Hemophilia A
Conditions
Brief summary
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of NXT007 prophylaxis compared with Factor VIII (FVIII) prophylaxis in participants with severe or moderate congenital hemophilia A without inhibitors. The study will include people aged ≥12 years old with severe or moderate congenital hemophilia A without inhibitors on previous FVIII prophylaxis treatment.
Interventions
COMBINATION_PRODUCTNXT007
NXT007 will be administered subcutaneously (SC) using an integrated drug-device combination product.
DRUGHuman Coagulation Factor VIII
Factor VIII (FVIII) prophylaxis standard of care (SOC) will be administered at the dose and frequency as stated in the local labels and per local country practice.
Sponsors
Hoffmann-La Roche
Chugai Pharmaceutical
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Diagnosis of severe (FVIII:C \<1 IU/dL \[International Unit per decilitre\]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A without inhibitors against FVIII * No documented inhibitor (i.e., \<0.6 BU/mL \[Bethesda unit per millilitre\]), FVIII half-life ≥6 hours, or FVIII recovery \>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
* 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 | 6 months |
Secondary
| Measure | Time frame |
|---|---|
| ABR for All Bleeds Over the Main Study Treatment Period | 6 months |
| ABR for Treated Spontaneous Bleeds Over the Main Study Treatment Period | 6 months |
| ABR for Treated Joint Bleeds Over the Main Study Treatment Period | 6 months |
| Adjusted Mean Treatment Burden Domain Score in Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire - Adult Version at Month 7 | Month 7 |
| ABR for Treated Target Joint Bleeds Over the Main Study Treatment Period | 6 months |
| Percentage of Participants with Zero Treated Bleeds Over the Main Study Treatment Period | 6 months |
| Number of Injections and Dose per Bleed of Coagulation Factors Administered to Treat a Bleed Over the Main Study Treatment Period | 6 months |
| Annualized FVIII Injection Rate Over the Main Study Treatment Period | 6 months |
| Annualized FVIII Consumption Rate Over the Main Study Treatment Period | 6 months |
| Mean Treatment Burden Domain Score in CATCH Questionnaire - Adolescent Version at Month 7 | Month 7 |
| Change From Baseline in Preoccupation Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions) | At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years) |
| Change From Baseline in Social Activity Impact Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions) | At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years) |
| Change From Baseline in Recreational Activity Impact Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions) | At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years) |
| Physical Impact Domain Score of the Treatment Administration Satisfaction Questionnaire (TASQ) at Specified Timepoints | At prespecified timepoints from Baseline to Month 10 |
| 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 | From Baseline until Study Completion (approximately 3.5 years) |
| Incidence and Severity of Thromboembolic Events and Thrombotic Microangiopathy | From Baseline until Study Completion (approximately 3.5 years) |
| Incidence and Severity of Injection-Site Reactions | From Baseline until Study Completion (approximately 3.5 years) |
| Incidence of Adverse Events Leading to Discontinuation of Assigned Study Treatment | From Baseline until Study Completion (approximately 3.5 years) |
| Incidence of Severe Hypersensitivity, Anaphylaxis, or Anaphylactoid Reactions | From Baseline until Study Completion (approximately 3.5 years) |
| Plasma Concentration of NXT007 | At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years) |
| Percentage of Participants With Anti-Drug Antibodies (ADAs) Against NXT007 at Baseline and During the Study | At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years) |
| Percentage of Participants With Neutralizing ADAs Against NXT007 | At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years) |
Countries
Japan
Contacts
CONTACTReference Study ID Number: WO45886 https://forpatients.roche.com/ No attachments to email below.
CONTACTFastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry
STUDY_DIRECTORClinical Trials
Hoffmann-La Roche
Outcome results
None listed