Phase IIb, Open-label, Single-dose, Single-arm, Multi-center Trial to Confirm the Factor IX Activity Level of the 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_2001 (CT-AMT-061-01)

This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase. The IMP 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 IMP is identified as AAV5-hFIXco-Padua (AMT- 061). The pharmaceutical form of AMT-061 is a solution for intravenous infusion. The administered dose of AMT-061 will be 2 x 10\^13 gc/kg.

2018-07-24

2018-10-30

2023-09-21

Completed

Early phase I

3

Inclusion Criteria: 1. Male 2. Age ≥18 years 3. Subjects with congenital hemophilia B classified as severe or moderately severe 4. \>20 previous exposure days of treatment with FIX protein Exclusion Criteria: 1. History of FIX inhibitors 2. Positive FIX inhibitor test at screening 3. Select screening laboratory values \> 2 times upper normal limit: 4. Positive human immunodeficiency virus (HIV) 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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'open-label, single-dose, single-arm, multi-center trial', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 3, 'type': 'ACTUAL'}}

2023-11-18