HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
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|ClinicalTrials.gov Identifier: NCT03569891|
Recruitment Status : Active, not recruiting
First Posted : June 26, 2018
Last Update Posted : October 15, 2021
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|
|Actual Primary Completion Date :||September 22, 2021|
|Estimated Study Completion Date :||March 2025|
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
- Annualized bleeding rate (ABR) [ Time Frame: 52 weeks following stable factor IX expression (months 6-18 post-treatment) ]Comparison of ABR between prophylaxis used in the lead-in and after administration of AMT-061
- Factor IX activity levels [ Time Frame: 18 Months ]Endogenous factor IX activity at 26 weeks, 52 weeks and 18 months after AMT-061 dosing
- Use of factor IX replacement therapy [ Time Frame: 52 weeks following stable factor IX expression (months 6-18 post-treatment) ]Patients will record all use of prophylactic factor IX replacement therapy in a 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
|Principal Investigator:||Steven Pipe, MD||University of Michigan|