Study of Efficacy and Safety of FRSW107 in Pediatric Patients With Severe Hemophilia A
Status:
Not yet recruiting
Trial end date:
2025-04-30
Target enrollment:
Participant gender:
Summary
This study was divided into four stages: screening period, main trial period, extension
period and follow-up period.
In the main trial, both groups received FRSW107 prophylactic therapy. The recommended initial
dose of prophylactic administration was 50 IU/kg, the dose range was 25 to 50 IU/kg, and the
recommended frequency of administration was once every three days (Q3D). The dose range could
be adjusted according to the patient's response. The main trial period was prophylaxis up to
≥50 exposure days (EDs) and ≥6 months.
The investigator may adjust the dose according to the clinical efficacy of the subjects (the
occurrence of bleeding and its clinical manifestations) and the concentration of FⅧ valley
according to the following principles.
If necessary, the investigator may adjust the dosing interval according to the clinical
efficacy of the subject (the occurrence of bleeding and its clinical manifestations) and the
concentration of FⅧ. Investigators are advised to inform sponsors or their research partners
when adjusting doses and dosing intervals during prophylaxis.
After participants completed prophylaxis until ≥50EDs and ≥6 months, participants'
willingness and investigator evaluation were used to decide whether to enter the extended
trial. All subjects entering the extended phase continued with the original prophylactic
regimen until 100EDs was dosed.
During the main trial period and the extended preventive treatment period, if the subjects
have breakthrough bleeding events requiring treatment, hemostatic treatment of breakthrough
bleeding with investigational drugs can be performed. The researchers can refer to the
treatment guidance for different degrees of bleeding in Table 6-1. Taking into account the
subject's prophylactic dose, severity of bleeding, site and extent of bleeding, clinical
status, and previous PK results (if any), the investigator determines the appropriate dose to
administer (recommended dose range: 25 to 50 IU/kg) and dosing times until the investigator
assessed significant control of bleeding episodes (e.g. reduction of pain and swelling) or
return to pre-bleeding activity. If the bleeding episode stops, the subject will continue
with the same dose and frequency of prophylactic medication as before the bleeding episode.