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Trial record 1 of 1 for:    NCT03569891
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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 : March 14, 2019
Sponsor:
Information provided by (Responsible Party):
UniQure Biopharma B.V.

Tracking Information
First Submitted Date  ICMJE June 14, 2018
First Posted Date  ICMJE June 26, 2018
Last Update Posted Date March 14, 2019
Actual Study Start Date  ICMJE June 27, 2018
Estimated Primary Completion Date March 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 14, 2018)
Factor IX activity levels [ Time Frame: 26 weeks ]
Assessment of factor IX activity after a single dose of AMT-061
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03569891 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: June 14, 2018)
  • Annualized bleeding rate (ABR) [ Time Frame: 52 weeks ]
    Comparison of ABR between prophylaxis used in the lead-in and after administration of AMT-061
  • 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
  • Adverse events [ Time Frame: 5 years ]
    Follow up and assess any adverse events reported for safety
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Outcome Measures  ICMJE Not Provided
Original Other Outcome Measures  ICMJE Not Provided
 
Descriptive Information
Brief Title  ICMJE HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
Official Title  ICMJE 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
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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase Phase 3
Study Design  ICMJE 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
Condition  ICMJE Hemophilia B
Intervention  ICMJE Genetic: AAV5-hFIXco-Padua
Single intravenous infusion of AAV5-hFIXco-Padua (AMT-061)
Other Name: AMT-061
Study Arms 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.

Intervention: Genetic: AAV5-hFIXco-Padua
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 14, 2018)
56
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date August 2024
Estimated Primary Completion Date March 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender
Sexes Eligible for Study: Male
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts  ICMJE
Contact: Alison Long, MD 339-970-7578 a.long@uniqure.com
Contact: uniQure HOPE-B uniqureHOPE-B@uniqure.com
Listed Location Countries  ICMJE Belgium,   Denmark,   Netherlands,   Sweden,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03569891
Other Study ID Numbers  ICMJE CT-AMT-061-02
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: Undecided
Responsible Party UniQure Biopharma B.V.
Study Sponsor  ICMJE UniQure Biopharma B.V.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Steven Pipe, MD University of Michigan
PRS Account UniQure Biopharma B.V.
Verification Date March 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP