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
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Ages | 5 Years to 45 Years |
| Sex | Men only |
| Enrollment | 20 (estimated) |
| Sponsor | Indiana Hemophilia &Thrombosis Center, Inc. Academic / other |
| Drugs / interventions | emicizumab |
| Locations | 1 site (Indianapolis, Indiana) |
| Status | Recruiting |
| Started | July 25, 2022 |
| Main results expected | July 2025 |
| Study ends | July 2027 |
| Registry record updated | March 15, 2024 |
| Trial ID | NCT04567511 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.
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NXT007 versus emicizumab to prevent bleeds in people with Hemophilia A
This will test whether NXT007 prevents bleeding better than emicizumab in people aged 12 and older with Hemophilia A, including those with or without factor VIII inhibitors.
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Emicizumab prophylaxis for people with Type 3 von Willebrand disease
This trial will test whether regular emicizumab injections reduce bleeds compared with on-demand standard therapy for people aged 1 month and older with Type 3 von Willebrand disease, while also studying people already on prophylaxis using their prior data.
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Switching from Emicizumab to Marstacimab in Severe Hemophilia A
This study is testing if switching from emicizumab to marstacimab is safe and how well the body handles the new medication in people with severe hemophilia A.
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Testing fitusiran to prevent bleeds in severe hemophilia A patients switching from emicizumab
This study is testing if fitusiran can help prevent bleeding in men with severe hemophilia A who are switching from another treatment called emicizumab.
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Using Nuwiq to manage bleeding in hemophilia A patients during major surgery
This study is testing if Nuwiq can help prevent bleeding during major surgery for boys and men over 12 with severe hemophilia A who are already using another treatment called emicizumab.
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Understanding antibody changes in hemophilia A patients treated with emicizumab
This study looks at how the immune system reacts to emicizumab treatment in people with severe hemophilia A by checking leftover samples from their regular doctor visits.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.