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
921 Terry Avenue
Seattle, Washington 98104
Seattle, Washington 98104
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Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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St. Jude Children's Research Hospital St. Jude is unlike any other pediatric treatment and research...
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Children's Healthcare of Atlanta Whether treating a toddler in an emergency or supporting a teen...
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1999 Marcus Avenue
New Hyde Park, New York 11042
New Hyde Park, New York 11042
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