Ignite Creation Date:
2024-05-06 @ 11:38 AM
Last Modification Date:
2024-10-26 @ 12:48 PM
Study NCT ID:
NCT03569891
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-06-28
First Post:
2018-06-14
Brief Title:
HOPE-B Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
Sponsor:
CSL Behring
Organization:
CSL Behring
Study Overview
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
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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 1013 gckg
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 1013 gckg
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None