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NCT02484092

Gene Therapy, Open-label, Dose-escalation Study of PF-06838435 (SPK-9001) [Adeno-associated Viral Vector With Human Factor IX Gene] in Subjects With Hemophilia B

Study Summary

A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.

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Interventions

SPK-9001BIOLOGICAL
A novel, bioengineered adeno-associated viral vector carrying human factor IX variant

Study Locations

FacilityCityStateCountry
UC Davis Comprehensive Cancer CenterSacramentoCaliforniaUnited States
UC Davis CTSC Clinical Research CenterSacramentoCaliforniaUnited States
UC Davis Ellison Ambulatory Care ClinicSacramentoCaliforniaUnited States
UC Davis Investigational PharmacySacramentoCaliforniaUnited States
UC Davis Medical CenterSacramentoCaliforniaUnited States
University of Mississippi Medical CenterJacksonMississippiUnited States
Mississippi Center for Advanced MedicineMadisonMississippiUnited States
Weill Cornell Medicine - New York Presbyterian HospitalNew YorkNew YorkUnited States
The Children's Hospital of PhiladelphiaPhiladelphiaPennsylvaniaUnited States
Royal Prince Alfred HospitalCamperdown/SydneyNew South WalesAustralia

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026