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: 20.0Level: LLTClassification code 10053751Term: Hemophilia A with anti factor VIIISystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2016-000073-21-IT
- Lead Sponsor
- F. HOFFMANN - LA ROCHE LTD.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- 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 the 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:
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
catheterassociated 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