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

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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.


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
3901 Beaubien Street
Detroit, Michigan 48201
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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Los Angeles, CA
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262 Danny Thomas Pl
Memphis, Tennessee 38105
(901) 495-3300
St. Jude Children's Research Hospital St. Jude is unlike any other pediatric treatment and research...
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Memphis, TN
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1405 Clifton Road NE
Atlanta, Georgia 30322
404-785-6000
Children's Healthcare of Atlanta Whether treating a toddler in an emergency or supporting a teen...
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Atlanta, GA
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Aurora, CO
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Chicago, Illinois 60612
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Chicago, IL
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New Hyde Park, NY
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700 Southwest Campus Drive
Portland, Oregon 97201
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Portland, OR
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500 5th Avenue
San Jose, 10110
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San Jose,
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Seattle, WA
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Winston-Salem, North Carolina 27157
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Winston-Salem, NC
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