Exploring potential ways to evolve hemophilia treatment

Learn how Pfizer is researching potential alternate approaches to hemophilia treatment.

As part of our longstanding commitment to the hemophilia community, Pfizer continues to develop potential treatments that we hope will help people living with this bleeding disorder. Our current research is focused on gene therapy as well as a study medicine that aims to reduce the burden associated with traditional factor replacement products.

We’re studying investigational gene therapies that are designed to help people with hemophilia A or B produce clotting factor. (Learn about gene therapy below.) In partnership with everyone who enrolls in our clinical trials, we are evaluating whether this helps prevent bleeding episodes or reduce how often replacement factor infusions are needed.

We’re also committed to exploring a once-weekly shot under the skin for people with severe hemophilia A or B. This study medicine is a non-factor hemophilia treatment option that works for people without or with inhibitors. It is designed to reduce how often the shots are required, compared with existing factor replacement.

If you or a loved one are considering taking part in a hemophilia clinical trial, find more information below.

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How we are exploring the potential of gene therapy

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Hemophilia B
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
NCT03861273
Recruiting
Males
18-65years
92study location(s)
Hemophilia A, Hemophilia B
A Study to Learn About the Safety and Effectiveness of the Study Medicines Called Giroctocogene Fitelparvovec and Fidanacogene Elaparvovec
NCT05568719
Recruiting
Males
18+ years
2study location(s)
Hemophilia B, Hemophilia A
A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
NCT03587116
Recruiting
Males
18-64years
108study location(s)
Hemophilia A
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
NCT04370054
Recruiting
Males
18-64years
74study location(s)
Hemophilia A, Hemophilia B
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
NCT03938792
Recruiting
Males
12-74years
104study location(s)
Hemophilia A, Hemophilia B
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
NCT05145127
Recruiting
Males
12-74years
74study location(s)
Hemophilia A, Hemophilia B
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
NCT05611801
Recruiting
Males
1-17years
17study location(s)
Hemophilia B
Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B
NCT03307980
Recruiting
Males
18-65years
28study location(s)

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For more information about participation in a Pfizer clinical trial, please email or call the Pfizer Clinical Trial Contact Center at 1-800-887-7002.

About hemophilia

Hemophilia is a disorder in which the blood does not clot properly. This can lead to bleeding after injuries or surgery. It can also lead to spontaneous bleeding, meaning that the bleeding occurs without a clearly identified cause.

A person’s blood typically has clotting factors that help to stop bleeding. People with hemophilia have low levels of either factor VIII (8) or factor IX (9) which results in reduced blood clotting and causes the bleeding described above.

The current standard treatment for hemophilia is to replace the clotting factor through regular IV infusions. For some people, the frequency and IV administration of this therapy can make it a burden.

About 10–15% of people with hemophilia have inhibitors that prevent the standard clotting factor replacement from working effectively. People with inhibitors require special treatment when experiencing bleeding, which can be less effective and more expensive than standard treatment.

About gene therapy

Gene therapy targets the underlying cause of a genetic disease. In gene therapy, a functioning gene is delivered to the cells of a targeted tissue in the body (for example the cells of the liver), enabling the production of a protein that was either missing or not functioning—in the case of hemophilia, factor VIII (8) or factor IX (9). Gene therapy has the potential to be used instead of traditional treatments.

The safety and efficacy of gene therapy is being studied in clinical trials.

We believe that gene therapy is a potentially transformative treatment approach that, if successful, may help reduce the burden of disease and of ongoing treatments for the people who are affected by rare diseases.

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