Investigating efficacy, safety and pharmacokinetics of concizumab prophylaxis in children below 12 years with haemophilia A or B with or without inhibitors

JRCT ID: jRCT2031220097

Registered date:28/05/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: haemophilia A or B with or without inhibitors
• Date of first enrollment: 22/03/2022
• Target sample size: 80
• Countries of recruitment: Bosnia and Herzegovina,Japan,Bulgaria,Japan,Canada,Japan,Estonia,Japan,France,Japan,Greece,Japan,India,Japan,Italy,Japan,Lithuania,Japan,Macedonia,,Japan,Norway,Japan,Poland,Japan,Romania,Japan,Russia,Japan,Spain,Japan,Sweden,,Japan,Thailand,Japan,Turkey,Japan,United Kingdom,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

Dose clinical trial drug
IMP: concizumab( no use of placebo)

Dosage and administration:.
-0.20 mg/kg subcutaneously once daily.
-Dose adjustment to 0.25 mg/kg, 0.20 mg/kg or 0.15 mg/kg depending on concizumab concentration in Visit 4

Outcome(s)

Primary Outcome

For inhibitor patients with at least 26 weeks on demand treatment during the last year before enrolment:
The number of treated spontaneous and traumatic bleeding episodes

Secondary Outcome

For non-inhibitor patients with at least 26 weeks PPX treatment during the last year before enrolment:
The number of treated spontaneous and traumatic bleeding episodes

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: < 12age old
• Gender: Male

Include 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

Exclude 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

Related Information

• Primary Sponsor: Shimizu Yu
• Secondary ID(s): NCT05135559