculizumab in terms of efficacy, pharmacodynamics (effects of the medication on the body), pharmacokinetics (how the body processes the medication), safety, and immunogenicity (ability to induce an immune response) in adult participants with PNH.
This is a randomised, double-blind, active-controlled, phase 3 study in adult participants with PNH. Approximately 40 participants will be randomly assigned to receive either ABP every 14 days for 52 weeks followed by eculizumab every 14 days for 26 weeks, or vice versa. The total duration of participation is approximately 24 months (6 months for enrolment and 18 months for treatment).
REC name
North West - Haydock Research Ethics Committee
REC reference
18/NW/0780
Date of REC Opinion
21 Nov 2018
REC opinion
Favourable Opinion