Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals Wi… (NCT03307980) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B
United States21 participantsStarted 2017-06-22
Plain-language summary
Long-term safety and efficacy follow-up for participants with Hemophilia B who were previously treated in the C0371005 (formerly SPK-9001-101) study, and a dose-escalation sub-study evaluating safety, tolerability, and kinetics of a higher dose with long-term safety and efficacy follow-up. Participants in the substudy do not need to have participated in C0371005.
Who can participate
Age range18 Years – 65 Years
SexMALE
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Inclusion criteria
✓. Able to provide informed consent and comply with requirements of the study
✓. Males age 18 to 65 years with confirmed diagnosis of hemophilia B (≤2 IU/dL or ≤2% endogenous factor IX)
✓. Received ≥50 exposure days to factor IX products
✓. No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein
✓. Agree to refrain from donating sperm and either abstain from intercourse or use reliable barrier contraception until 3 consecutive semen samples are negative for vector sequences
Exclusion criteria
✕. Evidence of active hepatitis B or C
✕. Currently on antiviral therapy for hepatitis B or C
✕. Have significant underlying liver disease
✕. Serological evidence\* of HIV-1 or HIV-2 with CD4 counts ≤200/mm3 (\* participants who are HIV+ and stable with CD4 count \>200/mm3 and undetectable viral load are eligible to enroll)
✕. Neutralizing antibody titers to the capsid portion of PF-06838435 above the established threshold
✕. Sensitivity to heparin or heparin induced thrombocytopenia; sensitivity to any of the study interventions, or components thereof, or drug or other allergy