Efanesoctocog Alfa Multicenter Prospective Observational Study in patients with factor VIII deficiency in Japan

JRCT ID: jRCT1031240049

Registered date:25/04/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Patients with blood coagulation factor VIII deficiency
• Date of first enrollment: 26/04/2024
• Target sample size: 150
• Study type: Observational

Outcome(s)

• Primary Outcome: To collect and assess the safety information of efanesoctocog alfa in clinical practice

Secondary Outcome

-Assessment of prophylaxis treatment
-Assessment of bleeding during prophylaxis treatment
-Assessment of treatment for acute bleeding in patients during prophylaxis therapy and on demand therapy
-Assessment during perioperative period
-Adverse events (AEs)
-Impact on Quality of Life (QOL) of patients

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: Not applicable

Include criteria

1.Patients who understand the purposes and risks of this study and voluntarily give written informed consent to participate in this study
*If the patient is under 16 years of age, or if the patient has completed junior high school or is 16<= and <18 years of age and is judged to have insufficient judgment capability, the consent of the legal representative shall be obtained.
2.Patients with coagulation factor VIII deficiency who are scheduled to receive efanesoctocog alfa or have started treatment within the past 8 months (240 days)
*Regardless of age, sex, hemophilia severity (FVIII activity), prior FVIII treatment, and presence of previous inhibitors.

• Exclude criteria: Patients unable or unwilling to provide informed consent

Related Information

• Primary Sponsor: Kidani Yoko