Phase III clinical trial of emicizumab given every 2 weeks and 4 weeks in hemophilia A pediatric patients without inhibitors (HOHOEMI)

Phase 3

Completed

• Conditions: Hemophilia A

• Registration Number: JPRN-jRCT2080223654
• Lead Sponsor: CHUGAI PHARMACEUTICAL CO., LTD

Brief Summary

Emicizumab administered Q2W or Q4W was efficacious and safe in paediatric patients with severe haemophilia A without inhibitors.

Detailed Description

Not available

Study Timeline

• Study Start: 2017-09-12
• Results First Posted: 2019-09-12
• Study Completion: 2020-03-05
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: completed
• Sex: All
• Target Recruitment: 13

Inclusion Criteria

1) Children < 12 years of age at the time of informed consent
2) Body weight > 3 kg at the time of informed consent
3) Diagnosis of severe congenital hemophilia A (i.e., endogenous FVIII activity level < 1%)
4) A negative test for FVIII inhibitors (i.e., < 0.6 BU/mL) in 8 weeks prior to enrollment
5) For patients < 2 years of age: Documentation of bleeds and treatment with coagulation factor in the last 12 weeks prior to enrollment
For patients >= 2 years of age: Documentation of bleeds and treatment with coagulation factor in the last 24 weeks prior to enrollment

Exclusion Criteria

1) Inherited or acquired bleeding disorder other than hemophilia A
2) Ongoing immune tolerance induction (ITI) therapy
3) Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
4) Other diseases (i.e., certain autoimmune disease [e.g., systemic lupus erythematosus, antiphospholipid antibody syndrome], cardiovascular disease [e.g., fallot's tetralogy, tricuspid atresia]) that may increase risk of bleeding or thrombosis
5) Patients who are at high risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or family history of TMA [e.g., thrombotic thrombocytopenic purpura, atypical hemolytic uremic syndrome]), in the investigator's judgment

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
efficacy<br>Observation

Secondary Outcome Measures

Name	Time	Method
efficacy<br>pharmacokinetics<br>safety<br>Observation, Laboratory tests