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Protocol Title: Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants with Moderately Severe to Severe Hemophilia A (FVIII:C≤1%)
EU CTIS ID: 2024-512075-12-00
What this study is testing
Evaluate the efficacy of a single infusion of PF-07055480 in participants≥18 and <65 years of age with moderately severe to severe hemophilia A (FVIII C ≤ 1%).
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Males who have been followed on routine Factor VIII prophylaxis therapy in 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%)
- Suspension of FVIII prophylaxis therapy post study drug infusion
You likely can't join if
- Anti-AAV6 neutralizing antibodies
- History of inhibitor to Factor VIII
- Laboratory values at screening visit that are abnormal or outside acceptable study limits
- Significant and/or unstable liver disease, biliary disease, significant liver fibrosis
- Planned surgical procedure requiring Factor VIII surgical prophylactic factor treatment 12 months from screening visit
- Active hepatitis B or C
See the full eligibility criteria
- Males who have been followed on routine Factor VIII prophylaxis therapy in 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%)
- Suspension of FVIII prophylaxis therapy post study drug infusion
- Anti-AAV6 neutralizing antibodies
- History of inhibitor to Factor VIII
- Laboratory values at screening visit that are abnormal or outside acceptable study limits
- Significant and/or unstable liver disease, biliary disease, significant liver fibrosis
- Planned surgical procedure requiring Factor VIII surgical prophylactic factor treatment 12 months from screening visit
- Active hepatitis B or C
- Serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with either Cluster of Differentiation 4 positive (CD4+) cell count ≤200 mm3 or viral load >20 copies/mL
The study team makes the final eligibility decision.
Where it's taking place
- Saudi Arabia
- Taiwan
- Korea, Republic of
- Australia
- Turkey
- United States
- United Kingdom
- Brazil
- Canada
- Japan
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Saudi Arabia; Taiwan; Korea, Republic of; Australia; Turkey; United States and 4 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.