Dose Confirmation Trial of AAV5-hFIXco-Padua
|ClinicalTrials.gov Identifier: NCT03489291|
Recruitment Status : Active, not recruiting
First Posted : April 5, 2018
Last Update Posted : December 5, 2018
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 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)|
|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|
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)
- 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 ]
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|