Status:
ACTIVE_NOT_RECRUITING
Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B
Lead Sponsor:
Pfizer
Conditions:
Hemophilia B
Eligibility:
MALE
18-65 years
Phase:
PHASE2
Brief 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,...
Detailed Description
Evaluation of the long-term level of persistence and potential late or delayed adverse events associated with PF-06838435 (formerly SPK-9001), assessment of the durability of the transgene expression,...
Eligibility Criteria
Inclusion
- This study is currently only enrolling into the dose-escalation substudy with subsequent long-term follow-up. The Eligibility Criteria for entry into the dose-escalation substudy is presented below:
- 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
- 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
- Previously dosed in a gene therapy research trial at any time or in an interventional clinical study within 3 months of screening visit
- Any concurrent clinically significant major disease or condition
- Unable or unwilling to comply with the study procedures
Key Trial Info
Start Date :
June 22 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 8 2029
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT03307980
Start Date
June 22 2017
End Date
June 8 2029
Last Update
September 3 2025
Active Locations (22)
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1
UC Davis Comprehensive Cancer Center
Sacramento, California, United States, 95817
2
UC Davis Ellison Ambulatory Care Clinic
Sacramento, California, United States, 95817
3
UC Davis Medical Center department of Radiology
Sacramento, California, United States, 95817
4
UC Davis Medical Center
Sacramento, California, United States, 95817