A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment (verITI-8 Study)
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|ClinicalTrials.gov Identifier: NCT03093480|
Recruitment Status : Recruiting
First Posted : March 28, 2017
Last Update Posted : December 18, 2018
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia A With Inhibitors||Biological: rFVIIIFc||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Non-controlled, Open-Label, Multicenter, Study of Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Subjects With Inhibitors Undergoing the First ITI Treatment|
|Actual Study Start Date :||June 30, 2017|
|Estimated Primary Completion Date :||November 2020|
|Estimated Study Completion Date :||November 2020|
Experimental: Recombinant coagulation factor VIII Fc (rFVIIIFc)
Participants will receive rFVIIIFc at a dose of 200 international units (IU)/kilogram (kg) as once daily injections or divided on several injections per day at the discretion of the Investigator, starting at baseline visit up to maximum of 48 Weeks in ITI Period. Participants who meet the criteria for immune tolerance induction (ITI) success will enter the tapering period and will receive rFVIIIFc at a dose adjusted according to Investigator judgment based on the FVIII activity levels and with the aim of tapering the rFVIIIFc dose to reach a prophylactic dosing regimen within 16 weeks (4 months). Follow-Up will be 32 weeks under an adjusted prophylactic regimen according to Investigator judgment.
rFVIIIFc 200 IU/kg/day in ITI Period, 50 or 100 IU/kg (adjusted according to Investigator judgement) in tapering Period, and prophylactic regimen in Follow-Up period as powder for injection administered intravenously.
Other Name: ELOCTATE/ELOCTA
- Time to Tolerization With rFVIIIFc [ Time Frame: Up to 48 Weeks ]Time required for participants to achieve ITI success where ITI success is defined as achieving all 3 of the following criteria: confirmed negative titers consisting of 2 consecutive negative inhibitor assessments within 2 weeks (less than [<] 0.6 Bethesda units/milliliter [mL] by the Nijmegen-modified Bethesda assay); incremental recovery (IR) greater than or equal to (>=) 66 percent (%) of the expected IR in 2 consecutive assessments; half-life (t½) >= 7 hours.
- Number of Participants With Immune Tolerance Induction (ITI) Success [ Time Frame: Up to 48 Weeks ]Number of participants who achieve ITI success where ITI success is defined as achieving all 3 of the following criteria: confirmed negative titers consisting of 2 consecutive negative inhibitor assessments within 2 weeks (<0.6 Bethesda units/mL by the Nijmegen-modified Bethesda assay); incremental recovery (IR) >= 66% of the expected IR at 2 consecutive assessments; half-life (t½) >=7 hours.
- Number of Participants Who Experience Relapse [ Time Frame: Approximately 48 Weeks ]Number of Participants with ITI success who reaches the criteria for relapse (defined as confirmed positive inhibitor titer >= 0.6 BU/mL or abnormal recovery after tolerance is achieved, and t½ less than [<] 7 hours) will be evaluated during the Tapering or Follow-Up Periods.
- Number of Bleeding Episodes During ITI, Tapering and Follow-Up periods [ Time Frame: Up to 2 Years ]A bleeding episode started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 72 hours apart were considered the same bleeding episode.
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) as a Measure of Safety and Tolerability [ Time Frame: Up to 2 Years ]An AE is any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; any other medically important event that, in the opinion of the Investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition.
- Number of Days Away From Work or School [ Time Frame: Up to 2 Years ]Number of days missed from school or work will be summarized descriptively.
- Number of Hospitalization Days [ Time Frame: Up to 2 Years ]Number of hospitalization days will be summarized descriptively.
- Adherence to Treatment Regimen [ Time Frame: Up to 2 Years ]Defined as percentage of administered doses versus planned doses.
- Consumption of rFVIIIFc [ Time Frame: Up to 2 Years ]Consumption will be assessed based on amount of administered study treatment.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03093480
|Contact: Bioverativ Therapeutics Inc, Waltham, MA, USAfirstname.lastname@example.org|
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