NCT03489291

Dose Confirmation Trial of AAV5-hFIXco-Padua

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: CSL Behring · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hemophilia B

Interventions

Type	Name	Description
GENETIC	AAV5-hFIXco-Padua (AMT-061)	Single intravenous infusion of AAV5-hFIXco-Padua (AMT-061)

Timeline

• Start date: 2018-07-24
• Primary completion: 2018-10-30
• Completion: 2023-09-21
• First posted: 2018-04-05
• Last updated: 2024-06-28
• Results posted: 2022-06-16

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study