Principal Investigator
Enrollment Status
Clinical Trial ID
Clinical Trial Summary
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)
Phase
Phase 2
Funding Agency/Sponsor
Industry
Disease
Other
Enrollment Eligibility
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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