What the trial was testing
The trial enrolled 26 patients with hemophilia a. The study was sponsored by Novo Nordisk and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Annual bleeding rates dropped to 3-7 per year on concizumab.
Blood · 2019 · NCT03196284
These findings — that on daily concizumab in hemophilia A or B with inhibitors — were published in the Blood and represent the headline result of the study.
Researchers tracked outcomes across 26 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with hemophilia a, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Concizumab (Alhemo) is FDA-approved and available now for hemophilia A and B with inhibitors in patients 12 and older. It is given as a daily under-the-skin injection. Ask a hemophilia treatment center about access if existing prophylaxis is not working.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open hemophilia a trials
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of NXT007 prophylaxis compared with Factor VIII (FVIII) prophylaxis in participants with severe or moderate congenital hemophilia A without FVIII inhibitors. The study will include people aged ≥12 years old who have been on FVIII prophylaxis treatment prior to study entry.
Liver Biopsy In Haemophilia Gene Therapy
To perform a liver biopsy in haemophilia A and B patients with endogenous FVIII:C/FIX:C expression at \>1% any time after gene transfer following AAV mediated gene transfer. This is to obtain tissue for analysis, to understand if FIX/FVIII transgenic protein expression is mediated by AAV proviral DNA that is integrated into the host cell DNA or if stable expression in humans is mediated by episomal maintained AAV genome.