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Dose Confirmation Trial of AAV5-hFIXco-Padua

NCT03489291 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2024-06-28

Study results available
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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

GENETIC

AAV5-hFIXco-Padua (AMT-061)

Single intravenous infusion of AAV5-hFIXco-Padua (AMT-061)

Sponsors & Collaborators

  • CSL Behring

    lead INDUSTRY

Principal Investigators

  • Steven Pipe, MD · University of Michigan

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-07-24
Primary Completion
2018-10-30
Completion
2023-09-21
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03489291 on ClinicalTrials.gov