Investigating the safety of aPCC after emicizumab treatment in hemophilia A patients
aPCC and Emicizumab Safety Study in Congenital Hemophilia A Patients With Inhibitors (SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis)
This study is testing if a personalized low dose of a treatment called aPCC is safe for people with hemophilia A who are already using emicizumab to help manage bleeding.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 5 (estimated) |
| Ages | 6 Years and up |
| Sex | All |
| Sponsor | Emory University Academic / other |
| Drugs / interventions | emicizumab |
| Locations | 2 sites (Atlanta, Georgia and 1 other locations) |
| Trial ID | NCT04563520 on ClinicalTrials.gov |
What this trial studies
This study aims to evaluate the safety and hemostatic efficacy of a low personalized dose of activated prothrombin complex concentrate (aPCC) in individuals with hemophilia A who are on emicizumab prophylaxis. It focuses on measuring thrombin generation to personalize dosing for future acute bleeding events and surgical prophylaxis. The study includes both children and adults with a history of high titer inhibitors and aims to improve treatment protocols for managing hemophilia A.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals aged 6 years and older with moderately severe hemophilia A and documented high titer inhibitors.
Not a fit: Patients with other inherited or acquired bleeding disorders, or those currently experiencing an active bleed, may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to more effective and personalized treatment options for patients with hemophilia A, potentially reducing bleeding episodes and improving quality of life.
How similar studies have performed: Previous studies have shown promise in using personalized dosing strategies in hemophilia treatment, but this specific approach is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Moderately severe hemophilia A, defined as FVIII level \<0.05 IU/mL before development of an inhibitor * Age ≥6 years of age at time of informed consent * Documented on 2 occasions a high titer inhibitor (\>5 BU/mL) with a 72-hour washout within 2 years of enrollment * Parent/guardian (Legally Authorized Representative) or the patient has provided written informed consent * Adequate hematologic function (Hgb \>8 g/dL and platelet count \>100,000 µL) * Adequate hepatic function (total bilirubin ≤1.5 x ULN and both AST/ALT ≤3x ULN at screening (excluding known Gilbert's) * Adequate renal function (≤2.5 x ULN and CrCl ≥30 mL/min) Exclusion Criteria: * Inherited or acquired bleeding disorder other than hemophilia A excluding low VWF (\>30% VWF:RCo or VWF:GP1bm) * Had an active bleed requiring factor therapy at screening * Previous or current treatment for thromboembolic disease or signs of thromboembolic disease (excluding previously resolved line-associated thrombosis) * Had a surgical procedure 14 days before screening * Conditions that may increase the risk of bleeding or thrombosis * If the patient is treated with rFVIIa or aPCC seven days before screening * History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection * Had current use of any medication other than emicizumab that could affect the coagulation system. * Known HIV infection with CD4 count \<200 cells/µL within 24 weeks before screening. Testing is not required if \<35 years of age. * Use of systemic immunomodulators at enrollment or planned use during the study * Participants who are at high risk for TMA (for example, have a previous medical/family history of TMA), in the investigator's judgment * Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose an additional risk, or would, in the opinion of the investigator, preclude the participant's safe participation in and completion of the study
Where this trial is running
Atlanta, Georgia and 1 other locations
- Children's Healthcare of Atlanta — Atlanta, Georgia, United States (Recruiting)
- Emory University Hospital — Atlanta, Georgia, United States (Recruiting)
Study contacts
- Principal investigator: Robert Sidonio, MD — Emory University
- Study coordinator: Robert Sidonio, MD
- Email: robert.sidonio.jr@emory.edu
- Phone: 404-785-1637
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.