BridgeMD
New treatment option for Factor VIII Deficiency, Congenital
Official title Hemlibra in Mild Hemophilia A
ClinicalTrials.gov ID: NCT04567511
What this study is testing
What is Emicizumab?
Emicizumab is an investigational medicine, given as an once-weekly injection under the skin, being studied as a potential treatment for factor viii deficiency, congenital.
Also referred to as Hemlibra.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 4: studies an already-approved treatment
- Time commitment: about 60 months
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 5 to 45, men only
You may be able to join if
- 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
You likely can't join if
- 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...
- High risk for TMA (eg, have a previous medical or family history of TMA), in the Study Investigator's judgment
See the full eligibility 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...
- 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...
- 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...
- 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
- 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...
- 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...
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Indianapolis, Indiana, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 60 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling male, 5 years to 45 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Indianapolis, Indiana, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.