Dose Confirmation Trial of AAV5-hFIXco-Padua

Status:	Active, not recruiting
Conditions:	Hematology
Therapuetic Areas:	Hematology
Healthy:	No
Age Range:	18 - Any
Updated:	12/7/2018
Start Date:	July 24, 2018
End Date:	August 2023

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

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.

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

We found this trial at

sites

University of California-Davis

1 Shields Ave
Sacramento, California 95616

(530) 752-1011