← Back to Search

Monoclonal Antibodies

FVIII + Emicizumab for Hemophilia A (PRIORITY Trial)

Phase 4

Waitlist Available

Led By Guy Young, MD

Research Sponsored by Children's Hospital Los Angeles

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age ≤12 years at the time of signing the informed consent

Male patients with severe (<1%) or moderate (<2%) hemophilia A

Must not have

Age >12 years at the time of signing the informed consent

Unwilling to receive exposure to intravenous FVIII concentrates.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up the goal is to complete all study procedures in 2 years.

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Drug Has Already Been Approved

Summary

This trial will study whether weekly FVIII injections are necessary to maintain tolerance in children with hemophilia A and inhibitors after successful immune tolerance induction.

Who is the study for?

This trial is for boys aged 12 or younger with severe or moderate hemophilia A who have had a high inhibitor response (>5 BU) but achieved immune tolerance. They must be currently on emicizumab or agree to switch to it, and within one year of successful immune tolerance induction as per ISTH criteria.

What is being tested?

The study tests if continuing weekly FVIII therapy alongside emicizumab is necessary to maintain immune tolerance in children post-successful ITI compared to just using emicizumab alone. It's a randomized, controlled study with two groups followed for up to two years.

What are the potential side effects?

Potential side effects are not detailed here, but generally could include reactions at the injection site, headaches, joint pain related to FVIII treatment, and possible allergic reactions or development of inhibitors against the treatments.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am 12 years old or younger.

Select...

I am a male with severe or moderate hemophilia A.

Select...

I am currently using emicizumab or open to switching to it.

Select...

I've successfully completed immune tolerance therapy for hemophilia within the last year.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am older than 12 years.

Select...

I do not want to receive IV treatments for clotting.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ the goal is to complete all study procedures in 2 years.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~the goal is to complete all study procedures in 2 years.

This trial's timeline: 3 weeks for screening, Varies for treatment, and the goal is to complete all study procedures in 2 years. 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 recurrence

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.

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

Trial Design

2Treatment groups

Active Control

Group I: Emicizumab + FVIII weeklyActive Control2 Interventions

In addition to emicizumab prophylaxis,participants will receive non-prophylactic exposure to FVIII concentrates through weekly 50 IU/kg ±10% doses - the choice of FVIII concentrate is at the discretion of the PI.

Group II: Emicizumab onlyActive Control1 Intervention

Participants will only receive emicizumab prophylaxis.

0 / 6Eligibility criteria met