What the trial was testing
The trial enrolled 15 patients with hemophilia. The study was sponsored by BioMarin Pharmaceutical 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
Six of seven participants reached normal clotting factor levels that lasted one year.
The New England journal of medicine · 2017 · NCT02576795
These findings — that participants who got the high dose achieved normal clotting factor levels for a year — were published in the The New England journal of medicine and represent the headline result of the study.
Researchers tracked outcomes across 15 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 only nine people. The gene therapy (valoctocogene roxaparvovec) later gained FDA approval in 2023 under the brand name Roctavian for adults with severe hemophilia A. Ask your doctor if this treatment is right for you.
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
SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors
Phase IV multi-center, US-centric, open-label, safety study enrolling participants with Hemophilia A or B with inhibitors, 12 years of age and older, who are either on long term prophylactic treatment (e.g., emicizumab) at risk of experiencing a breakthrough bleeding event (BE), or who are not on prophylactic treatment who may need to control a BE.
Manual Therapy in Hemophilic Arthropathy of the Ankle
Introduction: Haemophilic ankle arthropathy manifests as functional (deficit in muscle strength, mobility and proprioception), intra-articular degenerative alterations and chronic pain. Manual therapy techniques are characterised by treating the soft tissues with the aim of modifying their density, relieving pain, reducing tissue sensitivity and improving the ranges of mobility. The objective is to evaluate the safety and effectiveness of a manual therapy protocol in patients with haemophilic ankle arthropathy. Methods: Randomised crossover clinical trial. 13 patients with haemophilic ankle arthropathy from different regions of Spain will be recruited and randomised into two study groups (experimental and control). Each session of the experimental group will last 50 minutes, with 1 physiotherapy session per week for a period of 3 weeks. Patients will be evaluated at the beginning of the study, after the intervention and after a follow-up period of 4 weeks. The treatment programme includes 10 techniques that must be administered bilaterally. The study variables are the frequency of ankle haemarthrosis, range of movement, pressure pain threshold, pain intensity, joint status, biomechanical analysis of gait and balance, functionality and kinesiophobia. Expected results: To evaluate the safety of manual therapy in patients with haemophilia. To observe changes in pain, mobility, joint condition, stability and functionality of the ankle, and kinesiophobia.