What the trial was testing
The trial enrolled 10 patients with hemophilia. The study was sponsored by CSL Behring and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was an early-stage trial — researchers are still confirming safety and getting an early look at how well the treatment works. 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
Spontaneous bleeding dropped by up to 70% after a single gene therapy treatment.
Blood · 2018 · NCT02396342
These findings — that patients in the higher-dose group had 70% fewer spontaneous bleeding episodes after treatment. — were published in the Blood and represent the headline result of the study.
Researchers tracked outcomes across 10 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, 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
This was an early-stage study with 10 patients and the treatment is not yet FDA-approved. The results were promising, but gene therapy for hemophilia B is still being studied. If you have hemophilia B, ask your doctor about open clinical trials or other approved treatment options.
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 trials
ITI Using SCT800 Alone or Combining Daratumumab in Hemophilia A Adolescents and Adults With High Titer Inhibitor
To evaluate the time of response, sustained remission rate, and relapse rate of CD38 monoclonal antibody (Daratumumab) combined with SCT800 (rFVIII) in the treatment of hemophilia A adolescents and adults with high titer inhibitors.
Study of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) On-Demand Treatment
The indication for this product is to control bleeding in patients with hemophilia A (congenital deficiency of factor VIII). The primary objective: Evaluation of the efficacy of recombinant human coagulation factor VIII-Fc fusion protein for injection (FRSW107) as an on-demand treatment in previously treated patients with severe hemophilia A. Secondary objectives: Evaluation of the safety and immunogenicity of FRSW107 as an on-demand therapy in previously treated patients with severe hemophilia A. Evaluate the on-demand treatment's PK profile of FRSW107 in previously treated patients with severe hemophilia A based on population pharmacokinetic (PopPK) methods ; preliminarily investigate the exposure-response (E-R) relationship of FRSW107 on-demand treatment in these patients if data permit.