Haematological Disorders
Conditions
Interventions
NXT007 Test Product
Emicizumab Comparator group
Sponsors
F. HoffmannLa Roche Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Diagnosis of severe (FVIII:C 5 IU/dL and <40 IU/dL) congenital hemophilia A with chronic FVIII inhibitors, defined as documented FVIII inhibitor ( =0.6 BU/mL or =1.0 BU/mL only for laboratories with a historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) and chronic reduction of endogenous baseline FVIII:C to <5 IU/dL for =12 months • Documented historical FVIII inhibitor assay results within the 12 months prior to enrollment • Documentation of the details of prophylactic and episodic FVIII treatment, bypassing agent (BPA) treatment, emicizumab prophylaxis treatment, and the number and type of bleeding episodes for at least the last 6 months prior to screening • For potential participants taking on-demand treatments prior to study entry: agreement to move to a prophylaxis treatment with either emicizumab or NXT007, according to assigned randomization
Exclusion criteria
Exclusion criteria: • Sensitivity to any of the study investigations, or components thereof, or drug or other allergy that, in the opinion of the investigator, contraindicates participation in the study • Use of systemic immunomodulators (e.g., interferon or rituximab) at the time of enrollment or planned use during the study, except for antiretroviral therapy to treat HIV • Refusal to accept plasma-derived and/or blood product transfusion support in an emergency scenario • Planned surgery (excluding minor procedures, such as non-molar tooth extraction or incision and drainage) during the study • History of ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease (e.g., severe left ventricular systolic dysfunction, left ventricular hypertrophy), coronary heart disease (symptomatic or with ischemia demonstrated by diagnostic testing) • History or presence of an abnormal ECG that is deemed clinically significant, (e.g., complete left bundle branch block, second- or third-degree atrioventricular heart block) or evidence or clinical history of prior myocardial infarction
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the efficacy of NXT007 prophylaxis compared with emicizumab prophylaxis based on the non inferiority assessment of treated bleeds | — |
Secondary
| Measure | Time frame |
|---|---|
| To evaluate the efficacy of NXT007 prophylaxis compared with emicizumab prophylaxis based on non inferiority and superiority assessment in all bleeds, treated joint bleed and treated spontaneous bleeds;To evaluate the efficacy of NXT007 prophylaxis compared with emicizumab prophylaxis based on non-inferiority and superiority assessment in all bleeds, treated joint bleed and treated spontaneous bleeds.;To evaluate the efficacy of NXT007 prophylaxis compared with emicizumab prophylaxis based on non-inferiority and superiority assessment in all bleeds, treated joint bleed and treated spontaneous bleeds;To evaluate the efficacy of NXT007 prophylaxis compared with emicizumab prophylaxis based on superiority assessment of health-related quality of life as assessed through use of the CATCH questionnaire (treatment burden domain in the adult version) ;To evaluate the efficacy of NXT007 prophylaxis compared with emicizumab prophylaxis for other secondary endpoints ;To evaluate the quality of life of participants treated with NXT007 prophylaxis compared with emicizumab prophylaxis as assessed through the use of the CATCH questionnaires (adult and adolescent versions) ;To evaluate participants' satisfaction with SC treatment administration as assessed through the use of the TASQ-SC;To evaluate the safety of NXT007 prophylaxis compared with emicizumab prophylaxis;To characterize the pharmacokinetics of NXT007 ;To evaluate the immunogenicity of NXT007 | — |
Countries
South Africa
Contacts
Public ContactMaphuti Ratau
Country Approval Specialist
Outcome results
None listed