Trials / Completed
CompletedNCT03569891
HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
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
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 67 (actual)
- Sponsor
- CSL Behring · Industry
- Sex
- Male
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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
Interventions
| Type | Name | Description |
|---|---|---|
| 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. |
Timeline
- Start date
- 2018-06-27
- Primary completion
- 2021-09-22
- Completion
- 2025-03-19
- First posted
- 2018-06-26
- Last updated
- 2026-03-27
- Results posted
- 2022-10-10
Locations
33 sites across 8 countries: United States, Belgium, Denmark, Germany, Ireland, Netherlands, Sweden, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03569891. Inclusion in this directory is not an endorsement.