Summary

for males ages 18-64 (full criteria)
at San Francisco, California and other locations
study started
estimated completion:

Description

Summary

To establish a minimum of 6 months of prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for NAb to AAV-Spark100, prior to the Phase 3 gene therapy study.

Official Title

An Open-label, Non-investigational Product, Multi-center, Lead-in Study To Evaluate At Least 6 Months Of Prospective Efficacy And Selected Safety Data Of Factor Ix (Fix) Prophylaxis Replacement Therapy In The Usual Care Setting Of Moderately Severe To Severe Adult Hemophilia B Subjects (Fix:c≤2%) Who Are Negative For Neutralizing Antibodies (Nab) To Adeno-associated Virus Vector (Aav)-spark100

Keywords

Hemophilia B Hemophilia A Standard of Care FIX Replacement therapy

Eligibility

You can join if…

Open to males ages 18-64

  • Males ≥ 18 and < 65 years of age with moderate severe to severe hemophilia B and documented FIX activity (≤2%) within the last 12 months prior to baseline visit.
  • Willing and able to comply with scheduled visits, FIX prophylaxis treatment plan,laboratory tests and other study procedures.
  • Previous experience with FIX therapy (≥50 documented exposure days to a FIX protein product such as recombinant, plasma-derived or extended half-life FIX product)
  • Subjects as per usual care setting on FIX prophylaxis replacement therapy(recombinant, plasma-derived or extended half-life FIX product) must have the intention to remain on FIX prophylaxis replacement therapy for the duration of the study
  • No known hypersensitivity to FIX replacement product
  • No history of FIX inhibitor (clinical or laboratory-based assessment) defined as a titer ≥ to 0.6 BU/mL

You CAN'T join if...

  • Anti-AAV-Spark100 neutralizing antibodies titer (above the lowest detectable titer)performed by a central laboratory during screening.
  • Lack of patient compliance with documentation of bleeds and/or FIX prophylaxis replacement therapy administration.
  • A subject is not eligible if either HbsAg is positive or HBV-DNA is positive/detectable.
  • A subject who is currently undergoing anti-viral therapy for hepatitis B is not eligible
  • A subject who is currently undergoing anti-viral therapy for chronic hepatitis C is not eligible.
  • A subject is not eligible if his HCV-RNA load assay result is positive/detectable.
  • Currently on antiviral therapy for hepatitis B or C.
  • A subject is not eligible if any of the following pre-existing diagnoses, which are indicative of significant underlying liver disease, are present in the medical record:
  • Portal hypertension; or
  • Splenomegaly; or
  • Hepatic encephalopathy
  • A subject is not eligible if the serum albumin level is below the testing laboratory's lower limit of normal; and
  • At least one of the following diagnostic tests for liver fibrosis indicating ≥stage 3. The following results are indicative of fibrosis ≥ stage 3 and exclude the subject from participation:
  • FibroScan, with a score >8.3 kPa units;
  • FibroTest/FibroSURE with a result >0.48; or
  • AST-to-Platelet Ratio Index (APRI) >1.
  • Documented serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with Cluster of Differentiation 4 positive (CD4+) cell count ≤200 mm3 within the last 12 months prior to screening.
  • History of chronic infection or other chronic disease that the investigator deems an unacceptable risk. Any patient with a history of thrombotic events including but not limited to stroke or myocardial infarction. Any concurrent clinically significant major disease or condition that the investigator deems unsuitable for participation.
  • Participation in other studies involving investigational drug(s) within the last 3 months prior to study entry and/or during study participation or in a previous gene therapy clinical study within the last 12 months prior to screening.
  • Any subject with a planned surgical procedure requiring FIX surgical prophylactic factor treatment in the next 24 months.

Locations

  • University of California San Francisco - UCSF HTC not yet accepting patients
    San Francisco California 94117 United States
  • University of California, San Francisco not yet accepting patients
    San Francisco California 94117 United States
  • University of California, San Francisco - Clinical Research Center not yet accepting patients
    San Francisco California 94143 United States
  • University of California, San Francisco - Outpatient Hematology Clinic not yet accepting patients
    San Francisco California 94143 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Pfizer
Links
To obtain contact information for a study center near you, click here.
To obtain contact information for a study center near you, click here.
To obtain contact information for a study center near you, click here.
To obtain contact information for a study center near you, click here.
ID
NCT03587116
Phase
Phase 3
Study Type
Interventional
Last Updated