A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors
Status: | Active, not recruiting |
---|---|
Conditions: | Anemia, Hematology |
Therapuetic Areas: | Hematology |
Healthy: | No |
Age Range: | Any - 17 |
Updated: | 2/10/2019 |
Start Date: | July 22, 2016 |
End Date: | February 5, 2021 |
A Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Subcutaneous Administration of Emicizumab in Hemophilia A Pediatric Patients With Inhibitors
This non-randomized, multicenter, open-label, Phase III clinical study will evaluate the
efficacy, safety, and pharmacokinetics of emicizumab administered subcutaneously initially
once weekly (QW) in pediatric participants with hemophilia A with FVIII inhibitors. This
study will open two additional non-randomized cohorts to investigate once every 2 weeks (Q2W)
and once every 4 weeks (Q4W) regimens in pediatric participants.
efficacy, safety, and pharmacokinetics of emicizumab administered subcutaneously initially
once weekly (QW) in pediatric participants with hemophilia A with FVIII inhibitors. This
study will open two additional non-randomized cohorts to investigate once every 2 weeks (Q2W)
and once every 4 weeks (Q4W) regimens in pediatric participants.
Inclusion Criteria:
- Children less than (<) 12 years of age, with allowance for participants 12 to 17 years
of age who weigh <40 kilograms (kg) (Cohort A only); and participants <2 years of age
will be allowed to participate only after the protocol-defined interim data review
criteria are met (Cohort A only)
- Diagnosis of congenital hemophilia A of any severity and documented history of
high-titer inhibitor (that is [i.e.], greater than or equal to [>/=] 5 bethesda units
[BU])
- Requires treatment with bypassing agents
- Adequate hematologic, hepatic, and renal function
Exclusion Criteria:
- Inherited or acquired bleeding disorder other than hemophilia A
- Ongoing (or planning to receive during the study) immune tolerance induction (ITI)
therapy or prophylaxis treatment with FVIII
- Previous (in the past 12 months) or current treatment for thromboembolic disease or
signs of thromboembolic disease
- Other disease that may increase risk of bleeding or thrombosis
- History of clinically significant hypersensitivity associated with monoclonal antibody
therapy or components of the emicizumab injection
- Known infection with human immunodeficiency virus (HIV) or hepatitis B or C virus
- Use of systemic immunomodulators 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)
- Participants who are at high risk for thrombotic microangiopathy (TMA) (e.g., have a
previous medical or family history of TMA), in the investigator's judgement
We found this trial at
11
sites
Click here to add this to my saved trials
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
Click here to add this to my saved trials
St. Jude Children's Research Hospital St. Jude is unlike any other pediatric treatment and research...
Click here to add this to my saved trials
Children's Healthcare of Atlanta Whether treating a toddler in an emergency or supporting a teen...
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
1999 Marcus Avenue
New Hyde Park, New York 11042
New Hyde Park, New York 11042
Click here to add this to my saved trials
921 Terry Avenue
Seattle, Washington 98104
Seattle, Washington 98104
Click here to add this to my saved trials
Click here to add this to my saved trials