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AAV5-hFIXco-Padua

GENETIC2 trials

Sponsors

CSL Behring

Conditions

Hemophilia B

Phase 3

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
CompletedNCT03569891
CSL BehringHemophilia B
Start: 2018-06-27End: 2025-03-19Updated: 2026-03-27

Unknown Phase

Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
Enrolling by invitationNCT05962398
CSL BehringHemophilia B
Start: 2023-08-30End: 2035-03-23Target: 56Updated: 2025-07-18

Related Papers

Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B.
2025-12-071 citations
End-of-study analysis of the HOPE-B trial confirms the durable efficacy and safety of etranacogene dezaparvovec hemophilia b gene therapy over 5 years
Blood2025-11-03
Elevation of liver health biomarkers before and after etranacogene dezaparvovec gene therapy in hemophilia B: Associations with long-term clinical outcomes
Blood2025-11-03
Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years
Blood Advances2025-04-069 citations
Development of an Agent-Based Model to Investigate Response Durability in Hemophilia B Patients Treated with the AAV-Based Coagulation Factor IX (FIX) Gene Therapy Etranacogene Dezaparvovec
Blood2024-11-05
Invasive procedures and surgery following etranacogene dezaparvovec gene therapy in people with hemophilia B.
2024-09-263 citations
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial.
2024-03-0132 citations
Etranacogene Dezaparvovec Shows Sustained Efficacy and Safety in Adult Patients With Severe or Moderately Severe Haemophilia B 3 Years After Administration in the HOPE-B Trial
Hämostaseologie2024-02-011 citations
HIV Comorbid Infection and Etranacogene Dezaparvovec Therapy: Efficacy and Safety Results From Phase 2b and Pivotal Phase 3 HOPE-B Trials 3 Years after Administration
Hämostaseologie2024-02-01
Long-Term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption and Safety of Hemophilia B Gene Therapy: Results from the HOPE-B Trial 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec in Adult Patients with Severe or Moderately Severe Hemophilia B
Blood2023-11-0213 citations
HIV Comorbid Infection and Liver-Directed AAV-Based Gene Therapy in Adults with Severe and Moderately Severe Hemophilia B: Efficacy and Safety Results from Phase 2b and the Pivotal Phase 3 HOPE-B Trials 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec
Blood2023-11-02
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B
New England Journal of Medicine2023-02-22303 citations
Adults with severe or moderately severe haemophilia B receiving etranacogene dezaparvovec in the HOPE-B phase 3 trial experience a stable increase in mean Factor IX activity levels and durable haemostatic protection after 24 months’ follow-up
Hämostaseologie2023-02-011 citations
Durability of bleeding protection and Factor IX activity in individuals with and without adeno-associated virus serotype 5 neutralising antibodies (Titres <1:700) in the phase 3 HOPE-B trial of etranacogene dezaparvovec gene therapy for haemophilia B
Hämostaseologie2023-02-01
Adults with Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection after 24 Months’ Follow-up
Blood2022-11-1511 citations
Durability of Bleeding Protection and Factor IX Activity Levels Are Demonstrated in Individuals with and without Adeno-Associated Virus Serotype 5 Neutralizing Antibodies (Titers <1:700) with Comparable Safety in the Phase 3 HOPE-B Clinical Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B
Blood2022-11-154 citations
Durability of Factor IX Activity and Bleeding Rate in People with Severe or Moderately Severe Hemophilia B after 5 Years of Follow-up in the Phase 1/2 Study of AMT-060, and after 3 Years of Follow-up in the Phase 2b and 2 Years of Follow-up in the Phase 3 Studies of Etranacogene Dezaparvovec (AMT-061)
Blood2022-11-152 citations
Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B.
2022-10-2522 citations
First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies
Blood2020-11-1916 citations
Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler–Najjar Syndrome
Human Gene Therapy2019-09-1060 citations