Mild or moderate hemophilia A MedDRA version: 20.0 Level: LLT Classification code 10053753 Term: Hemophilia A without inhibitors System Organ Class: 100000004850
Conditions
Sponsors
Roche Farma S. A. U. que realiza el ensayo en España y actúa como representante F. Hoffmann-La Roche Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Diagnosis of mild (FVIII level between > 5% and = 1% and = 3 kg - Need for prophylaxis based on investigator assessment - A negative test for inhibitor (i.e., =65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: -Inherited or acquired bleeding disorder other than mild (FVIII level between > 5% and = 1% and <= 5%) congenital hemophilia A - History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the investigator’s judgment - Previous (within the last 12 months) or current treatment for thromboembolic disease or signs of thromboembolic disease - Other conditions that may currently increase the risk of bleeding or thrombosis - History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection - Planned surgery during the emicizumab loading dose phase. Surgeries in patients on emicizumab from Week 5 onwards are allowed - Known HIV infection with CD4 counts < 200 cells/micro L - Concomitant disease, condition, significant abnormality on screening evaluation or laboratory tests, or treatment that could interfere with the conduct of the study, or that would in the opinion of the investigator, pose an additional unacceptable risk in administering study drug to the patient - Receipt of any of the following: o An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration with the exception of prior emicizumab prophylaxis o A non-hemophilia-related investigational drug within last 30 days or 5 half-lives, whichever is shorter o Any other investigational drug currently being administered or planned to be administered - Inability to comply with the study protocol in the opinion of the investigator - Pregnant or breastfeeding, or intending to become pregnant during the study - Women of childbearing potential must have a negative serum pregnancy test result within 7 days prior to initiation of study drug
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: There is no formal hypothesis testing in this study. Safety: To evaluate safety profile of emicizumab in patients with non-severe hemophilia A without inhibitors Primary Efficacy: To evaluate the efficacy of emicizumab on the basis of number of treated bleeds over time;Secondary Objective: Secondary Efficacy: 1.To evaluate the efficacy of emicizumab on basis of number of all bleeds, joint bleeds over time, target joint bleeds over time, spontaneous bleeds over time, joint health, health-related quality of life, preference for emicizumab compared with previous FVIII treatment and effect of emicizumab prophylaxis on physical activity compared with physical activity at baseline Pharmacokinetic: 2.To characterize emicizumab pharmacokinetic profile Immunogenicity: 3. To evaluate immune response to emicizumab;Primary end point(s): Safety: 1.Incidence and severity of adverse events 2.Incidence of thromboembolic events and thrombotic microangiopathy 3.Incidence of laboratory abnormalities, injection-site reactions, adverse events leading to drug discontinuation, severe hypersensitivity, anaphylaxis, and anaphylactoid events 4.Change from baseline in physical examination findings, vital signs and ECG parameters Primary Efficacy: 5.Number of treated bleeds over time;Timepoint(s) of evaluation of this end point: 1-3. From Screening (Day -28 to -1) to 24 weeks after final dose of emicizumab or Study completion/discontinuation 4. From baseline (Day [D]-28 to -1) to 24 weeks after final dose of emicizumab or Study completion/discontinuation | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary Efficacy: 1.Number of all bleeds (i.e., those treated and untreated with FVIII), joint bleeds, target joint bleeds and spontaneous bleeds over time 2.Joint health, as assessed through use of the Hemophilia Joint Health Score at specified timepoints 3.Health-related quality of life, as assessed through use of the Comprehensive Assessment Tool of Challenges in Hemophilia Questionnaire over time 4.Preference for emicizumab compared with previous FVIII regimen, as assessed through use of the Emicizumab Preference Survey 5.Effect of emicizumab prophylaxis treatment on physical activity compared with physical activity at baseline Pharmacokinetic: 6.Plasma concentration of emicizumab at specified timepoints Immunogenicity: 7.Prevalence of anti-drug antibodies (ADAs) at baseline and incidence of ADAs during the study 8.Number and proportion of patients who develop anti-FVIII inhibitors (titer>= 0.6 BU/mL) at specified timepoints;Timepoint(s) of evaluation of this end point: 1. At least 52 weeks 2. D-7 to -1, Week 25, 49, at every 24 weeks till Study completion/discontinuation 3. Week 1, 13, 25, 37 and 49; at every 12 weeks (Q12W) till Study completion/discontinuation 4. Week 17 5. Up to week 49 6. Week 1-5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45 and 49, Q12W, at safety follow up (FU), Study completion/discontinuation 7. Week 1, 5, 13, 25, 33, 41 and 49, Q12W, at safety FU, Study completion/discontinuation 8. D-28 to -1, Week 1, 13, 25, 37 and 49, Q12W, at safety FU, Study completion/discontinuation | — |
Countries
Belgium, Canada, France, Germany, Italy, Netherlands, Poland, South Africa, Spain, United Kingdom, United States
Contacts
Public ContactTrial Information Support Line-TISL
F. Hoffmann-La Roche Ltd
Outcome results
None listed