HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03569891|
Recruitment Status : Recruiting
First Posted : June 26, 2018
Last Update Posted : August 14, 2019
This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile.
The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). 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 pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10^13 gc/kg.
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia B||Genetic: AAV5-hFIXco-Padua||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||56 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||The reference therapy is prophylactic factor IX replacement therapy used during the lead-in phase prior to treatment with AAV5-hFIXco-Padua (AMT-061).|
|Masking:||None (Open Label)|
|Official Title:||Phase III, Open-label, Single-dose, Multi-center, Multinational Trial Investigating a 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 :||June 27, 2018|
|Estimated Primary Completion Date :||March 2020|
|Estimated Study Completion Date :||August 2024|
Single infusion of AMT-061
Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline. After study drug administration (post study drug), subjects will be monitored for tolerance to the study drug and detection of potential immediate AEs at the clinical trial site for a few hours after dosing.
Single intravenous infusion of AAV5-hFIXco-Padua (AMT-061)
Other Name: AMT-061
- Factor IX activity levels [ Time Frame: 26 weeks ]Assessment of factor IX activity after a single dose of AMT-061
- Annualized bleeding rate (ABR) [ Time Frame: 52 weeks ]Comparison of ABR between prophylaxis used in the lead-in and after administration of AMT-061
- Use of factor IX replacement therapy [ Time Frame: 52 weeks ]Patients will record all use of prophylactic factor IX replacement therapy in an e-diary, including reason for factor IX use, date, and time of infusion and total dose
- Adverse events [ Time Frame: 5 years ]Follow up and assess any adverse events reported for safety
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): NCT03569891
|Contact: Alison Long, MDemail@example.com|
|Contact: uniQure HOPE-B||uniqureHOPE-B@uniqure.com|
|Principal Investigator:||Steven Pipe, MD||University of Michigan|