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

Timeline

Screening 3 weeks

Treatment Varies

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

Awards & highlights

PRIORITY Trial 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.Check my eligibility

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.See study design

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.

PRIORITY Trial Eligibility Criteria

Inclusion Criteria

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

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I am 12 years old or younger.

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I am a male with severe or moderate hemophilia A.

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

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

PRIORITY Trial 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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Inhibitor recurrence

PRIORITY 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 / 4Eligibility criteria met