Investigating efficacy, safety and pharmacokinetics ofconcizumab prophylaxis in children below 12 years with haemophilia A or B with or withoutinhibitors

Phase 3

Recruiting

• Conditions: haemophilia A or B with or without inhibitors

• Registration Number: JPRN-jRCT2031220097
• Lead Sponsor: Shimizu Yu

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-05-28
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Recruiting
• Sex: Male
• Target Recruitment: 80

Inclusion Criteria

Diagnosis of congenital severe haemophilia A (FVIII <1%) or moderate/severe congenital haemophilia(<=2%), or congenital haemophilia with inhibitors.
-For arm 1 only: Male aged <12 years of age at the time of signing informed consent.
-For arm 1 only: Patients with historical medical records of a total of at least 26 weeks of treatment within the last 52 weeks prior to enrolmenta
-Patients with HAwI with medical records of a total of at least 26 weeks of on demand treatment within the last 52 weeks prior to enrolment.
-Patients with HBwI with medical records of a total of at least 26 weeks of on demand treatment within the last 52 weeks prior to enrolment
-Patients with HBwI regardless of the regimen and duration of previous haemophilia treatment
-Patients without inhibitors with medical records of a total of at least 26 weeks of PPX treatment within the last 52 weeks prior to enrolment

Exclusion Criteria

-Known or suspected hypersensitivity to study intervention or related products.
-Previous participation in this study. Participation is defined as signed informed consent.
-Participation (i.e., signed informed consent) in any study or programme with investigational
drug other than concizumab within 5 half-lives or 30 days before screening, whichever is
longer.
-Platelets <=100x109/L at screening.
-Fibrinogen below laboratory lower normal limit at screening.
-Hepatic dysfunction defined as AST and/or ALT >3 times the upper limit combined with total
bilirubin >1.5 times the upper limit at screening.
-Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) <=30 ml/min/1.73 m2

Study & Design

• Study Type: Interventional
• Study Design: Not specified