Iptacopan is a complement inhibitor that it is taken orally, twice daily. It works by inhibiting Complement Factor B (CFB ). Without CFB , C5 cannot be activated to create the membrane attack complex which becomes so damaging to those susceptible to aHUS.
Iptacopan has been developed by Novartis, another Swiss pharmaceutical company. It has the approval of the FDA to be tested on patients who onset with aHUS but who have not received any other complement inhibitor drug.
The three trial sites listed so far are in Vienna,Austria, Thessaloniki ,Greece and Iruma-Gun, Japan. All sites are currently recruiting and 50 participants are being sought. How those numbers are achieved from only three sites is unclear.
The usual primary outcome measures apply, I.e. number of TMAs stopped in 26 weeks as measured by platelets and LDH levels during treatment and compared with baseline prior to treatment. Similarly improvement in kidney function EFGR. But additionally long term ( one year) safety and efficiency outcomes including adverse and serious adverse events.
Secondary outcomes include time it takes to resolve TMA within 26 weeks , increase in haemoglobin during the 26 weeks , and platelet , LDH and haemoglobin levels at 26 weeks,. Also whether any dialysis is stopped and whether there is any change to kidney disease stages.
Unusually there are four patient reported outcomes measures . In addition to Fatigue there is patient feedback on health state, disease severity and quality of life impact.
What patients think about the drug administration does not seem to be covered.
The usual sort of exclusion criteria apply for an aHUS complement inhibitor trial I.e. Other primary TMA and secondary TMA patients as well as those with a DGKE mutation. Also those on long term dialysis.
As mentioned recruitment has begun, it is for adults only and its estimated completion dates are end of 2024 /early 2025.
By the mid to late 2020s we may well see more options for aHUS patients around the world.
Full information about the trial can be seen NCT04889430
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