Dose Confirmation Trial of AAV5-hFIXco-Padua

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

3 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-07-24

Study Completion Date

2023-09-21

Brief Summary

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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.

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Detailed Description

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Conditions

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Hemophilia B

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

open-label, single-dose, single-arm, multi-center trial

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Single infusion of AMT-061

Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline. After IMP administration (post IMP), subjects will be monitored for tolerance to the IMP and detection of potential immediate AEs at the clinical trial site for 24 hours (overnight stay).

Group Type EXPERIMENTAL

AAV5-hFIXco-Padua (AMT-061)

Intervention Type GENETIC

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

Interventions

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AAV5-hFIXco-Padua (AMT-061)

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

Intervention Type GENETIC

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No