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

Phase 3

Completed

• Conditions: Hemophilia A with inhibitors

• Registration Number: JPRN-jRCT2080223243
• Lead Sponsor: CHUGAI PHARMACEUTICAL CO., LTD.

Brief Summary

Emicizumab prophylaxis achieved substantial efficacy and was well tolerated in children with hemophilia A with FVIII inhibitors.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-06-14
• Study Completion: 2019-04-11
• Results First Posted: 2019-12-12
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: completed
• Sex: All
• Target Recruitment: 88

Inclusion Criteria

1) Aged younger than 12 years old or 12~17 years old who weigh < 40 kg at the time of informed consent
2) Body weight > 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

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
efficacy<br>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.<br><br>primary outcome method: Observation

Secondary Outcome Measures

Name	Time	Method
safety<br>efficacy<br>pharmacokinetics<br>secondary outcome: To evaluate the efficacy, safety and pharmacokinetics of prophylactic emicizumab in hemophilia A pediatric patients with inhibitors.<br><br>secondary outcome method: Observation, Laboratory tests