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
Longitudinal Cohort of Thrombosis and Hemostasis Diseases
This is a multicenter, prospective, longitudinal, observational cohort study to investigate thrombosis and hemostasis diseases in Chinese patients. This study will collect basic information, diagnostic and treatment information, as well as medical expense information of patients from medical records.The incidence and risk factors of thrombosis and hemostasis diseases, the treatment methods, prognosis and medical expenses of these patients in China will be analyzed. The study will use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A.