A study on the long-term safety and effectiveness of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B

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What is this study about?

This study focuses on individuals living with Hemophilia A or Hemophilia B. These are rare bleeding disorders where the blood does not clot properly due to a lack of specific proteins needed for clotting. The purpose of this study is to describe the long-term safety and effectiveness of a single-dose treatment previously received by participants. The medications being observed are fidanacogene elaparvovec, used for those with Hemophilia A, and giroctocogene fitelparvovec, used for those with Hemophilia B.

The treatment involves gene therapy, which is a method used to introduce genetic material into cells to help the body produce the missing clotting proteins. Participants in this study will be monitored over a long period of time to observe how the body responds to these treatments. This includes tracking the level of clotting factor, which is the protein responsible for stopping bleeding, and checking for any potential issues such as thromboembolic events, which are blood clots that can travel through the bloodstream, or changes in liver health.

Who Can Join the Study?

  • You must be a person who has already received one of two specific gene therapies, called giroctocogene fitelparvovec or fidanacogene elaparvovec, during a previous study organized by Pfizer.
  • You must be male.
  • You must be a child, as the age range specified for this study is for younger participants.

Who Cannot Join the Study?

  • There are no specific reasons listed that would prevent a person from participating in this study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name City Country Status
University Clinical Hospital Virgen De La Arrixaca Murcia Spain
Rheinische Friedrich-Wilhelms-Universitaet Bonn Bonn Germany
Hospices Civils De Lyon Lyon France
Skåne University Hospital, Department of Hematology, Oncology and Radiation Physics Malmo Sweden
Hospital Universitario Río Hortega Valladolid Spain
Hôpital Universitaire Necker Enfants Malades Paris France
Azienda Ospedaliera Universitaria Federico II Naples Italy
Vrcqsjku Kwquzxvd Fczjwlojubqfpi Berlin Germany
Lcdld Grmuhff Hgvumaoo Oo Ahtojw Athens Greece

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Not yet recruiting
15.07.2026
Germany Germany
Not yet recruiting
15.07.2026
Greece Greece
Not yet recruiting
15.07.2026
Italy Italy
Not yet recruiting
15.07.2026
Spain Spain
Not yet recruiting
15.07.2026
Sweden Sweden
Not yet recruiting
15.07.2026

Trial locations

Fidanacogene elaparvovec is a gene therapy designed to help people with Hemophilia A. It is given through an IV infusion to help the body produce the blood clotting factor that is missing or not working correctly due to the condition.

Giroctocogene fitelparvovec is a gene therapy designed to help people with Hemophilia B. It is given through an IV infusion to help the body produce the blood clotting factor that is missing or not working correctly due to the condition.

Hemophilia A – This is a genetic bleeding disorder caused by a deficiency or lack of clotting factor VIII. Because this protein is missing, the blood cannot clot properly when an injury occurs. Over time, the condition can lead to frequent and spontaneous bleeding into joints and muscles. This repeated bleeding can cause long-term damage to the affected areas.

Hemophilia B – This condition is a genetic disorder resulting from a deficiency of clotting factor IX. The lack of this specific protein prevents the blood from forming clots effectively. As the disease progresses, individuals may experience bleeding episodes in various parts of the body. These episodes can occur following minor injuries or even without a clear cause.

Trial ID:
2025-523660-21-00
Protocol code:
C0371017
Trial Phase:
Therapeutic confirmatory (Phase III)

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