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

Use our guide to learn which trials are right for you!

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
Winston-Salem, North Carolina 27157
2275
mi
from 98109
Winston-Salem, NC
Click here to add this to my saved trials
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...
957
mi
from 98109
Los Angeles, CA
Click here to add this to my saved trials
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...
1864
mi
from 98109
Memphis, TN
Click here to add this to my saved trials
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...
2180
mi
from 98109
Atlanta, GA
Click here to add this to my saved trials
1026
mi
from 98109
Aurora, CO
Click here to add this to my saved trials
Chicago, Illinois 60612
1733
mi
from 98109
Chicago, IL
Click here to add this to my saved trials
3901 Beaubien Street
Detroit, Michigan 48201
1932
mi
from 98109
Detroit, MI
Click here to add this to my saved trials
2415
mi
from 98109
New Hyde Park, NY
Click here to add this to my saved trials
700 Southwest Campus Drive
Portland, Oregon 97201
148
mi
from 98109
Portland, OR
Click here to add this to my saved trials
500 5th Avenue
San Jose, 10110
2401
mi
from 98109
San Jose,
Click here to add this to my saved trials
1
mi
from 98109
Seattle, WA
Click here to add this to my saved trials