HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

PHASE3CompletedINTERVENTIONAL
Enrollment

67

Participants

Timeline

Start Date

June 27, 2018

Primary Completion Date

September 22, 2021

Study Completion Date

March 19, 2025

Conditions
Hemophilia B
Interventions
GENETIC

AAV5-hFIXco-Padua

Single intravenous infusion of AAV5-hFIXco-Padua (AMT-061)

BIOLOGICAL

Factor IX (FIX)

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.

Trial Locations (33)

1200

Cliniques Universitaires Saint-Luc, Brussels

2100

Righospitalet, Copenhagen

3000

University Hospital Leuven, Leuven

10249

Vivantes Klinikum im Friedrichshain, Berlin

14209

Hemophilia Center of Western New York, Buffalo

20010

Children's National Medical Center Hematology and Oncology, Washington D.C.

27599

University of North Carolina, Chapel Hill, Chapel Hill

33612

University of South Florida, Tampa

37232

Vanderbilt University Medical Center, Nashville

38163

University of Tennessee Health Science Center, Memphis

48109

University of Michigan, Ann Arbor

60590

Klinikum der Johann Wolfgang Goethe Universitat, Frankfurt am Main

72202

Arkansas Children's Hospital, Little Rock

77030

University of Texas Health Science Center & Medical School, Houston

80045

University of Colorado Denver, Aurora

84108

University of Utah, Salt Lake City

85016

Phoenix Children's Hospital, Phoenix

90007

Los Angeles Orthopedic Hospital, Los Angeles

90027

Children's Hospital of Los Angeles, Los Angeles

92161

University of California, San Diego, San Diego

95817

University of California, Davis, Sacramento

97239

Oregon Health & Science University, Portland

98101

Washington Institute for Coagulation, Seattle

98104

Bloodworks Northwest, Seattle

D08 A978

National Coagulation Centre, St James's Hospital, Dublin

1105 AZ

Amsterdam UMC - Locatie AMC, Amsterdam

9713 GZ

Universitair Medisch Centrum Groningen, Groningen

3015 CE

Erasmus MC, Rotterdam

3508 GA

UMC Utrecht, Van Creveldkliniek, Utrecht

SE-205 02

Center for Thrombosis and Hemostasis Skåne University Hospital Malmö, Malmo

CB2 0QQ

The Cambridge Haemophilia and Thrombophilia Centre Camridge University Hospitals NHS Foundation Trust - Box 217 Addenbrooke's Hospital, Cambridge

E1 2ES

The Royal London Hospital (Barts Health NHS Trust), London

SO16 6YD

University Hospital Southampton NHS Foundation Trust, Southampton

Sponsors

Lead Sponsor

All Listed Sponsors
lead

CSL Behring

INDUSTRY