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Bioverativ scientists are committed to the discovery and development of new medicines. We conduct clinical trials to determine the safety and efficacy of investigational therapies and better understand how to use our approved products.

Pursuing the discovery of safe, effective therapies

Clinical Trials

A key step in advancing a program from research into an approved medicine is conducting clinical trials to test the safety and efficacy of an investigational therapy. We also run clinical trials to better understand how to use our approved products. 

Clinical trials follow strict, scientific standards that help protect patients and produce reliable trial results. This process spans three phases of clinical trials, each with a different purpose and involving an increasing number of people. 

Every clinical trial has a study plan that describes what will be done during the trial, how the trial will be conducted, and why each part of the trial is necessary. While the process may seem slow, it is critical to test whether the therapy is safe and effective.

We are grateful to the members of our community that participate in the clinical trials for testing our potential therapies, including patients, healthy volunteers, health care professionals, hospitals, and clinics. Each person’s participation contributes tremendously to our understanding of disease and has the potential to benefit patients around the world.

Beginning the process

Before a clinical trial on a potential therapy can start, Bioverativ submits an Investigational New Drug (IND) Application in the United States, or a Clinical Trial Application (CTA), as it’s called outside the U.S., to the local regulatory agency. We work with regulatory agencies around the world, including the Food and Drug Administration (FDA) in the U.S. and the European Medicines Agency (EMA) in Europe. The application describes the results of preclinical testing and clearly outlines the plan for conducting clinical trials.

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Phase I

Trials in Phase One assess safety and dosage, and are usually conducted with healthy volunteers. The goal is to learn how the potential therapy is absorbed, metabolized, and eliminated from the body, and to uncover its most frequent and serious side effects.

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Phase II

Trials in Phase Two gather preliminary data on how the potential therapy works for people with the target disease or condition. Some trials compare results for two different test groups: participants receiving the potential therapy and participants receiving a different treatment. Researchers continue to pay close attention to safety and possible side effects.

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Phase III

In this final phase, investigators gather more comprehensive information about a potential therapy’s safety and effectiveness. Trials might examine different patient populations, compare different dosage amounts of the potential therapy, or combine it with other treatments.

Once Phase Three is completed, the potential therapy can be filed with regulatory authorities like the FDA or EMA, which can then approve it as a therapy for public use.

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Post-market research

After a therapy has been approved, regulatory agencies often require additional studies. These post-marketing studies may evaluate the therapy on new patient populations, monitor the long-term impact or compare the therapy with other therapies.

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Current clinical trials:

Bioverativ is conducting clinical trials in hemophilia to evaluate the safety and efficacy of potential therapies, including: 

 

PUPs B-LONG: 

Study 998HB303 is an open-label, single-arm, multicenter study to evaluate the safety and efficacy of rFIXFc in Previously Untreated Patients (PUPs) when used according to the local standard of care (either episodic or prophylactic dosing) in PUPs with severe hemophilia B.

 

PUPs A-LONG: 

Study 997HA306 is an open-label, single-arm, multicenter study to evaluate the safety and efficacy of rFVIIIFc in Previously Untreated Patients (PUPs) when used according to local standard of care (either episodic or prophylactic dosing) in PUPs with severe hemophilia A. 

 

Learn more about all of our currently ongoing clinical trials at www.clinicaltrials.gov.

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