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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 : September 14, 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, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Michael Recht, MD    503-494-8716    rechtm@ohsu.edu   
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: September 14, 2018
Last Verified: September 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