Hemophilia B

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

NCT03569891 | PHASE 3 | INTERVENTIONAL

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.

33 Sites

67 Participants

Recruiting

18 Years to 17 Years

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Find closest sites:

SUBMIT

Phoenix Children's Hospital

Phoenix,Arizona,United States,85016

Arkansas Children's Hospital

Little Rock,Arkansas,United States,72202

Los Angeles Orthopedic Hospital

Los Angeles,California,United States,90007

Children's Hospital of Los Angeles

Los Angeles,California,United States,90027

University of California, Davis

Sacramento,California,United States,95817

University of California, San Diego

San Diego,California,United States,92161

University of Colorado Denver

Aurora,Colorado,United States,80045

Children's National Medical Center Hematology and Oncology

Washington,District of Columbia,United States,20010

University of South Florida

Tampa,Florida,United States,33612

University of Michigan

Ann Arbor,Michigan,United States,48109

Hemophilia Center of Western New York

Buffalo,New York,United States,14209

University of North Carolina, Chapel Hill

Chapel Hill,North Carolina,United States,27599

Oregon Health & Science University

Portland,Oregon,United States,97239

University of Tennessee Health Science Center

Memphis,Tennessee,United States,38163

Vanderbilt University Medical Center

Nashville,Tennessee,United States,37232

University of Texas Health Science Center & Medical School

Houston,Texas,United States,77030

University of Utah

Salt Lake City,Utah,United States,84108

Washington Institute for Coagulation

Seattle,Washington,United States,98101

Bloodworks Northwest

Seattle,Washington,United States,98104

Cliniques Universitaires SaintLuc

Bruxelles,Belgium,1200

University Hospital Leuven

Leuven,Belgium,3000

Righospitalet

Copenhagen,Denmark,2100

Vivantes Klinikum im Friedrichshain

Berlin,Germany,10249

Klinikum der Johann Wolfgang Goethe Universitat

Frankfurt am main,Germany,60590

National Coagulation Centre, St James's Hospital

Dublin,Ireland,D08A978

Amsterdam UMC Locatie AMC

Amsterdam,Netherlands,1105AZ

Universitair Medisch Centrum Groningen

Groningen,Netherlands,9713GZ

Erasmus MC

Rotterdam,Netherlands,3015CE

UMC Utrecht, Van Creveldkliniek

Utrecht,Netherlands,3508GA

Center for Thrombosis and Hemostasis Skne University Hospital Malm

Malmö,Sweden,SE-20502

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

Cambridge,United Kingdom,CB20QQ

The Royal London Hospital (Barts Health NHS Trust)

London,United Kingdom,E12ES

University Hospital Southampton NHS Foundation Trust

Southampton,United Kingdom,SO166YD

Study Eligibility Criteria

Inclusion Criteria

1. Male
2. Age ≥18 years
3. Subjects with congenital hemophilia B, classified as severe or moderately severe, and are currently on factor IX prophylaxis
4. \>150 previous exposure days of treatment with factor IX protein

Exclusion Criteria

1. History of factor IX inhibitors
2. Positive factor IX inhibitor test at screening
3. Select screening laboratory value \>2 times upper limit of normal
4. Positive human immunodeficiency virus (HIV) test at screening, not controlled with anti-viral therapy
5. Active infection with hepatitis B or C virus at screening
6. History of Hepatitis B or C exposure, currently controlled by antiviral therapy at the end of the lead-in phase
7. Previous gene therapy treatment
8. Receipt of an experimental agent within 60 days prior to screening
9. Current participation or anticipated participation within one year after study drug administration in this trial in any other interventional clinical trial involving drugs or devices

Additional Studies

Additional studies can be found at ClinicalTrials.gov