Ignite Creation Date:
2024-05-06 @ 10:15 AM
Last Modification Date:
2024-10-26 @ 12:27 PM
Study NCT ID:
NCT03204539
Status:
TERMINATED
Last Update Posted:
2020-05-29
First Post:
2017-06-13
Brief Title:
INdividualized ITI Based on FviiiATE Protection by VWF
Sponsor:
University of California Davis
Organization:
University of California Davis
Study Overview
Official Title:
INdividualized ITI Based on FviiiATE Protection by VWF INITIATE
Status:
TERMINATED
Status Verified Date:
2020-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Termination of funding from sponsor
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
INITIATE
Brief Summary:
The primary goal of the INITIATE trial is to compare the clinical outcome of individualized lot selection to random lot selection utilizing one plasma-derived von Willebrand factor VWFcoagulation factor FVIII complex concentrate for immune tolerance induction ITI in subjects with congenital Hemophilia A FVIII activity 2 and a historical high-titer inhibitor 5 Bethesda Unit BU
Detailed Description:
Participants will be randomized on a one-to-one basis between one of two study arms individualized lot selection alternative treatment arm and random lot selection standard treatment arm current US clinical practice in ITI Study sites participants and investigators will be blinded to the treatment status assigned
Alternative treatment arm
Half of the participants will be randomized to blinded individualized lot selection for ITI The target initial dose of FVIII for ITI is 200 IUkgday intravenously The suggested maximum dose is 20000 IUday Investigators may adjust the dose to a minimum dose of 150 unitskg if infusion volume is not feasible in patients without central venous access or in patients with von Willebrand factor levels 250 Splitting dose into two infusions per day must be approved by the Steering Committee and if approved will be considered a protocol deviation Wilate will be the VWFFVIII complex concentrate Octapharma USA Inc US License No 1646 prescribed for ITI
Individualized lot selection will be performed according to a modified Oxford method in a central laboratory by testing subjects plasma against 4-6 lots of Wilate and selecting the one with the highest residual FVIII lowest Oxford titer activity remaining after incubation The same lot will be used throughout the entire ITI course for each subject If the selected lot is depleted prior to the completion of ITI a second individualized lot selection will be performed using the original plasma sample provided at baseline
Each Wilate batch includes 16-18 million IU and is expected to last for about 3-57 months depending on the weight of the subject and prescribed dose
Standard treatment arm
The other half of the participants will receive random lot selection for ITI The dose and concentrate used will be the same Concentrate will be randomly selected from available Wilate lots The same lot will be used throughout the entire ITI course for each subject If the random lot is depleted prior to the completion of ITI a second lot will be randomly selected In both cases the random lot will be tested against subjects plasma to measure the residual FVIII activity left after incubation but this result will not affect lot selection
The primary hypothesis is that the time to negative inhibitor 06 BU will be shorter with individualized lot selection compared to random lot selection and that this will impact monthly break-through bleeding and reduce costs
Alternative treatment arm
Half of the participants will be randomized to blinded individualized lot selection for ITI The target initial dose of FVIII for ITI is 200 IUkgday intravenously The suggested maximum dose is 20000 IUday Investigators may adjust the dose to a minimum dose of 150 unitskg if infusion volume is not feasible in patients without central venous access or in patients with von Willebrand factor levels 250 Splitting dose into two infusions per day must be approved by the Steering Committee and if approved will be considered a protocol deviation Wilate will be the VWFFVIII complex concentrate Octapharma USA Inc US License No 1646 prescribed for ITI
Individualized lot selection will be performed according to a modified Oxford method in a central laboratory by testing subjects plasma against 4-6 lots of Wilate and selecting the one with the highest residual FVIII lowest Oxford titer activity remaining after incubation The same lot will be used throughout the entire ITI course for each subject If the selected lot is depleted prior to the completion of ITI a second individualized lot selection will be performed using the original plasma sample provided at baseline
Each Wilate batch includes 16-18 million IU and is expected to last for about 3-57 months depending on the weight of the subject and prescribed dose
Standard treatment arm
The other half of the participants will receive random lot selection for ITI The dose and concentrate used will be the same Concentrate will be randomly selected from available Wilate lots The same lot will be used throughout the entire ITI course for each subject If the random lot is depleted prior to the completion of ITI a second lot will be randomly selected In both cases the random lot will be tested against subjects plasma to measure the residual FVIII activity left after incubation but this result will not affect lot selection
The primary hypothesis is that the time to negative inhibitor 06 BU will be shorter with individualized lot selection compared to random lot selection and that this will impact monthly break-through bleeding and reduce costs
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