A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A

Completed · Phase 2

Conditions studied: Hemophilia A

In brief

The purpose of the study is to evaluate the safety, tolerability and time-course profile of FVIII activity after dosing with SB-525 (PF-07055480)

Key facts

Study ID

NCT03061201

Run by

Pfizer

People needed

13

Starts

2017-06-21

Expected to finish

2024-07-16

Last updated by the study team

2025-09-30

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Male ≥18 years of age
• Severe hemophilia A (past evidence of circulating FVIII activity of < 1% normal)
• Treated or exposed to FVIII concentrates or cryoprecipitate for at least 150 exposure days
• ≥12 bleeding episodes if receiving on-demand therapy over the preceding 12 months
• Agree to use double barrier contraceptive until at least 3 consecutive semen samples are negative for AAV 2/6 after SB-525 infusion

You may not qualify if…

• Presence of neutralizing antibodies
• Current inhibitor, or history of FVIII inhibitor (except for transient low titer inhibitor detected in childhood)
• History of hypersensitivity response to FVIII
• History of Hepatitis B or HIV-1/2 infection
• History of Hepatitis C, unless viral assays in two samples, collected at least 6 months apart, are negative
• Evidence of any bleeding disorder in addition to hemophilia A
• Markers of hepatic inflammation or overt or occult cirrhosis
• History of chronic renal disease or creatinine ≥ 1.5 mg/dL
• Presence of liver mass on magnetic resonance imaging (MRI), or, positive alpha fetoprotein
• Presence of > grade 2 liver fibrosis on elastography for subjects with history of treated Hepatitis C or suspicion of chronic liver disease

Where it is running

• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• City of Hope Medical Center — Duarte, California, United States
• Midtown Ambulatory Care Center — Sacramento, California, United States
• UC Davis Ambulatory Care Clinic — Sacramento, California, United States
• UC Davis Comprehensive Cancer Center — Sacramento, California, United States
• UC Davis CTSC Clinical Research Center — Sacramento, California, United States
• UC Davis Hemophilia Treatment Center — Sacramento, California, United States
• UC Davis Investigational Drug Services Pharmacy — Sacramento, California, United States
• UC Davis Medical Center — Sacramento, California, United States
• University of California, San Francisco - Investigational Drug Service (IDS) Pharmacy — San Francisco, California, United States
• University of California, San Francisco - Outpatient Hematology Clinic — San Francisco, California, United States
• University of California, San Francisco -Moffitt Hospital — San Francisco, California, United States
• University Of Miami Hospital and Clinics/Sylvester Comprehensive Cancer Center — Miami, Florida, United States
• USF Health Morsani Center For Advanced Healthcare — Tampa, Florida, United States
• Emory University School of Medicine — Atlanta, Georgia, United States
• Hemophilia Center of Western PA — Pittsburgh, Pennsylvania, United States
• UPMC Montefiore Clinical and Translational Research Center — Pittsburgh, Pennsylvania, United States
• UPMC, Investigational Drug Service — Pittsburgh, Pennsylvania, United States
• Vanderbilt University Medical Center Clinical Research Center — Nashville, Tennessee, United States
• Vanderbilt Hemostasis Treatment Clinic — Nashville, Tennessee, United States
• Vanderbilt Hemostasis-Thrombosis Clinic — Nashville, Tennessee, United States
• Washington Institute for Coagulation — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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