Phase 2
Completed N=3
Dose Confirmation Trial of AAV5-hFIXco-Padua
Source: ClinicalTrials.gov NCT03489291 ↗Enrolled (actual)
3
Serious AEs
33.3%
Results posted
Jun 2022
Primary outcomePrimary: Factor IX Activity Levels — 30.6 Factor IX activity (%)
Summary
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Factor IX Activity Levels |
45.67 | — |
| SECONDARY Annualized Exogenous Factor IX Usage |
7.1 | — |
| SECONDARY Annualized Bleeding Rate (ABR) |
0.14; 0.07; 0.07; 0.00 | — |
| SECONDARY Factor IX Activity Levels |
45.67 | — |
| SECONDARY Number of Participants Remaining Free of Continuous Prophylaxis |
3 | — |
| SECONDARY Annualized Exogenous Factor IX Usage Post-Continuous Prophylaxis |
342.1 | — |
| SECONDARY Number of Participants With Treatment Emergent (TE): Adverse Events (AE), Mild, Moderate, and Severe AEs, AEs Related and Unrelated to the Study Treatment, and Serious AEs |
3; 3; 2; 2; 1; 3 | — |
| SECONDARY Number of Participants With Clinically Meaningful Findings in Hematology and Serum Chemistry Parameters |
— | — |
| SECONDARY Number of Participants With Newly Occurring or Worsening Potentially Clinically Significant Changes in Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST) Levels |
1; 1 | — |
| SECONDARY Number of Participants Receiving Corticosteroids for AST and ALT Elevations |
— | — |
| SECONDARY Number of Participants Positive With AAV5 and Factor IX Neutralising Antibodies in Serum |
3; 3; 0; 0 | — |
| SECONDARY Number of Participants With AAV5 Capsid-specific T Cell Response |
1 | — |
| SECONDARY Number of Participants With Inflammatory Marker Levels Outside Normal Ranges |
1; 1; 1; 1 | — |
| SECONDARY Time to First Negative Results for Vector Deoxyribonucleic Acid (DNA) From Semen and Blood |
26.21; 78.29 | — |
| SECONDARY Number of Participants With Abnormal Values in Alpha-fetoprotein (AFP) Levels |
— | — |
| SECONDARY Number of Participants With Abnormal Results in Abdominal Ultrasound |
3; 2; 3; 2; 3 | — |
Eligibility Criteria
Inclusion Criteria
- Male
- Age ≥18 years
- Subjects with congenital hemophilia B classified as severe or moderately severe
- >20 previous exposure days of treatment with FIX protein
Exclusion Criteria
- History of FIX inhibitors
- Positive FIX inhibitor test at screening
- Select screening laboratory values > 2 times upper normal limit:
- Positive human immunodeficiency virus (HIV) at screening, not controlled with anti-viral therapy
- Active infection with Hepatitis B or C virus at screening
- History of Hepatitis B or C exposure, currently controlled by antiviral therapy
Data sourced from ClinicalTrials.gov (NCT03489291). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.