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Factor VIII Replacement

The Hemophilia Inhibitor Prevention Trial

Phase 3
Waitlist Available
Led By Margaret V Ragni, MD, MPH
Research Sponsored by Margaret Ragni
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 48 weeks
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial will compare two treatments to see which is better at preventing inhibitors (a protein that stops blood from clotting) in people with severe hemophilia A.

Eligible Conditions
  • Hemophilia A

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~48 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 48 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Inhibitor Formation
Secondary study objectives
Bleeding Events
FVIII Mutation
FVIII Trough Level
+2 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups
Experimental Treatment
Active Control
Group I: EmicizumabExperimental Treatment1 Intervention
Arm B: Emicizumab 1.5 mg/kg will be administered weekly by subcutaneous injection (following 3 mg/kg/wk x4 induction) in previously untreated children with severe hemophilia A beginning before the first bleed and continue up to 48 weeks.
Group II: EloctateActive Control1 Intervention
Arm A: Eloctate 65 IU/kg will be administered weekly by intravenous infusion in previously untreated children with severe hemophilia A beginning before the first bleed and continued up to 48 weeks.

Find a Location

Who is running the clinical trial?

Health Resources and Services Administration (HRSA)FED
85 Previous Clinical Trials
151,652 Total Patients Enrolled
1 Trials studying Hemophilia A
1 Patients Enrolled for Hemophilia A
Margaret RagniLead Sponsor
6 Previous Clinical Trials
70 Total Patients Enrolled
2 Trials studying Hemophilia A
1 Patients Enrolled for Hemophilia A
Margaret V Ragni, MD, MPHPrincipal InvestigatorUniversity of Pittsburgh
5 Previous Clinical Trials
63 Total Patients Enrolled
2 Trials studying Hemophilia A
1 Patients Enrolled for Hemophilia A
~0 spots leftby Jan 2026