Dose Confirmation Trial of AAV5-hFIXco-Padua
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ClinicalTrials.gov Identifier: NCT03489291 |
Recruitment Status :
Active, not recruiting
First Posted : April 5, 2018
Last Update Posted : December 5, 2018
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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.
Condition or disease | Intervention/treatment | Phase |
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Hemophilia B | Genetic: AAV5-hFIXco-Padua (AMT-061) | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 3 participants |
Intervention Model: | Single Group Assignment |
Intervention Model Description: | open-label, single-dose, single-arm, multi-center trial |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | July 24, 2018 |
Actual Primary Completion Date : | October 30, 2018 |
Estimated Study Completion Date : | August 2023 |

Arm | Intervention/treatment |
---|---|
Experimental: 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).
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Genetic: AAV5-hFIXco-Padua (AMT-061)
Single intravenous infusion of AAV5-hFIXco-Padua (AMT-061) |
- FIX activity levels [ Time Frame: 6 weeks ]To confirm that a single dose of 2x10^13 gc/kg AMT-061 will result in FIX activity levels of ≥5% at 6 weeks after dosing.
- Usage of FIX replacement therapy [ Time Frame: 52 weeks post-dose ]Patients will record all use of prophylactic and on-demand FIX replacement therapy in an e-diary, including reason for FIX use, date and time of infusion and total dose.
- Annualized Bleeding Rate [ Time Frame: 52 weeks post-dose ]
- Adverse Events [ Time Frame: 5 years post dose ]

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | Male |
Gender Based Eligibility: | Yes |
Gender Eligibility Description: | Hemophilia B is an X-linked, recessive condition, since it occurs almost exclusively in males. Females typically are asymptomatic carriers. Therefore eligibility is restricted to male participants. |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male
- Age ≥18 years
- Subjects with congenital hemophilia B classified as severe or moderately severe
- >20 previous exposure days of treatment with FIX protein
Exclusion Criteria:
- History of FIX inhibitors
- Positive FIX inhibitor test at screening
- Select screening laboratory values > 2 times upper normal limit:
- Positive human immunodeficiency virus (HIV) at screening, not controlled with anti-viral therapy
- Active infection with Hepatitis B or C virus at screening
- History of Hepatitis B or C exposure, currently controlled by antiviral therapy

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03489291
United States, California | |
Los Angeles Orthopedic Hospital | |
Los Angeles, California, United States, 90007 | |
University of California, Davis | |
Sacramento, California, United States, 95817 | |
University of California, San Diego | |
San Diego, California, United States, 92122 | |
United States, Michigan | |
University of Michigan | |
Ann Arbor, Michigan, United States, 48109 |
Principal Investigator: | Steven Pipe, MD | University of Michigan |
Responsible Party: | UniQure Biopharma B.V. |
ClinicalTrials.gov Identifier: | NCT03489291 History of Changes |
Other Study ID Numbers: |
CT-AMT-061-01 |
First Posted: | April 5, 2018 Key Record Dates |
Last Update Posted: | December 5, 2018 |
Last Verified: | December 2018 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Studies a U.S. FDA-regulated Drug Product: | Yes | |
Studies a U.S. FDA-regulated Device Product: | No | |
Product Manufactured in and Exported from the U.S.: | No |
Keywords provided by UniQure Biopharma B.V.:
Hemophilia, Gene Therapy Bleeding Factor IX |
FIX viral vector Padua |
Additional relevant MeSH terms:
Hemophilia A Hemophilia B Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn Genetic Diseases, X-Linked |