What side effects are associated with this type of intervention?

Common treatments for Hemophilia A, such as recombinant factor VIII products, can cause side effects including the development of inhibitors (antibodies against factor VIII), allergic reactions, and infusion-related reactions. Gene therapies, like PF-07055480 / giroctocogene fitelparvovec, are promising but may lead to immune responses to the viral vector, liver enzyme elevations, and other long-term unknown effects. These treatments aim to reduce bleeding episodes but come with their own set of potential risks.

What prior FDA approvals exist?

The FDA has approved several treatments for Hemophilia A, focusing on recombinant factor VIII products and gene therapies. Notable approvals include recombinant factor VIII-Fc fusion (Eloctate), which extends half-life by binding to the neonatal Fc receptor, and PEGylated recombinant factor VIII, which uses polyethylene glycol to prolong activity. Additionally, gene therapies like PF-07055480 / giroctocogene fitelparvovec are being studied to provide long-term solutions by introducing functional copies of the factor VIII gene.

What other types of research exist?

Promising alternatives to PF-07055480 / giroctocogene fitelparvovec for Hemophilia A patients include extended-half-life recombinant FVIII products such as rFVIIIFc (efmoroctocog alfa, Elocta®/Eloctate®), BAY 94-9027 (damoctocog alfa pegol, Jivi®), BAX 855 (rurioctocog alfa pegol, Adynovate®), and N8-GP (turoctocog alfa pegol, Esperoct®). These products have demonstrated efficacy and safety in clinical studies and offer the benefit of less frequent dosing.

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Gene Therapy

Gene Therapy for Hemophilia A (AFFINE Trial)

Phase 3

Waitlist Available

Research Sponsored by Pfizer

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Males who have been followed on routine Factor VIII prophylaxis therapy during the lead-in study (C0371004) and have >= 150 documented exposure days to a Factor VIII protein product

Moderately severe to severe hemophilia A (Factor VIII activity <=1%)

Must not have

Significant and/or unstable liver disease, biliary disease, significant liver fibrosis

Active hepatitis B or C

Timeline

Screening 3 weeks

Treatment Varies

Follow Up yearly up to 5 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial tests a single treatment for adult males with severe hemophilia A. The treatment aims to help their bodies produce a necessary protein, reducing the need for regular preventive treatments. Research has been ongoing for a long time to enable the body to produce this protein and reduce bleeding episodes.

Who is the study for?

Adult males with moderately severe to severe hemophilia A (Factor VIII activity ≤1%) who have been on routine Factor VIII prophylaxis and participated in the lead-in study C0371004 with ≥150 exposure days to a Factor VIII product. Excluded are those with anti-AAV6 antibodies, history of Factor VIII inhibitors, abnormal lab values, thrombosis risk, planned surgeries requiring prophylaxis within 12 months, active hepatitis B or C, significant HIV infection or liver disease.

What is being tested?

The trial is testing PF-07055480 (giroctocogene fitelparvovec), a gene therapy delivered through a single IV infusion aimed at treating hemophilia A. The efficacy and safety will be monitored over five years in participants who suspend their usual FVIII prophylaxis post-infusion.

What are the potential side effects?

Potential side effects may include immune reactions to the AAV6 vector used for gene delivery such as inflammation or allergic responses. There could also be risks related to blood clotting abnormalities due to changes in Factor VIII levels.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am a male with >= 150 days of Factor VIII treatment from the C0371004 study.

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My hemophilia A is classified as moderately severe to severe.

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I will stop my FVIII treatments after starting the study drug.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have serious or unstable liver or biliary disease.

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I have active hepatitis B or C.

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I am HIV positive with a CD4 count of 200 or less and/or a viral load over 20.

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I have a condition that increases my risk of blood clots.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ yearly up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~yearly up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and yearly up to 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change in joint health using HJHS (Hemophilia Joint Health Score)

Hemophilia A

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: PF-07055480 (giroctocogene fitelparvovec)Experimental Treatment1 Intervention

Single administration of PF-07055480

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Hemophilia A include recombinant factor VIII products, gene therapy, and monoclonal antibodies. Recombinant factor VIII products, such as rFVIII-Fc and turoctocog alfa, replace the deficient factor VIII in patients, helping to prevent and control bleeding episodes. Gene therapy, like PF-07055480 / giroctocogene fitelparvovec, introduces a functional copy of the factor VIII gene into the patient's cells, potentially providing a long-term solution by enabling the body to produce its own factor VIII. Monoclonal antibodies, such as emicizumab, mimic the function of factor VIII by bridging activated factor IX and factor X, facilitating blood clotting. These treatments are crucial for Hemophilia A patients as they help manage bleeding risks, improve quality of life, and reduce the frequency of treatment administration.

Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B.Turoctocog alfa for the treatment of hemophilia a.