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NCT05164471

A Dose Confirmation Study of FLT180a (Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene) in Adult Subjects With Hemophilia B

Study Summary

Study of FLT180a gene therapy in adults with Hemophilia B. Up to 9 patients will be enrolled to receive a single dose of FLT180a and be followed for 52 weeks. Results will confirm the dose for a future Phase 3 study.

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Interventions

verbrinacogene setparvovecGENETIC
FLT180a is a gene therapy intended to increase endogenous FIX production in adults with Hemophilia B

Study Locations

FacilityCityStateCountry
Children's Hospital of Los AngelesLos AngelesCaliforniaUnited States
University of South FloridaTampaFloridaUnited States
University of MichiganAnn ArborMichiganUnited States
Glasgow Royal InfirmaryGlasgowUnited Kingdom
Guys HospitalLondonUnited Kingdom
Royal Free London NHS Foundation TustLondonUnited Kingdom
Royal Victoria InfirmaryNewcastleUnited Kingdom

Official Trial Information

View on ClinicalTrials.gov

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