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

CSL222_3001 (CT-AMT-061-02)

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.

2018-06-27

2021-09-22

2025-03-01

Active (not recruiting)

Early phase I

67

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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'The reference therapy is prophylactic factor IX replacement therapy used during the lead-in phase prior to treatment with AAV5-hFIXco-Padua (AMT-061).', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 67, 'type': 'ACTUAL'}}

2024-04-03