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

NCT03569891 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 67

Last updated 2026-03-27

Study results available
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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.

Conditions

  • Hemophilia B

Interventions

GENETIC

AAV5-hFIXco-Padua

Single intravenous infusion of AAV5-hFIXco-Padua (AMT-061)

BIOLOGICAL

Factor IX (FIX)

During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.

Sponsors & Collaborators

  • CSL Behring

    lead INDUSTRY

Principal Investigators

  • Steven Pipe, MD · University of Michigan

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-06-27
Primary Completion
2021-09-22
Completion
2025-03-19
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Denmark
  • Germany
  • Ireland
  • Netherlands
  • Sweden
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03569891 on ClinicalTrials.gov