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NCT03569891

Phase III, Open-label, Single-dose, Multi-center, Multinational Trial Investigating a Serotype 5 Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene (AAV5-hFIXco-Padua, AMT-061) Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B

Study Summary

This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10\^13 gc/kg.

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Interventions

AAV5-hFIXco-PaduaGENETIC
Single intravenous infusion of AAV5-hFIXco-Padua (AMT-061)
Factor IX (FIX)BIOLOGICAL
During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.

Study Locations

FacilityCityStateCountry
Phoenix Children's HospitalPhoenixArizonaUnited States
Arkansas Children's HospitalLittle RockArkansasUnited States
Los Angeles Orthopedic HospitalLos AngelesCaliforniaUnited States
Children's Hospital of Los AngelesLos AngelesCaliforniaUnited States
University of California, DavisSacramentoCaliforniaUnited States
University of California, San DiegoSan DiegoCaliforniaUnited States
University of Colorado DenverAuroraColoradoUnited States
Children's National Medical Center Hematology and OncologyWashington D.C.District of ColumbiaUnited States
University of South FloridaTampaFloridaUnited States
University of MichiganAnn ArborMichiganUnited States
Hemophilia Center of Western New YorkBuffaloNew YorkUnited States
University of North Carolina, Chapel HillChapel HillNorth CarolinaUnited States
Oregon Health & Science UniversityPortlandOregonUnited States
University of Tennessee Health Science CenterMemphisTennesseeUnited States
Vanderbilt University Medical CenterNashvilleTennesseeUnited States
University of Texas Health Science Center & Medical SchoolHoustonTexasUnited States
University of UtahSalt Lake CityUtahUnited States
Washington Institute for CoagulationSeattleWashingtonUnited States
Bloodworks NorthwestSeattleWashingtonUnited States
Cliniques Universitaires Saint-LucBrusselsBelgium
University Hospital LeuvenLeuvenBelgium
RighospitaletCopenhagenDenmark
Vivantes Klinikum im FriedrichshainBerlinGermany
Klinikum der Johann Wolfgang Goethe UniversitatFrankfurt am MainGermany
National Coagulation Centre, St James's HospitalDublinIreland
Amsterdam UMC - Locatie AMCAmsterdamNetherlands
Universitair Medisch Centrum GroningenGroningenNetherlands
Erasmus MCRotterdamNetherlands
UMC Utrecht, Van CreveldkliniekUtrechtNetherlands
Center for Thrombosis and Hemostasis Skåne University Hospital MalmöMalmoSweden
The Cambridge Haemophilia and Thrombophilia Centre Camridge University Hospitals NHS Foundation Trust - Box 217 Addenbrooke's HospitalCambridgeUnited Kingdom
The Royal London Hospital (Barts Health NHS Trust)LondonUnited Kingdom
University Hospital Southampton NHS Foundation TrustSouthamptonUnited Kingdom

Official Trial Information

View on ClinicalTrials.gov

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