Haematological Disorders
Conditions
Interventions
FVIII prophylaxis
Sponsors
F. Hoffmann La Roche Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Diagnosis of severe (FVIII:C 66% in the last 3 years prior to screening Documented historical negative test for FVIII inhibitor (i.e., <0.6 BU/mL) within 12 months prior to enrollment Documentation of the details of prophylactic and episodic FVIII treatment and of the number and type of bleeding episodes for at least the last 6 months prior to screening Agreement to adhere to the contraception requirements (for potential participants with childbearing potential)
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 anti-retroviral therapy to treat HIV Planned surgery (excluding minor procedures such as non-molar tooth extraction, incision and drainage) during the study 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 ECG evidence or clinical history of prior myocardial infarction Refusal to accept plasma-derived and/or blood product transfusion support in an emergency scenario 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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Annualized Bleed Rate (ABR) for Treated Bleeds Over the Main Study Treatment Period | — |
Secondary
| Measure | Time frame |
|---|---|
| ABR for All Bleeds Over the Main Study Treatment Period;ABR for Treated Spontaneous Bleeds Over the Main Study Treatment Period;ABR for Treated Joint Bleeds Over the Main Study Treatment Period ;Adjusted Mean Treatment Burden Domain Score in Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire - Adult Version at Month 7;ABR for Treated Target Joint Bleeds Over the Main Study Treatment Period;Percentage of Participants with Zero Treated Bleeds Over the Main Study Treatment Period ;Number of Injections and Dose per Bleed of Coagulation Factors Administered to Treat a Bleed Over the Main Study Treatment Period;Annualized FVIII Injection Rate Over the Main Study Treatment Period;Annualized FVIII Consumption Rate Over the Main Study Treatment Period;Mean Treatment Burden Domain Score in CATCH Questionnaire - Adolescent Version at Month 7;Change From Baseline in Preoccupation Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions);Change From Baseline in Social Activity Impact Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions);Change From Baseline in Recreational Activity Impact Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions);Physical Impact Domain Score of the Treatment Administration Satisfaction Questionnaire (TASQ) at Specified Timepoints;Incidence and Severity of Adverse Events, With Severity Determined According To National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5.0 (NCI CTCAE V5.0) Grading Scale;Incidence and Severity of Thromboembolic Events and Thrombotic Microangiopathy;Incidence and Severity of Injection-Site Reactions;Incidence of Adverse Events Leading to Discontinuation of Assigned Study Treatment;Incidence of Severe Hypersensitivity, Anaphylaxis, or Anaphylactoid Reactions;Plasma Concentration of NXT007;Percentage of Participants With Anti-Drug Antibodies (ADAs) Against NXT007 at Baseline and During the Study;Percentage of Participants | — |
Countries
South Africa
Contacts
Public ContactJohnny Mahlangu
National PI
Outcome results
None listed