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Number of Visits: 1

Duration: 1 day

76 Participants Needed

Gene Therapy for Hemophilia A
(AFFINE Trial)

Recruiting at 38 trial locations

Overseen ByPfizer CT.gov Call Center

Age: 18 - 65

Sex: Male

Trial Phase: Phase 3

Sponsor: Pfizer

Must be taking: Factor VIII

Disqualifiers: Liver disease, Thrombophilia, Hepatitis, HIV, others

No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval

Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

Will I have to stop taking my current medications?

Yes, participants will need to stop their routine Factor VIII prophylaxis therapy after receiving the study drug infusion.

What data supports the effectiveness of the treatment PF-07055480 (giroctocogene fitelparovec) for hemophilia A?

Gene therapy for hemophilia, using similar adeno-associated viral (AAV) vectors, has shown promising results in increasing clotting factor levels and reducing bleeding episodes in patients. Other treatments like Roctavian and Hemgenix, which use AAV vectors, have been approved based on their effectiveness in treating hemophilia A and B, suggesting potential for similar therapies like PF-07055480.¹ ² ³ ⁴ ⁵

Is gene therapy for hemophilia A generally safe for humans?

Gene therapy for hemophilia, including treatments like PF-07055480, has shown promising safety data in clinical trials. Most patients experienced short-term liver inflammation, which was managed with medications, and no long-term safety issues have been reported so far.¹ ³ ⁵ ⁶ ⁷

How is the treatment giroctocogene fitelparovec unique for hemophilia A?

Giroctocogene fitelparovec is a gene therapy that uses an adeno-associated virus (AAV) vector to deliver a functional gene for factor VIII, allowing the body to produce its own factor VIII and potentially reducing the need for regular factor replacement therapy. This approach aims to provide a more sustained and stable increase in factor VIII levels, which could lead to fewer bleeding episodes compared to traditional treatments.¹ ³ ⁴ ⁸ ⁹

What is the purpose of this trial?

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.

Research Team

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Eligibility Criteria

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.

Inclusion Criteria

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

My hemophilia A is classified as moderately severe to severe.

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

Exclusion Criteria

Anti-AAV6 neutralizing antibodies

History of inhibitor to Factor VIII

Abnormal or outside acceptable study limits laboratory values at screening visit

See 5 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Single IV infusion of PF-07055480 / giroctocogene fitelparvovec gene therapy

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

5 years

Yearly assessments

Treatment Details

Interventions

PF-07055480 (giroctocogene fitelparovec)

Trial Overview 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.

Participant Groups

1Treatment groups

Experimental Treatment

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

Single administration of PF-07055480

Find a Clinic Near You

Who Is Running the Clinical Trial?

Pfizer

Lead Sponsor

Trials

4,712

Recruited

50,980,000+

Known For

Vaccine Innovations

Findings from Research

Gene therapies like Roctavian® and Hemgenix® have been approved for treating severe hemophilia A and B, respectively, showing significant increases in clotting factor levels and reductions in bleeding episodes based on phase III clinical trial data.

While most patients experienced short-term liver inflammation that was manageable with immune suppression, there remains variability in treatment response, and further research is needed to determine the long-term stability of factor expression and the potential for a permanent cure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hemophilia Gene Therapy: The End of the Beginning?De Wolf, D., Singh, K., Chuah, MK., et al.[2023]

Etranacogene dezaparvovec, a gene therapy for hemophilia, has shown the ability to provide sustained factor IX expression for over 2 years, potentially allowing most patients to live without bleeding episodes or the need for infusions.

The therapy utilizes a specific adeno-associated virus vector and has demonstrated promising efficacy and safety in clinical trials, making it a significant advancement in hemophilia treatment options.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Etranacogene dezaparvovec for hemophilia B gene therapy.Thornburg, CD.[2023]