Trial Profile
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:
Active, no longer recruiting
Phase of Trial:
Phase III
Latest Information Update: 08 Apr 2024
Price :
$35
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At a glance
- Drugs Etranacogene dezaparvovec (Primary)
- Indications Haemophilia B
- Focus Registrational; Therapeutic Use
- Acronyms HOPE-B
- Sponsors CSL Behring; uniQure
- 15 Jan 2024 According to a CSL media release, based on results from this ongoing Phase III HOPE-B trial, Swissmedic has authorised HEMGENIX (etranacogene dezaparvovec; first and currently only gene therapy for the treatment of male adults aged >18 years with severe or moderately severe hemophilia B) in Switzerland.
- 01 Jan 2024 Results from HOPE-B, PROLONG-9FP, B-LONG and Paradigm 2, published in the Haemophilia
- 12 Dec 2023 Results reporting long-term efficacy and safety data over a period of 3-years of post-treatment presented at the 65th American Society of Hematology Annual Meeting and Exposition