nursing involvement in an early phase study on the effects of a novel

Leela Goldstein1, Jake Cinco1, Adam Rumjon1, Iain C Macdougall1
Renal Unit, King’s College Hospital, London
BACKGROUND: Currently, the major treatments for the anaemia associated with chronic
kidney disease are EPO therapy, intravenous iron, and blood transfusions. Several new therapies
for treating anaemia are currently being investigated in clinical trials. One strategy that is being
explored is to antagonise the effect of a substance called hepcidin, which is the key regulator of
iron availability in the body. CKD patients, and particularly those on dialysis, have levels of
hepcidin around four times as high as normal healthy individuals, and this is now believed to be
part of the reason why some patients are resistant to EPO therapy.
Recently, our unit was privileged to conduct the first study in the world of a new anti-hepcidin
molecule called lexaptepid. Since this drug had never been given to any dialysis patients before,
it was critical to ascertain that it was safe and well-tolerated in this patient population.
STUDY PROTOCOL AND DESIGN: Under the supervision of the Principal Investigator,
the research nurses conducted a single-blind placebo-controlled crossover study in 8 patients
recruited from our main and satellite dialysis units.The patients attended a total of 8 visits: a
screening visit and 7 other study visits on days 1, 8, 10, 12, 15, 22 and 36. All patients were
administered a single IV injection of lexaptepid and a single IV injection ofa placebo one week
apart. Blood samples were taken at 0h, 3h, 6h, 9h, 12h, 18h and 24h on days 1 and 8. Patients
were required to stay overnight in our Clinical Trials Facility (CTF)staffed by our own team of
renal research nurses, and they were also dialysed in the CTF by the dialysis-trained members
of our research team.
RESULTS:9 participants were recruited: 3 males and 6 females with a mean age of 58 + 10
years (1 participant dropped-out due to an unrelated adverse event and was replaced).
8 participants completed the study.
The findings showed an increase in serum iron levels 3 hours post-dosing of lexaptepid that was
not seen with placebo; peak levels were seen at 12 hours post-dose (Figure).
There were no significant changes with the placebo treatment.
There were no treatment-related adverse events or other safety concerns in this study.
CONCLUSIONS:This is the first study ofan anti-hepcidin therapy in haemodialysis patients
who have EPO-resistant anaemia. The initial findings are promising and merit further study.
The main challenge from the research nursing point-of-view was covering the entire 24-hour
period following the two intravenous injections, and being able to dialyse the patients in the
Clinical Trials Facility. The research nurses continue to be involved in the next phase of
development with this novel and exciting drug.