CLNP023F12302: aHUS Switch Study
Research type
Research Study
Full title
A multicenter, single-arm, open-label study to evaluate efficacy and safety of switching from anti-C5 antibody treatment to iptacopan treatment in study participants with aHUS
IRAS ID
1005877
Contact name
Nathalie Lauper Von Raumer
Contact email
Sponsor organisation
Novartis Pharma Services AG
Clinicaltrials.gov Identifier
Research summary
The purpose of this study is to determine if it is safe to switch participants with atypical haemolytic uremic syndrome (aHUS) from an intravenous/subcutaneous treatment with anti-C5 antibody (eculizumab or ravulizumab) to oral iptacopan treatment.
aHUS is a very rare progressive, and life-threatening disease characterised by destruction of red blood cells, a decrease in the number of platelets, blood clotting problems thereby damaging the cells that line blood vessels of the kidney causing inflammation. The constant inflammation causes continued damage to the kidneys which then leads to the inability for the kidneys to filter waste from your blood. aHUS is caused by uncontrolled activation of an important system in the body called the alternative complement pathway (AP). The “AP” usually provides protection against infections.
Iptacopan is a new oral medication being developed in aHUS, which blocks the activity of a protein called factor B which is part of the “AP”. By preventing activation of AP, it is expected to reduce the signs, symptoms, and consequences of the disease in patients with aHUS.
The main question the trial is designed to answer is: do participants with aHUS previously treated with anti-C5 antibody and who no longer have evidence of the disease, continue to remain stable after switching to iptacopan 200 mg twice a day?
The trial will be conducted in around 15 countries across the world. Approximately 50 participants with aHUS, previously treated with eculizumab or ravulizumab for at least 3 months and who no longer have evidence of active aHUS disease, will be enrolled. 35-40 participants will have modifications (mutations) in aHUS specific genes or will have tested positive for an aHUS-specific disease biomarker, namely anti-FH antibody.
All participants in the trial will receive iptacopan for about 2 years (24 months). There will be 2-3 sites in the UK.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
23/EM/0273
Date of REC Opinion
11 Jan 2024
REC opinion
Further Information Favourable Opinion