Dose Confirmation Trial of AAV5-hFIXco-Padua

CSL Behring

Status and phase: Completed; Phase 2

Conditions: Hemophilia B

Treatments: Genetic: AAV5-hFIXco-Padua (AMT-061)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03489291

CSL222_2001 (CT-AMT-061-01)

Details and patient eligibility

About

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.

Enrollment: 3 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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3 participants in 1 patient group

Single infusion of AMT-061

Experimental group

Description:

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).

Treatment:

Genetic: AAV5-hFIXco-Padua (AMT-061)