A Phase III Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Emicizumab in Hemophilia A Pediatric Patients With Inhibitors
- Conditions
- Hemophilia A with InhibitorsMedDRA version: 20.0 Level: LLT Classification code 10053751 Term: Hemophilia A with anti factor VIII System Organ Class: 100000011928Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2016-000073-21-GB
- Lead Sponsor
- F. Hoffmann-La Roche Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- Not specified
- Target Recruitment
- 88
- Children < 12 years of age at time of informed consent with allowance for the following: for patients 12 17 years of age and who weigh < 40 kilograms (kg) at the time of informed consent(Cohort A only); and for patients < 2 years of age will be allowed to participate only after the protocol defined interim data review criteria are met (Cohort A only)
- Body weight > 3 kg at time of informed consent
- Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures including the completion of applicable patient-reported outcome (PRO) questionnaires
- Caregivers must have the willingness and ability to comply with all study procedures including the completion of the bleed/medication questionnaire and applicable HRQoL questionnaires
- Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (i.e., >=5 BU)
- Requires treatment with bypassing agents
- For patients >= 2 years of age (Cohort A only): if on episodic bypassing agent regimen annualized bleeding rate (ABR) of >=6 ; or if on prophylactic bypassing agent regimen inadequately controlled or central venous access device (CVAD) placement medically not feasible or deemed unsafe
- For patients < 2 years of age determined by investigator to be in high unmet medical need
- Adequate hematologic, hepatic and renalfunctions
- Female patients of childbearing potential who have negative serum pregnancy test result and an agreement to remain abstinent or use contraceptive methods specified in the study
Are the trial subjects under 18? yes
Number of subjects for this age range: 80
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Inherited or acquired bleeding disorder other than hemophilia A
- Ongoing (or plan to receive during the study) immune tolerance induction (ITI) therapy or prophylaxis treatment with FVIII. Patients awaiting initiation of ITI will be eligible and patients in whom ITI has failed will be eligible with a 72-hour washout period prior to the first emicizumab administration
- Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
- Other diseases (i.e., certain autoimmune diseases [e.g., systemic lupus erythematosus], cardiovascular disease) that may increase risk of bleeding or thrombosis
- Known infection with human immunodeficiency virus, hepatitis B virus, hepatitis C virus
- Patients who are at high risk for TMA (e.g., have a previous medical or family history of TMA), in the investigator’s judgment
- Use of systemic immunomodulators (e.g., interferon or corticosteroids) at enrollment or planned use during the study period
- Planned surgery (excluding minor procedures such as tooth extraction or incision and drainage) during the study
- Inability (or unwillingness by caregiver) to receive (allow receipt of) blood or blood products (or any standard-of-care treatment for a life-threatening condition)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method