Clinical trial Recruiting at 1 site Phase 4 Interventional Ages 5 to 45 Men only

Using Hemlibra to treat mild hemophilia A

Official title: Prospective, Single-Arm, Open-Label Use of Hemlibra (Emicizumab) in the Treatment of Mild Hemophilia A

Run by Indiana Hemophilia &Thrombosis Center, Inc. · NCT04567511 · Data updated 2026-11-12

This study is testing if Hemlibra can safely help men with mild hemophilia A manage their condition and improve their joint health and quality of life.

Quick facts

PhasePhase 4
Study typeInterventional
Ages5 Years to 45 Years
SexMen only
Enrollment20 (estimated)
SponsorIndiana Hemophilia &Thrombosis Center, Inc.
Academic / other
Drugs / interventionsemicizumab
Locations1 site (Indianapolis, Indiana)
StatusRecruiting
StartedJuly 25, 2022
Main results expectedJuly 2025
Study endsJuly 2027
Registry record updatedMarch 15, 2024
Trial IDNCT04567511 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

Ideal candidates for this study are male patients aged 5 to 45 years with mild congenital hemophilia A and baseline FVIII levels between 5% and 30%, without a history of FVIII inhibitors.

Probably not a fit

Patients with FVIII inhibitors or those under 5 years of age will not benefit from this study.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a single-arm, phase 4, open-label study that aims to evaluate the hemostatic efficacy and safety of Hemlibra (emicizumab) in male patients with mild hemophilia A, characterized by baseline Factor VIII activity levels between 5% and 30%. The study will enroll approximately 20 participants aged 5 to 45 years who do not have FVIII inhibitors. Primary outcomes will focus on coagulation laboratory parameters, while secondary outcomes will assess changes in joint health and quality of life. The study will be conducted at a single center in the United States.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option for patients with mild hemophilia A, improving their hemostatic control and overall quality of life.

How similar studies have gone: Other studies have shown success with Hemlibra in treating hemophilia, but this specific application in mild hemophilia A is less explored.

Who can join: the full rules

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Signed informed consent form from the subject, parent or guardian
* Male sex
* Diagnosis of mild congenital hemophilia A (baseline FVIII level of \>5% to 30%) without a current FVIII inhibitor or a history of FVIII inhibitor
* Any number of FVIII exposure days, including PUPs
* BMI \<30
* Age ≥5 years to ≤45 years
* Medical documentation of bleeding events, outcomes and hemostatic product usage for 12 months prior to study enrollment
* Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, including the health-related questionnaires, activity tracking, and bleed diaries, using systems provided during the study
* Willingness to undergo a Stimate/DDAVP challenge (only if the subject reports no adverse event associated with prior Stimate \[DDAVP/desmopressin acetate\] use); Stimate/DDAVP challenge will not be performed if the patient has a documented history of lack of response as defined by an increase of FVIII \< 2 times baseline level
* Adequate hepatic function, defined as total bilirubin ≤1.5 × age-adapted upper limit of normal (ULN) (excluding Gilbert's syndrome) and both AST and ALT ≤3 × age-adapted ULN at the time of screening, and no clinical signs or known laboratory/radiographic evidence consistent with cirrhosis
* Adequate hematologic function, defined as a platelet count ≥100,000/μL and a PT≤1.5 times the ULN at the time of screening
* Adequate renal function, defined as serum creatinine ≤2.5 × age-adapted ULN and creatinine clearance ≥30 mL/min by Cockcroft-Gault formula

Exclusion Criteria:

* Inherited or acquired bleeding disorder other than mild congenital hemophilia A (baseline FVIII level of \>5% to 30%)
* Any bleeding disorder other than or in addition to mild hemophilia A
* Current or prior inhibitor to FVIII (any titer)
* Female sex
* History of CVD, risk of CVD by the ASCVD risk estimator (defined as a subject having \>20% risk of a cardiovascular event within the next 10 years if the subject is ≥20 years of age) and/or a history of ischemic heart disease \[ICD codes 120-125\]
* High risk for TMA (eg, have a previous medical or family history of TMA), in the Study Investigator's judgment
* History of illicit drug or alcohol abuse by report or in the Study Investigator's judgment
* Previous (within the last 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 signs of thromboembolic disease
* Other conditions (eg, certain autoimmune diseases) that may currently increase the risk of bleeding or thrombosis
* History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the Hemlibra (emicizumab) injection
* Known HIV infection with CD4 counts \<200 cells/μL. HIV infection with CD4 counts ≥200 cells/μL permitted
* Use of systemic immunomodulators (eg, interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy
* Concomitant disease, condition, significant abnormality on screening evaluations or laboratory tests, or treatment that could interfere with the conduct of the study, or that would, in the opinion of the Study Investigator, pose an additional unacceptable risk in administering study drug to the patient
* Receipt of any of the following:

  * Hemlibra (emicizumab) in a prior investigational study
  * An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration
  * A non-hemophilia-related investigational drug within last 30 days or 5 half-lives, whichever is shorter
  * Any other investigational drug currently being administered or planned to be administered
* Inability to comply with the study protocol in the opinion of the Study Investigator

Where it's running

  • Indiana Hemophila @Thrombosis Center
    Indianapolis, Indiana, United States
    Recruiting

Study contacts

  • Principal investigator: Amy D Shapiro, MD — Indiana Hemophilia &Thrombosis Center, Inc.
  • Study coordinator: Amy D Shapiro, MD
  • Email: ashapiro@ihtc.org
  • Phone: 3178710000

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.