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HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients | Clinical Trials | Clareo Health

COMPLETEDPHASE3

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

NCT03569891•CSL Behring

View on ClinicalTrials.gov

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.

Eligibility

Age

18 - No limit years

Sex

MALE

Healthy Volunteers

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

Locations (33)

Phoenix Children's Hospital

Phoenix, Arizona, United States

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Los Angeles Orthopedic Hospital

Los Angeles, California, United States

Children's Hospital of Los Angeles

Los Angeles, California, United States

University of California, Davis

Sacramento, California, United States

University of California, San Diego

San Diego, California, United States

University of Colorado Denver

Aurora, Colorado, United States

Children's National Medical Center Hematology and Oncology

Washington D.C., District of Columbia, United States

University of South Florida

Tampa, Florida, United States

University of Michigan

Ann Arbor, Michigan, United States

Key Dates

Start Date

June 27, 2018

Primary Completion Date

September 22, 2021

Completion Date

March 19, 2025

Last Updated

May 4, 2025

Enrollment

ACTUAL participants

Conditions

Hemophilia B

Interventions

AAV5-hFIXco-Padua

GENETIC

Factor IX (FIX)

BIOLOGICAL

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

NCT06379789

Hemophilia B

View study

RECRUITING

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

NCT06008938

Hemophilia B

View study

RECRUITING

National Longitudinal Cohort of Hematological Diseases

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: May 4, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

National Longitudinal Cohort of Hematological Diseases

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B

Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors

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

Inclusion Criteria

Exclusion Criteria

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

National Longitudinal Cohort of Hematological Diseases

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to &lt; 18 Years of Age) With Severe or Moderately Severe Hemophilia B

An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B

Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B