Dose Confirmation Trial of AAV5-hFIXco-Padua

Study Description

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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.

Condition or disease	Intervention/treatment	Phase
Hemophilia B	Genetic: AAV5-hFIXco-Padua (AMT-061)	Phase 2

Study Design

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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

Arms and Interventions

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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).	Genetic: AAV5-hFIXco-Padua (AMT-061); Single intravenous infusion of AAV5-hFIXco-Padua (AMT-061)

Outcome Measures

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Primary Outcome Measures :

1. 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.

Secondary Outcome Measures :

1. 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.
2. Annualized Bleeding Rate [ Time Frame: 52 weeks post-dose ]
3. Adverse Events [ Time Frame: 5 years post dose ]

Eligibility Criteria

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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

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

Contacts and Locations

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Locations

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

Sponsors and Collaborators

UniQure Biopharma B.V.

Investigators

Principal Investigator:	Steven Pipe, MD	University of Michigan

More Information

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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
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