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NCT04394286

An Open-Label, Multinational, Phase 1/2 Study of the Safety and Dose Escalation of SHP648, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing FIX Padua in Hemophilia B Subjects

Study Summary

The purpose of this study is to evaluate the safety and dose escalation of SHP648 an adeno-associated viral vector for gene transfer in hemophilia B participants.

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Interventions

SHP648GENETIC
Participants will receive a single IV infusion of SHP648 in Cohort 1, 2, 3 on Day 0.

Study Locations

FacilityCityStateCountry
Hospital Universitario Virgen de la ArrixacaEl PalmarSpain
Ege University Medical FacultyIzmirTurkey (Türkiye)

Official Trial Information

View on ClinicalTrials.gov

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