Active not recruitingPHASE3INTERVENTIONAL

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults

This research is looking at a new type of treatment called gene therapy, named PF-07055480, for men aged 18 to 64 who have moderate to severe haemophilia A. Haemophilia A is a condition where your blood doesn't clot properly. The gene therapy is given as a single drip into a vein. It’s designed to help the body produce more of a specific protein (Factor VIII) that's crucial for blood clotting, which could reduce the need for regular treatments and prevent bleeding episodes. The study will check how well this treatment works and if it's safe over five years. Participants will be men who have already been taking regular Factor VIII treatments as part of a previous study.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Pfizer

Enrolment target

Start

18 Aug 2020

Estimated completion

25 Oct 2026

Hemophilia A

What is this study about?

This study is investigating a new and potentially long-lasting treatment for haemophilia A, a condition that mainly affects men and causes problems with blood clotting. People with haemophilia A don't have enough of a protein called Factor VIII, which is essential for stopping bleeds. Currently, many people manage their haemophilia by regularly injecting Factor VIII to prevent bleeding.

The new treatment being tested is called a 'gene therapy' (specifically, PF-07055480). Gene therapy is a very advanced approach that aims to correct the underlying problem by delivering a working copy of the gene that makes Factor VIII into the body. The idea is that if your body can start making more of its own Factor VIII, you might have fewer bleeding episodes and need less, or even stop, your regular Factor VIII injections.

This is a Phase 3 study, which means it's one of the final steps in testing a new treatment before it might be considered for wider use. Researchers want to understand two main things: firstly, how effective this gene therapy is at preventing bleeds and helping the body produce Factor VIII, and secondly, how safe it is for people to use. They will be carefully watching participants over five years to gather all this important information.

Key takeaways

This study tests a new gene therapy for severe haemophilia A.
It's for adult men already participating in a previous study.
The goal is to help your body make its own Factor VIII.
It involves a single treatment followed by five years of monitoring.
Your regular Factor VIII treatments will be stopped after the gene therapy.
The study assesses both how well it works and its safety.

Who may be eligible?

This study is specifically designed for adult men, aged between 18 and 64, who have moderate to severe haemophilia A. To be considered, you must have already participated in another study (called C0371004) where your regular Factor VIII treatments were monitored. This shows you have a history of needing and responding to Factor VIII replacement therapy. Importantly, your haemophilia A must be quite severe, meaning your body naturally produces very little Factor VIII (1% or less).

There are also some reasons why you might not be able to join. For example, if your body has already built up special antibodies against certain viruses (called anti-AAV6 neutralizing antibodies), or if you've developed an 'inhibitor' to Factor VIII in the past, which means your body has rejected earlier treatments. Also, if you have other serious health conditions like significant liver problems, a high risk of blood clots, or certain active infections like hepatitis B, C, or uncontrolled HIV, you wouldn't be able to take part.

Finally, if you're planning to have surgery that needs Factor VIII treatment within the next year, or if your blood tests show other significant health issues, you wouldn't be eligible. The study plans to stop your regular Factor VIII treatments once you've had the gene therapy infusion, so you need to be prepared for this.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you a man between 18 and 64 years old?
Do you have moderate to severe haemophilia A (Factor VIII less than or equal to 1%)?
Have you participated in the previous study C0371004?
Do you have a history of at least 150 times receiving Factor VIII products?
Do you NOT have an active infection like hepatitis B, C, or uncontrolled HIV?
Have you NOT had an inhibitor to Factor VIII in the past?

Answer every question to see your result.

What does participation involve?

If you join this study, you will receive a single dose of the gene therapy, PF-07055480, given as a drip into a vein (intravenously). A significant part of taking part will involve stopping your usual preventative Factor VIII treatments after you receive the study drug. The research team will regularly check your health, your Factor VIII levels, and monitor for any bleeding episodes over a period of five years. This will involve regular visits to the clinic for blood tests, physical examinations, and discussions about your well-being and any side effects. You'll need to keep a careful record of any bleeds you have. The total duration of your involvement will be five years from the time you receive the gene therapy.

Potential risks and benefits

Participating in this study could offer the potential benefit of a long-term solution for your haemophilia A, possibly reducing or removing your need for frequent Factor VIII injections and potentially lowering your risk of bleeding episodes. However, as with any new treatment, there are potential risks and side effects that will be carefully explained to you by the study team. These could include reactions to the infusion, changes in your liver health, or other unexpected effects from the gene therapy. It's important to understand that your regular preventative Factor VIII treatment will be stopped after receiving the gene therapy, and you will be carefully monitored. You'll have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (45)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

NOW Physical Therapy
Verified postcode
Mountain View, United States
Regents of The University of California
Verified postcode
Oakland, United States
Clinical and Translational Research Unit (CTRU)
Verified postcode
Palo Alto, United States
Lucile Packard Childrens Hospital
Verified postcode
Palo Alto, United States
Imaging Clinic at Stanford Medicine Outpatient Center in Redwood City
Verified postcode
Redwood City, United States
The Board of Trustees of the Leland Stanford Junior University
Verified postcode
Redwood City, United States
UCSF Outpatient Radiology.
Verified postcode
San Francisco, United States
UCSF IDS Pharmacy
Verified postcode
San Francisco, United States
UCSF lnvestigational Drug Service
Verified postcode
San Francisco, United States
UCSF Outpatient Radiology (alternate location)
Verified postcode
San Francisco, United States
UCSF Outpatient Radiology
Verified postcode
San Francisco, United States
University of California, San Francisco - Clinical Research Center
Verified postcode
San Francisco, United States

Common questions

What is gene therapy?

Gene therapy is a treatment that aims to fix the root cause of a disease by introducing new genetic material into your body to help it work correctly, in this case, to help produce Factor VIII.

Will I still need my usual Factor VIII treatment?

After you receive the gene therapy in this study, your regular preventative Factor VIII treatments will be stopped so that the researchers can see how well the gene therapy works on its own.

How long will the study last?

If you participate, you will be part of the study for five years after receiving the single gene therapy treatment.

Is this a new treatment?

Yes, PF-07055480 is a new gene therapy being tested in a Phase 3 study, which means it's still being evaluated before it can be widely used.

Can I join if I'm not in the previous study?

No, this particular study is only for adult men who have already taken part in a specific previous study called C0371004.