Brief Title
Study of Emicizumab Prophylaxis in Participants With Hemophilia A With or Without Inhibitors Undergoing Minor Surgical Procedures
Official Title
A Phase IV, Multicenter, Single-Arm, Open-Label Study of Emicizumab Prophylaxis in Patients With Hemophilia A With or Without Inhibitors Undergoing Minor Surgical Procedures
Brief Summary
This Phase IV, multicenter study will evaluate whether participants with Hemophilia A (PwHA)
with or without inhibitors receiving emicizumab prophylaxis can safely undergo minor surgical
procedures without additional prophylactic bypassing agents (BPA; for participants with
inhibitors) or factor VIII (FVIII; for participants without inhibitors).
Study Phase
Phase 4
Study Type
Interventional
Primary Outcome
Percentage of Participants Without Excessive Bleeding at Surgical Sites and Did Not Require BPA/FVIII Use for Bleeding Related to the Surgery, From the Start of Surgery Until Discharge, as Measured by the ISTH Hemostatic Efficacy Scale
Condition
Hemophilia A
Intervention
Emicizumab
Study Arms / Comparison Groups
PwHA With Inhibitors, Emicizumab: Surgery Not Performed Cohort
Description: This cohort included participants with Hemophilia A (PwHA) with inhibitors that were enrolled but did not have surgery. All participants received emicizumab via subcutaneous (SC) injection at a loading dose of 3 milligrams of medication per kilogram of body weight (mg/kg) once weekly for the first 4 weeks, followed by 1.5 mg/kg once weekly, or by any other approved maintenance regimen, as long as they continued to derive sufficient benefit. Participants must have received all loading doses prior to surgery and planned to continue emicizumab for a minimum of 1 month after surgery.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
14
Start Date
June 28, 2018
Completion Date
March 13, 2020
Primary Completion Date
March 13, 2020
Eligibility Criteria
Inclusion Criteria:
- Any age (newborn and older)
- Ability to comply with the study protocol, in the investigator's judgment
- Diagnosis of hemophilia A and current or history of an inhibitor (Bethesda titer ≥0.6
Bethesda units) and currently using bypassing agents (BPAs) for breakthrough bleeds
(for PwHA with inhibitors)
- Diagnosis of hemophilia A and no history of an inhibitor (Bethesda titer <0.6 Bethesda
units), or a history of an inhibitor that has been tolerized for >5 years and using
FVIII for breakthrough bleeds (for PwHA without inhibitors)
- Plan to receive at least 4 loading doses of emicizumab and been adherent to emicizumab
prophylaxis by the time of surgery
- Undergoing minor surgery within 60 days of study enrollment. Other minor surgical
procedures could be included upon consultation and approval of Medical Monitor, but
examples include central venous catheter insertion/removal/replacement, simple dental
extractions, colonoscopy, cystoscopy, or endoscopy with biopsy, excisional skin biopsy
- Must plan to continue emicizumab prophylaxis for at least 1 month after surgery
- For women of childbearing potential: agreement to remain abstinent (refrain from
heterosexual intercourse) or use contraceptive methods that result in a failure rate
of <1% per year during the study period
Exclusion Criteria:
- Diagnosis of a bleeding disorder other than hemophilia A
- Participants who have been tolerized to Factor VIII products (for PwHA with
inhibitors)
- Tolerized to FVIII products for <5 years (for PwHA without inhibitors)
- Using FVIII products to treat breakthrough bleeds (for PwHA with inhibitors)
- Treatment with BPAs or FVIII within 24 hours prior to surgical procedure
- Undergoing a major surgical procedure
- Previous (in the past 12 months) or current treatment for thromboembolic disease (with
the exception of previous catheter-associated thrombosis for which anti-thrombotic
treatment is not currently ongoing) or current signs of thromboembolic disease
- Other conditions (e.g., certain autoimmune diseases, including but not limited to
diseases such as systemic lupus erythematosus, inflammatory bowel disease, and
antiphospholipid syndrome) that may increase the risk of bleeding or thrombosis
- Patients who are at high risk for thrombotic microangiopathy (TMA), e.g., have a
previous medical or family history of TMA, in the investigator's judgment
- Would refuse treatment with blood or blood products, if necessary
- Any serious medical condition or abnormality in clinical laboratory tests that, in the
investigator's judgment, precludes the participant's safe participation in and
completion of the study
- Pregnant or lactating, or intending to become pregnant during the study; women of
childbearing potential must have a negative serum pregnancy test result within 7 days
before Study Day 1
- Treatment with any of the following: An investigational drug to treat or reduce the
risk of hemophilic bleeds within 5 half-lives of last drug administration before Study
Day 1; A non-hemophilia-related investigational drug within the last 30 days or 5
half-lives before Study Day 1 (whichever is longer); An investigational drug
concurrently
- History of clinically significant hypersensitivity associated with monoclonal antibody
therapies or components of the emicizumab injection
- Known human immunodeficiency virus (HIV) infection with CD4 count < 200
cells/microlitre within 24 weeks prior to enrollment
Gender
All
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
Clinical Trials, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT03361137
Organization ID
ML39791
Responsible Party
Sponsor
Study Sponsor
Genentech, Inc.
Study Sponsor
Clinical Trials, Study Director, Hoffmann-La Roche
Verification Date
February 2021