Six Month lead-in Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%)
a study on Hemophilia
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.
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
Hemophilia BHemophilia AStandard of Care FIX Replacement therapy
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.
- 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
- accepting new patients
- Start Date
- Completion Date
- To obtain contact information for a study center near you, click here.
- Phase 3
- Study Type
- Last Updated
Please contact me about this study
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If you do not hear from the study team, please call 888-689-8273 and tell them you’re interested in study number NCT03587116.