Phase III clinical trial of emicizumab in hemophilia A pediatric patients with inhibitors

A MULTICENTER, OPEN-LABEL, PHASE III CLINICAL TRIAL TO EVALUATE THE EFFICACY, SAFETY, AND PHARMACOKINETICS OF SUBCUTANEOUS ADMINISTRATION OF EMICIZUMAB IN HEMOPHILIA A PEDIATRIC PATIENTS WITH INHIBITORS

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080223243

Enrollment

Registered

2016-06-14

Start date

2016-07-28

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia A with inhibitors

Interventions

investigational material(s) Generic name etc : emicizumab (ACE910, RO5534262) INN of investigational material : emicizumab Therapeutic category code : 634 Human blood preparations Dosage and Administr

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1) Aged younger than 12 years old or 12~17 years old who weigh 3 kg at the time of informed consent 3) Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (i.e.,>= 5 Bethesda Units) 4) Requires treatment with bypassing agents

Exclusion criteria

Exclusion criteria: 1) Inherited or acquired bleeding disorder other than hemophilia A 2) Ongoing (or plan to receive during the study) immune tolerance induction therapy(ITI) or prophylaxis with FVIII. Patients awaiting initiation of ITI will be eligible. Patients in whom ITI has failed will be eligible with a 72-hour washout period prior to the first emicizumab administration. 3) Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which antithrombotic treatment is not currently ongoing) or signs of thromboembolic disease

Design outcomes

Primary

Measure	Time frame
efficacy primary outcome: To evaluate the clinical effect of prophylactic emicizumab in hemophilia A pediatric patients with inhibitors on the basis of number of bleeds over time. primary outcome method: Observation	—

Secondary

Measure	Time frame
safety efficacy pharmacokinetics secondary outcome: To evaluate the efficacy, safety and pharmacokinetics of prophylactic emicizumab in hemophilia A pediatric patients with inhibitors. secondary outcome method: Observation, Laboratory tests	—

Countries

10 countries including Japan, Japan

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026