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

Recruitment & Eligibility

Status: Active, not recruiting

Sex: Male

Target Recruitment: Not specified

Inclusion 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

Exclusion Criteria

Not provided

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Annualized Bleeding Rate (ABR) for All Bleeding Episodes

Secondary Outcome Measures

Name	Time	Method
Factor IX Activity Levels After AMT-061 Dosing;Annualized Exogenous Factor IX Consumption;Adjusted Annualized Infusion Rate of FIX Replacement Therapy;Percent of Subjects Who Discontinued FIX Prophylaxis and Remained Free of Routine FIX Prophylaxis After AMT-061 Dosing;Percentage of Subjects With Trough FIX Activity <12% of Normal;ABR for FIX-treated Bleeding Episodes;Number of Spontaneous Bleeding Episodes;Number of Joint Bleeding Episodes;Mean FIX Activity (%) in Subjects With Pre-Existing Neutralizing Antibodies to AAV5 After AMT-061 Dosing;Mean FIX Activity (%) in Subjects Without Pre-Existing Neutralizing Antibodies to AAV5 After AMT-061 Dosing;Number of New Target Joints and the Number of New Target Joints Resolved.;Percent of Participants With Zero Bleeding Episodes During the 52 Weeks Following Stable FIX Expression (6 to 18 Months) After AMT-061 Dosing;International Physical Activity Questionnaire (iPAQ) Overall Score;EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) VAS Overall Score;Number of Adverse Events

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

Recruitment & Eligibility

Study & Design

Related Trials

A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT-061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.

A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT-061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.

A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.

A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT-061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.

A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT-061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.

A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT-061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.

A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT-061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.

A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT-061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.

A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT-061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.

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