Ignite Creation Date:
2024-05-06 @ 3:28 PM
Last Modification Date:
2024-10-26 @ 1:50 PM
Study NCT ID:
NCT04647227
Status:
RECRUITING
Last Update Posted:
2024-06-26
First Post:
2020-11-23
Brief Title:
SEVENFACT for Bleeding Events in Hemophilia With Inhibitors
Sponsor:
American Thrombosis and Hemostasis Network
Organization:
American Thrombosis and Hemostasis Network
Study Overview
Official Title:
Safety of SEVENFACT for the Treatment of Bleeding Events in Patients With Hemophilia A or B With Inhibitors
Status:
RECRUITING
Status Verified Date:
2024-09
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:
Phase IV multi-center US-centric open-label safety study enrolling participants with Hemophilia A or B with inhibitors12 years of age and older who are either on long term prophylactic treatment eg emicizumab at risk of experiencing a breakthrough bleeding event BE or who are not on prophylactic treatment who may need to control a BE
Detailed Description:
Primary Objective
To evaluate the safety of SEVENFACT when used to treat bleeding episodes in participants with Hemophilia A or B with inhibitors either with or without prophylactic treatment
Study Design
Phase IV multi-center US-centric open-label safety study enrolling participants with Hemophilia A and B with inhibitors12 years of age and older who are either on long term prophylactic treatment eg emicizumab at risk of experiencing a breakthrough bleeding event BE or who are not on prophylactic treatment who may need to control a BE
Study Duration
Participants will be followed longitudinally from the time of enrollment to the end of their participation in the study The maximal study duration for any participant in the study will be up to 2 years from the time of enrollment
Target Accrual
This is a multi-site study in which it is anticipated approximately 28 to 55 participants will be enrolled in order to achieve treatment of approximately 100 bleeding events The study will target enrollment of a minimum of 23 participants on emicizumab prophylactic treatment and 5 participants on other treatments
Data Analysis
Sample Size Determination
Results from the Haven 1 study were used to calculate the annual bleeding rates ABR in individuals receiving prophylactic treatment These were calculated at an ABR of three for participants receiving emicizumab prophylactic treatment and 20 for participants receiving other treatments
Based on these data it was calculated that between 28 and 55 participants would be necessary to reach 100 BEs with a minimum of 23 participants on emicizumab prophylactic treatment and 5 participants on other treatments
Analysis Populations
The Safety Analysis Set is defined as all participants who received at least a single dose of SEVENFACT All analyses of safety will be performed based on the safety population and participants will be analyzed according to the dose of SEVENFACT that they actually received
Baseline Characteristics
Baseline characteristics will be summarized using descriptive statistics for continuous variables and frequencies and percentages for categorical variables
Safety Evaluations
All Adverse Events AEs will be graded for severity utilizing Common Terminology Criteria for Adverse Events CTCAE v50 and coded using Medical Dictionary of Regulatory Activities MedDRA version 23x The number and percentage of participants with treatment-emergent AEs TEAEs serious AEs SAEs serious TEAEs and treatment related TEAEs ie adverse drug reactions ADRs will be presented for all participants
The number of TEAEs as well as the number and percentage of participants with TEAEs serious TEAEs and treatment-related TEAEs will be presented by MedDRA System Organ Class SOC and preferred term for all participants
The number and percentage of participants with treatment-emergent adverse event andor allergic and anaphylactic reactions will be presented for all participants
Efficacy Evaluations
There are no pre-specified efficacy endpoints
To evaluate the safety of SEVENFACT when used to treat bleeding episodes in participants with Hemophilia A or B with inhibitors either with or without prophylactic treatment
Study Design
Phase IV multi-center US-centric open-label safety study enrolling participants with Hemophilia A and B with inhibitors12 years of age and older who are either on long term prophylactic treatment eg emicizumab at risk of experiencing a breakthrough bleeding event BE or who are not on prophylactic treatment who may need to control a BE
Study Duration
Participants will be followed longitudinally from the time of enrollment to the end of their participation in the study The maximal study duration for any participant in the study will be up to 2 years from the time of enrollment
Target Accrual
This is a multi-site study in which it is anticipated approximately 28 to 55 participants will be enrolled in order to achieve treatment of approximately 100 bleeding events The study will target enrollment of a minimum of 23 participants on emicizumab prophylactic treatment and 5 participants on other treatments
Data Analysis
Sample Size Determination
Results from the Haven 1 study were used to calculate the annual bleeding rates ABR in individuals receiving prophylactic treatment These were calculated at an ABR of three for participants receiving emicizumab prophylactic treatment and 20 for participants receiving other treatments
Based on these data it was calculated that between 28 and 55 participants would be necessary to reach 100 BEs with a minimum of 23 participants on emicizumab prophylactic treatment and 5 participants on other treatments
Analysis Populations
The Safety Analysis Set is defined as all participants who received at least a single dose of SEVENFACT All analyses of safety will be performed based on the safety population and participants will be analyzed according to the dose of SEVENFACT that they actually received
Baseline Characteristics
Baseline characteristics will be summarized using descriptive statistics for continuous variables and frequencies and percentages for categorical variables
Safety Evaluations
All Adverse Events AEs will be graded for severity utilizing Common Terminology Criteria for Adverse Events CTCAE v50 and coded using Medical Dictionary of Regulatory Activities MedDRA version 23x The number and percentage of participants with treatment-emergent AEs TEAEs serious AEs SAEs serious TEAEs and treatment related TEAEs ie adverse drug reactions ADRs will be presented for all participants
The number of TEAEs as well as the number and percentage of participants with TEAEs serious TEAEs and treatment-related TEAEs will be presented by MedDRA System Organ Class SOC and preferred term for all participants
The number and percentage of participants with treatment-emergent adverse event andor allergic and anaphylactic reactions will be presented for all participants
Efficacy Evaluations
There are no pre-specified efficacy endpoints
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