HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
Public ClinicalTrials.gov record NCT03569891. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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 identification
- NCT ID
- NCT03569891
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 67 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 27, 2018 (Actual)
- Primary completion
- Sep 22, 2021 (Actual)
- Completion
- Mar 19, 2025 (Actual)
- Last update posted
- Mar 27, 2026
2018 – 2025
United States locations
- U.S. sites
- 19
- U.S. states
- 14
- U.S. cities
- 17
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Phoenix Children's Hospital | Phoenix | Arizona | 85016 | — |
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | — |
| Los Angeles Orthopedic Hospital | Los Angeles | California | 90007 | — |
| Children's Hospital of Los Angeles | Los Angeles | California | 90027 | — |
| University of California, Davis | Sacramento | California | 95817 | — |
| University of California, San Diego | San Diego | California | 92161 | — |
| University of Colorado Denver | Aurora | Colorado | 80045 | — |
| Children's National Medical Center Hematology and Oncology | Washington D.C. | District of Columbia | 20010 | — |
| University of South Florida | Tampa | Florida | 33612 | — |
| University of Michigan | Ann Arbor | Michigan | 48109 | — |
| Hemophilia Center of Western New York | Buffalo | New York | 14209 | — |
| University of North Carolina, Chapel Hill | Chapel Hill | North Carolina | 27599 | — |
| Oregon Health & Science University | Portland | Oregon | 97239 | — |
| University of Tennessee Health Science Center | Memphis | Tennessee | 38163 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | — |
| University of Texas Health Science Center & Medical School | Houston | Texas | 77030 | — |
| University of Utah | Salt Lake City | Utah | 84108 | — |
| Washington Institute for Coagulation | Seattle | Washington | 98101 | — |
| Bloodworks Northwest | Seattle | Washington | 98104 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 14 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03569891, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 27, 2026 · Synced Oct 27, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03569891 live on ClinicalTrials.gov.