A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors (STASEY)
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|ClinicalTrials.gov Identifier: NCT03191799|
Recruitment Status : Active, not recruiting
First Posted : June 19, 2017
Last Update Posted : September 23, 2019
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia A||Drug: Emicizumab||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||195 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Single-Arm, Multicenter Phase IIIB Clinical Trial to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors|
|Actual Study Start Date :||September 5, 2017|
|Estimated Primary Completion Date :||September 4, 2020|
|Estimated Study Completion Date :||September 4, 2020|
Participants will receive initial weekly doses of prophylactic emicizumab subcutaneously for 4 weeks, followed by maintenance doses consisting of half the initial dose, administered subcutaneously for the remainder of the 2-year treatment period
Initial dosing will be 3 mg/kg/week subcutaneously for 4 weeks; Maintenance dosing will follow at 1.5 mg/kg/week subcutaneously for the remainder of the 2-year treatment period
Other Name: RO5534262
- Incidence and severity of all adverse events (AEs) including thromboembolic events, microangiopathic hemolytic anemia or TMA (e.g. hemolytic uremic syndrome), systemic hypersensitivity, anaphylaxis, and anaphylactoid events [ Time Frame: Up to approximately 2 years ]Incidence and severity of AEs will be monitored throughout the study to assess the safety and tolerability of emcizumab.
- Numbers of bleeds over time [ Time Frame: Up to approximately 2 years ]To evaluate the efficacy of prophylactic administration of emicizumab, the number of bleeds over time will be recorded for all of the enrolled participants. The final analysis will be conducted when all participants have completed 2 years of treatment or have withdrawn, whichever occurs sooner. Participants, or their legally authorized representative, will be asked to report bleed information, including site and type of bleed, time of each individual bleed (day, start and stop time), and treatment for bleed.
- Hemophilia Adult Quality of Life Questionnaire (Haem-A-QoL) (>= 18 y) [ Time Frame: Up to approximately 2 years ]The Haem-A-QoL (>= 18 y) was designed for adult participants with hemophilia. It comprises 10 dimensions (physical health, feelings, view of yourself, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, and partnerships and sexuality).
- Hemophilia Quality of Life Short Form (Haemo-QoL-SF) (12-17 y) [ Time Frame: Up to approximately 2 years ]The Haemo-QoL-SF (12-17 y) was designed as a series of age-related questionnaires to measure HRQoL in children and adolescents with hemophilia. This version covers nine dimensions considered relevant for children's HRQoL (physical health, feelings, view of yourself, family, friends, other people, sports, dealing with hemophilia, and treatment).
- EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) [ Time Frame: Up to approximately 2 years ]The EQ-5D-5L is a generic, self-reported, preference-based health utility measure that consists of six questions and is used to assess health status and inform pharmacoeconomic evaluations.
- Participant preference for the emicizumab regimen compared with the previous regimen, as measured by the EmiPref questionnaire [ Time Frame: Month 3 ]The EmiPref questionnaire asks participants to specify the treatment they would prefer to continue to receive after receiving treatment with their previous episodic or prophylactic regimen and subcutaneous (SC) emicizumab.
- Incidence and clinical significance of anti-emicizumab antibodies [ Time Frame: Up to approximately 2 years ]Immunogenicity will be monitored by incidence and clinical significance of antibodies to emicizumab. For the assessment of anti-FVIII antibodies, functional assays for FVIII inhibitors that utilize a clotting readout (classic Bethesda or Nijmegen assay) cannot be used for participants on emicizumab therapy as emicizumab drives clotting even in the presence of FVIII inhibitors, causing a false-negative test result. After the first dose, local measurement of FVIII inhibitors, if indicated, requires use of an enzyme linked immunosorbent assay- (ELISA-) based test or a chromogenic Bethesda assay. At the discretion of the local investigator, any additional urgent request to assess FVIII inhibitors will need to be sent to a central laboratory.
- Ctrough of emicizumab [ Time Frame: Week 1, Week 2, Week 3, and Week 5, Month 3, Month 6, Month 12, Month 18, Month 24, at safety follow-up (up to approximately 2 years) ]Ctrough is a measure of plasma concentration of a study drug at the end of of the dosage interval.
- Clearance of emicizumab [ Time Frame: Week 1, Week 2, Week 3, and Week 5, Month 3, Month 6, Month 12, Month 18, Month 24, at safety follow-up (up to approximately 2 years) ]Clearance is the rate at which a study drug is removed from the body.
- Volume of distribution of emicizumab [ Time Frame: Week 1, Week 2, Week 3, and Week 5, Month 3, Month 6, Month 12, Month 18, Month 24, at safety follow-up (up to approximately 2 years) ]Volume of distribution is the theoretical volume that would be necessary to contain the total amount of an administered drug at the same concentration that it is observed in the blood plasma.
- Area under the plasma drug concentration-time curve (AUC) of emicizumab [ Time Frame: Week 1, Week 2, Week 3, and Week 5, Month 3, Month 6, Month 12, Month 18, Month 24, at safety follow-up (up to approximately 2 years) ]AUC reflects the actual body exposure of a study drug after dosing.
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): NCT03191799
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|Study Director:||Clinical Trials||Hoffmann-La Roche|