Actively Recruiting
Hemlibra in Mild Hemophilia A
Led by Indiana Hemophilia &Thrombosis Center, Inc. · Updated on 2024-03-15
20
Participants Needed
1
Research Sites
257 weeks
Total Duration
On this page
Sponsors
I
Indiana Hemophilia &Thrombosis Center, Inc.
Lead Sponsor
G
Genentech, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a single arm, phase 4, prospective, open-label, United States single-center study to determine the hemostatic characteristics of Hemlibra (emicizumab) as measured by coagulation laboratory parameters in the mild hemophilia A male patient population with endogenous altered FVIII (baseline FVIII activity of \>5% to 30%). The safety and hemostatic efficacy of Hemlibra (emicizumab) in this patient population will be investigated. Secondary outcomes will assess changes in joint health and quality of life in treated patients.
CONDITIONS
Official Title
Hemlibra in Mild Hemophilia A
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent form from the subject, parent or guardian
- Male sex
- Diagnosis of mild congenital hemophilia A with baseline Factor VIII level greater than 5% to 30% without current or history of Factor VIII inhibitor
- Any number of Factor VIII exposure days, including previously untreated patients
- Body mass index less than 30
- Age between 5 years and 45 years
- Medical documentation of bleeding events, outcomes, and hemostatic product use for 12 months prior to enrollment
- Willingness and ability to comply with visits, treatment plans, laboratory tests, questionnaires, activity tracking, and bleed diaries
- Willingness to undergo Stimate/DDAVP challenge if no prior adverse reaction and not previously non-responsive
- Adequate liver function as defined by bilirubin, AST, and ALT levels and no cirrhosis
- Adequate blood counts and coagulation parameters at screening
- Adequate kidney function as defined by serum creatinine and creatinine clearance
You will not qualify if you...
- Inherited or acquired bleeding disorder other than mild congenital hemophilia A
- Any bleeding disorder in addition to mild hemophilia A
- Current or prior Factor VIII inhibitor of any titer
- Female sex
- History or high risk of cardiovascular disease or ischemic heart disease
- High risk for thrombotic microangiopathy as judged by investigator
- History of illicit drug or alcohol abuse
- Recent or current treatment for thromboembolic disease except resolved catheter-associated thrombosis
- Conditions increasing bleeding or thrombosis risk such as autoimmune diseases
- History of severe allergic reactions to monoclonal antibody therapies or Hemlibra components
- Known HIV infection with CD4 counts below 200 cells/µL
- Use of systemic immunomodulators at enrollment except antiretroviral therapy
- Significant other diseases or conditions interfering with study conduct or increasing risk
- Prior Hemlibra use in investigational studies
- Recent or current use of other investigational drugs
- Inability to comply with study protocol as judged by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Indiana Hemophila @Thrombosis Center
Indianapolis, Indiana, United States, 46260
Actively Recruiting
Research Team
A
Amy D Shapiro, MD
CONTACT
U
Usha Sirimalle, MS
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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