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

Brief Title

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

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

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.

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Annualized Bleeding Rate (ABR) for All Bleeding Episodes

Secondary Outcome

 Factor IX Activity Levels After AMT-061 Dosing

Condition

Hemophilia B

Intervention

AAV5-hFIXco-Padua

Study Arms / Comparison Groups

 AMT-061

Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Genetic

Estimated Enrollment

Start Date

June 27, 2018

Completion Date

March 2025

Primary Completion Date

September 22, 2021

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

Gender

Male

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Steven Pipe, MD, ,

Location Countries

Belgium

Location Countries

Belgium

Administrative Informations

NCT ID

NCT03569891

Organization ID

CSL222_3001 (CT-AMT-061-02)

Responsible Party

Sponsor

Study Sponsor

CSL Behring

Study Sponsor

Steven Pipe, MD, Principal Investigator, University of Michigan

Verification Date

March 2023