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

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ClinicalTrials.gov Identifier: NCT03569891
Recruitment Status : Recruiting
First Posted : June 26, 2018
Last Update Posted : December 11, 2018
Sponsor:
Information provided by (Responsible Party):
UniQure Biopharma B.V.

Brief 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.


Condition or disease Intervention/treatment Phase
Hemophilia B Genetic: AAV5-hFIXco-Padua Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 56 participants
Intervention Model: Single Group Assignment
Intervention Model Description: The reference therapy is prophylactic Factor IX replacement therapy used during the lead-in phase prior to treatment with AAV5-hFIXco-Padua (AMT-061).
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : June 27, 2018
Estimated Primary Completion Date : March 2020
Estimated Study Completion Date : August 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: AMT-061

Single infusion of AMT-061

Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline. After study drug administration (post study drug), subjects will be monitored for tolerance to the study drug and detection of potential immediate AEs at the clinical trial site for a few hours after dosing.

Genetic: AAV5-hFIXco-Padua
Single intravenous infusion of AAV5-hFIXco-Padua (AMT-061)
Other Name: AMT-061




Primary Outcome Measures :
  1. Factor IX activity levels [ Time Frame: 26 weeks ]
    Assessment of Factor IX activity after a single dose of AMT-061


Secondary Outcome Measures :
  1. Annualized bleeding rate (ABR) [ Time Frame: 52 weeks ]
    Comparison of ABR between prophylaxis used in the lead-in and after administration of AMT-061

  2. Use of Factor IX replacement therapy [ Time Frame: 52 weeks ]
    Patients will record all use of prophylactic Factor IX replacement therapy in an e-diary, including reason for Factor IX use, date, and time of infusion and total dose

  3. Adverse events [ Time Frame: 5 years ]
    Follow up and assess any adverse events reported for safety



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03569891


Contacts
Contact: Alison Long, MD 339-970-7578 a.long@uniqure.com
Contact: uniQure HOPE-B uniqureHOPE-B@uniqure.com

Locations
United States, Arizona
Phoenix Children's Hospital Recruiting
Phoenix, Arizona, United States, 85016
Contact: Erica Olson       Eolson1@phoenixchildrens.com   
United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
Contact       uniqureHOPE-B@uniqure.com   
United States, California
Los Angeles Orthopedic Hospital Recruiting
Los Angeles, California, United States, 90007
Contact: Manny Mangilit    213-742-1402      
Principal Investigator: Doris Quon, MD         
Children's Hospital of Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Jeanie Lee    323-361-3478    hslee@chla.usc.edu   
Principal Investigator: Guy Young, MD         
United States, Colorado
University of Colorado Denver Recruiting
Aurora, Colorado, United States, 80045
Contact: Jason Thomas    303-724-3856    Jason.r.thomas@ucdenver.edu   
United States, Florida
University of South Florida Recruiting
Tampa, Florida, United States, 33612
Contact: Hazina Joseph    813-974-4088    hjoseph1@health.usf.edu   
United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Andy Brosius    734-764-9692    abrosius@med.umich.edu   
Michigan State University Recruiting
East Lansing, Michigan, United States, 48824
Contact: Kathleen Anderson    517-353-9385      
United States, North Carolina
University of North Carolina, Chapel Hill Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Nigel Key, MD    919-966-4736 ext 6    unchtcresearch@med.unc.edu   
United States, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Michael Recht, MD    503-494-8716    rechtm@ohsu.edu   
United States, South Carolina
Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 29425
Contact       uniqureHOPE-B@uniqure.com   
United States, Tennessee
University of Tennessee Health Science Center Recruiting
Memphis, Tennessee, United States, 38163
Contact: Osha Smith-Payne       osha.smith-payne@uthsc.edu   
Contact: Sandeep Rajan, MD       srajan2@uthsc.edu   
Principal Investigator: Sandeep Rajan, MD         
Vanderbilt University Medical Center Recruiting
Nashville, Tennessee, United States, 37232
Contact       uniqureHOPE-B@uniqure.com   
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States, 84108
Contact: Richard Lemons, MD    801-662-4700      
United Kingdom
The Cambridge Haemophilia and Thrombophilia Centre Cambridge University Hospitals NHS Foundation Trust - Box 217 - Addenbrooke's Hospital Recruiting
Cambridge, United Kingdom, CB2 0QQ
Contact: Emily Symington, MD       haemophilia.centre@addenbrookes.nhs.uk   
The Royal London Hospital (Barts Health NHS Trust) Recruiting
London, United Kingdom, E1 2ES
Contact: Fran Watson    02032460261    fran.watson1@nhs.net   
Principal Investigator: John Pasi         
Sponsors and Collaborators
UniQure Biopharma B.V.
Investigators
Principal Investigator: Steven Pipe, MD University of Michigan

Responsible Party: UniQure Biopharma B.V.
ClinicalTrials.gov Identifier: NCT03569891     History of Changes
Other Study ID Numbers: CT-AMT-061-02
First Posted: June 26, 2018    Key Record Dates
Last Update Posted: December 11, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by UniQure Biopharma B.V.:
Gene therapy
FIX
Factor IX
Bleeding
Viral vector
Padua
Hemophilia
AAV (adeno-associated virus)
serotype 5 AAV (adeno-associated virus)
serotype 5

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked