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 BHemophilia AStandard 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 HTCaccepting new patients
    San FranciscoCalifornia94117United States
  • University of California, San Francisco - Clinical Research Centeraccepting new patients
    San FranciscoCalifornia94143United States
  • University of California, San Francisco - Outpatient Hematology Clinicaccepting new patients
    San FranciscoCalifornia94143United 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.
ID
NCT03587116
Phase
Phase 3
Study Type
Interventional
Last Updated