CAPSTONE (ongoing) and Planned ASSET (future) Clinical Trials
In March 2018, we opened our first site for CAPSTONE, a three-part, two-arm, randomized, open label, Phase III clinical trial of nomacopan in transfusion dependent PNH patients in specific countries in Europe and other countries, where Soliris® is not the standard of care.
Upon completion of the trial, patients have the option of remaining on nomacopan and being entered into our long-term safety and efficacy CONSERVE study.
The objective of this trial is to demonstrate efficacy of nomacopan plus standard of care to standard of care in patients with uncontrolled haemolysis due to PNH and to assess safety and tolerability. The primary endpoint of the study is haemoglobin stabilization rate and the avoidance of PRBC transfusions during the treatment period.
This Phase III clinical trial is currently recruiting.
We plan in the future to conduct a small proof of principle study followed by ASSET, a Phase III clinical trial in PNH patients who have been treated with Soliris in the United States, Europe and other countries where Soliris is the current standard of care.
Phase II COBALT PNH Trial (completed)
In the fourth quarter of 2016, we commenced enrollment for a 90-day open-label Phase II, single-arm clinical trial in patients with PNH in five centers in the European Union, known as the COBALT trial. The trial was concluded in December 2017.
We enrolled and treated eight patients with nomacopan self-administered subcutaneous injections twice a day for approximately the first month and then switched to once daily injections. Of those eight patients, seven completed the 90-day trial while one patient with a suspected co-morbidity unrelated to treatment was withdrawn on day 43 of the trial.
The primary endpoint of the trial was defined as a reduction in LDH to ≤1.8 times the ULN, at day 28.
Results from the COBALT trial showed that patients were comfortable with self-administration of nomacopan. Those results showed that there were four serious adverse events, SAEs, and no SAEs related to nomacopan. The most commonly reported adverse events were mild self-limiting injection site reactions.
All patients that completed the trial saw declines in lactate dehydrogenase, LDH, levels (although in some cases there were intermittent rises). The trial met its primary endpoint defined as an LDH of ≤1.8 X ULN at Day 28.
Of the seven patients who completed the study, six were transfusion-dependent prior to the trial. Of those six patients, three have not required transfusions while on nomacopan during the trial.