Summary

Eligibility
for males ages 18 years and up (full criteria)
Location
at UC Davis UCSF
Dates
study started
completion around

Description

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)

Official Title

A PHASE 1/2, OPEN-LABEL, ADAPTIVE, DOSE-RANGING STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF SB-525 (PF-07055480) (RECOMBINANT AAV2/6 HUMAN FACTOR 8 GENE THERAPY) IN ADULT SUBJECTS WITH SEVERE HEMOPHILIA A

Details

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.

Keywords

Hemophilia A

Eligibility

You can join if…

Open to males ages 18 years and up

  • 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 CAN'T join 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

Locations

  • UC Davis Ambulatory Care Clinic
    Sacramento California 95817 United States
  • UC Davis Comprehensive Cancer Center
    Sacramento California 95817 United States
  • UC Davis CTSC Clinical Research Center
    Sacramento California 95817 United States
  • UC Davis Hemophilia Treatment Center
    Sacramento California 95817 United States
  • UC Davis Investigational Drug Services Pharmacy
    Sacramento California 95817 United States
  • UC Davis Medical Center
    Sacramento California 95817 United States
  • University of California, San Francisco - Investigational Drug Service (IDS) Pharmacy
    San Francisco California 94143-0622 United States
  • University of California, San Francisco - Outpatient Hematology Clinic
    San Francisco California 94143 United States
  • University of California, San Francisco -Moffitt Hospital
    San Francisco California 94143 United States
  • Midtown Ambulatory Care Center
    Sacramento California 95817 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Pfizer
Links
To obtain contact information for a study center near you, click here.
ID
NCT03061201
Phase
Phase 2 Hemophilia Research Study
Study Type
Interventional
Participants
About 11 people participating
Last Updated