Ignite Creation Date:
2024-05-06 @ 12:51 PM
Last Modification Date:
2024-10-26 @ 1:05 PM
Study NCT ID:
NCT03861273
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-05-03
First Post:
2019-03-01
Brief Title:
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Sponsor:
Pfizer
Organization:
Pfizer
Study Overview
Official Title:
Phase 3 Open-label Single-arm Study to Evaluate Efficacy and Safety of FIX Gene Transfer With PF-06838435 rAAV-Spark100-hFIX-R338L in Adult Male Participants With Moderately Severe to Severe Hemophilia B FIXC 2 BeneGene-2
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:
BENEGENE-2
Brief Summary:
This study will evaluate the efficacy and safety of PF-06838435 a gene therapy drug in adult male participants with moderately severe to severe hemophilia B participants that have a Factor IX circulating activity of 2 or less The gene therapy is designed to introduce genetic material into cells to compensate for missing or non-functioning Factor IX Eligible study participants will have completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead in study C0371004 Participants will be dosed once intravenously and will be evaluated over the course of 6 years The main objective of the study will evaluate the annualized bleeding rate ABR for participants treated with gene therapy versus standard of care SOC therapy FIX prophylaxis replacement regimen
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?:
True
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2018-003086-33 | EUDRACT_NUMBER | None | None |
| 2022-502844-11-00 | OTHER | EU CT | None |