Ignite Creation Date:
2024-05-06 @ 10:52 AM
Last Modification Date:
2024-10-26 @ 12:36 PM
Study NCT ID:
NCT03370172
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-07-08
First Post:
2017-11-22
Brief Title:
A Study of BAX 888 in Male Adults With Severe Hemophilia A
Sponsor:
Baxalta now part of Shire
Organization:
Takeda
Study Overview
Official Title:
A Global Open-Label Multicenter Phase 12 Study of the Safety and Dose Escalation of BAX 888 an Adeno-Associated Virus Serotype 8 AAV8 Vector Expressing B-Domain Deleted Factor VIII BDD-FVIII in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-07
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:
The main aim of this study is to check if there are side effects from BAX 888 and to determine the dose of BAX 888 for treating severe hemophilia A in male adults
Participants will receive one infusion with BAX 888 at the hemophilia treatment center During the study participants will visit their study clinic multiple times
Participants will receive one infusion with BAX 888 at the hemophilia treatment center During the study participants will visit their study clinic multiple times
Detailed Description:
This study consists of 3 cohorts Participants will be assigned to 1 of 3 dose cohorts with a minimum of 24 hours between dosing of each participant Initially 2 participants will be dosed in a cohort with up to a total of 5 participants if the cohort is expanded based on safety and activity levels data
Dose escalation After dosing first 2 participants in cohort 1 the decision will be made on the following If week 4 FVIII activity levels of both participants are less than 2 then dose escalation to cohort 2 will be triggered with no further dosing in cohort 1 If FVIII activity levels 2 are observed in at least 1 participant among the 2 participants the decision to escalate dose or expand the cohort with dosing of additional participants will be based on all available data through Week 14
Dose expansion After dose escalation and administration of BAX 888 to the first 2 participants in 3 cohorts If sustained Week 14 FVIII activity levels are 30 are not achieved in both participants first 2 participants in cohorts 1 and 2 then escalation to immediate next cohort will be triggered after Data Monitoring Committee DMC review of all available safety and FVIII activity levels data For cohort 3 dosing of additional participants will be paused until further review of available data If sustained Week 14 FVIII levels are 30 in at least 1 of the 2 participants first 2 participant in cohort 1 2 3 then expansion of cohorts 1 2 with up to 5 participants 3 with up to 3 additional participants will be initiated with dosing or study could be completed with no further dosing
23 APRIL 2020 Enrollment of new patients into this study has been paused due to the COVID-19 situation The duration of this pause is dependent on the leveling and control of the COVID-19 pandemic
Dose escalation After dosing first 2 participants in cohort 1 the decision will be made on the following If week 4 FVIII activity levels of both participants are less than 2 then dose escalation to cohort 2 will be triggered with no further dosing in cohort 1 If FVIII activity levels 2 are observed in at least 1 participant among the 2 participants the decision to escalate dose or expand the cohort with dosing of additional participants will be based on all available data through Week 14
Dose expansion After dose escalation and administration of BAX 888 to the first 2 participants in 3 cohorts If sustained Week 14 FVIII activity levels are 30 are not achieved in both participants first 2 participants in cohorts 1 and 2 then escalation to immediate next cohort will be triggered after Data Monitoring Committee DMC review of all available safety and FVIII activity levels data For cohort 3 dosing of additional participants will be paused until further review of available data If sustained Week 14 FVIII levels are 30 in at least 1 of the 2 participants first 2 participant in cohort 1 2 3 then expansion of cohorts 1 2 with up to 5 participants 3 with up to 3 additional participants will be initiated with dosing or study could be completed with no further dosing
23 APRIL 2020 Enrollment of new patients into this study has been paused due to the COVID-19 situation The duration of this pause is dependent on the leveling and control of the COVID-19 pandemic
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2015-005576-22 | EUDRACT_NUMBER | None | None |