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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%)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5-year study period
Awards & highlights

AFFINE Trial Summary

This trial will test the efficacy and safety of a new gene therapy for hemophilia A.

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.Check my eligibility
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.See study design
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.

AFFINE Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am a male with >= 150 days of Factor VIII treatment from the C0371004 study.
Select...
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.

AFFINE Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5-year study period
This trial's timeline: 3 weeks for screening, Varies for treatment, and 5-year study period for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Total annualized bleeding rate (ABR)
Secondary outcome measures
Annualized FVIII consumption
Annualized bleeding rate (ABR)
Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location
+6 more

AFFINE Trial Design

1Treatment groups
Experimental Treatment
Group I: PF-07055480 (giroctocogene fitelparvovec)Experimental Treatment1 Intervention
Single administration of PF-07055480

Find a Location

Who is running the clinical trial?

PfizerLead Sponsor
4,567 Previous Clinical Trials
10,911,797 Total Patients Enrolled
43 Trials studying Hemophilia A
6,139 Patients Enrolled for Hemophilia A
Pfizer CT.gov Call CenterStudy DirectorPfizer
3,474 Previous Clinical Trials
8,092,965 Total Patients Enrolled
42 Trials studying Hemophilia A
2,880 Patients Enrolled for Hemophilia A

Media Library

PF-07055480 (giroctocogene fitelparovec) (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04370054 — Phase 3
Hemophilia A Research Study Groups: PF-07055480 (giroctocogene fitelparvovec)
Hemophilia A Clinical Trial 2023: PF-07055480 (giroctocogene fitelparovec) Highlights & Side Effects. Trial Name: NCT04370054 — Phase 3
PF-07055480 (giroctocogene fitelparovec) (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04370054 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What medical condition does PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy help patients with the most?

"von willebrand disease is most often treated with PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy. However, this same treatment can also be useful for other conditions such as desmopressin, type 3 von willebrand's disease, and hemophilia."

Answered by AI

Can you please tell us about other times that PF-07055480 (giroctocogene fitelparovec) has been used in medical trials?

"At this time, there are 4 clinical trials investigating PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy. One of these trials is in Phase 3. Most of the studies for this medication are based in Seattle, Washington, but there are 73 total locations running trials for it."

Answered by AI

Has this clinical trial been conducted before?

"PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy has been studied since 2007, when the first clinical trial was sponsored by Grifols Biologicals, LLC. 15 patients were involved in this initial study. Following the successful completion of this Phase 1 trial, PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy received its Phase 4 drug approval. As of today, there are 4 live studies for PF-07055480 (giroctoc"

Answered by AI

Are there any available slots left for this experiment?

"Yes, this study is still open to patient recruitment as of the latest update on September 16th, 2020. The original posting was 18 August 2020."

Answered by AI

Does this research require participants to be of a certain age?

"According to the inclusion criteria, only patients aged 18 to 64 can enroll in this clinical trial. There are other trials available for both younger and older demographics."

Answered by AI

Could I join the ranks of this research?

"Sixty-three male patients with hemophilia A, aged 18 to 64, who have been following a prophylactic Factor VIII therapy routine and have over 150 documented exposure days to a Factor VIII protein product are eligible for this study. Additionally, applicants should be moderately severe to severely affected by hemophilia A (Factor VIII activity at or below 1%) and have suspended their FVIII prophylaxis post-study drug infusion."

Answered by AI

How many individuals are being given this opportunity to participate in the trial?

"In order to conduct this study, 63 individuals who meet the pre-determined requirements need to enroll. These individuals can come from different locations such as Hamilton, Ontario and Palo Alto, California."

Answered by AI

In how many different places is this trial taking place?

"There are 14 sites recruiting patients for this study. The locations include McMaster University Medical Centre - Hamilton Health Sciences in Hamilton, Washington Institute for Coagulation in Palo Alto, and Clinical and Translational Research Unit (CTRU) in San Francisco."

Answered by AI

What is the regulatory status of PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy?

"Because this is a Phase 3 trial, there is some data supporting efficacy and multiple rounds of data supporting safety, so our team at Power estimates the safety of PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy to be a 3."

Answered by AI

Who else is applying?

What site did they apply to?
University of California, San Francisco - Moffitt/Long Inpatient Hematology
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
2020. "Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04370054.
All > Hemophilia A > Hemophilia
~2 spots leftby Jun 2024
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