Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Pfizer
The proposed clinical study uses a recombinant adeno-associated virus 2/6 (AAV2/6) vector encoding the cDNA for the B-domain deleted human F8 (hF8). The secreted FVIII has the same amino acid sequence as approved recombinant anti hemophilic factors (Refacto® and Xyntha®). The SB-525 (PF-07055480) vector encodes a liver-specific promotor module and AAV2/6 exhibits liver tropism, thus providing the potential for long-term hepatic production of FVIII in hemophilia A subjects.
The constant production of FVIII after a single SB-525 (PF-07055480) administration may provide potential benefit in durable protection against bleeding and the complications thereof without lifelong repetitive IV factor replacement administration.
Age
18–any
Sex
MALE
Healthy volunteers
Not accepted
You may be eligible 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 be 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
Clareo Health | A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A
