Brief Title
Dose Confirmation Trial of AAV5-hFIXco-Padua
Official Title
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
Brief 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.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
FIX activity levels
Secondary Outcome
Usage of FIX replacement therapy
Condition
Hemophilia B
Intervention
AAV5-hFIXco-Padua (AMT-061)
Study Arms / Comparison Groups
Single infusion of AMT-061
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).
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Genetic
Estimated Enrollment
3
Start Date
July 24, 2018
Completion Date
September 20, 2023
Primary Completion Date
October 30, 2018
Eligibility Criteria
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
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Steven Pipe, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT03489291
Organization ID
CT-AMT-061-01
Responsible Party
Sponsor
Study Sponsor
UniQure Biopharma B.V.
Study Sponsor
Steven Pipe, MD, Principal Investigator, University of Michigan
Verification Date
May 2020