Prospective, Multicenter, Observational Study to Evaluate Physical Activity, Bleeding Events, Quality of Daily Life, and Safety in Patients with Congenital Hemophilia A Without FVIII Inhibitors Under Emicizumab Prophylaxis

Prospective, Multicenter, Observational Study to Evaluate Physical Activity, Bleeding Events, Quality of Daily Life, and Safety in Patients with Congenital Hemophilia A Without FVIII Inhibitors Under Emicizumab Prophylaxis - TSUBASA Study An Observational Study of Emicizumab, a Bispecific Antibody recognizing coagulation factors IX and X, on Sports and physical Activities of people with haemophilia without FVIII inhibitors

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-UMIN000037448

Enrollment

160

Registered

2019-08-01

Start date

2019-11-01

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Congenital hemophilia A

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: (1) Informed consent form signed by the patient or legally acceptable representative Assent to participate in the study with signed informed assent form from pediatric patients when possible. (2) Diagnosis of congenital hemophilia A without FVIII inhibitors at the time of enrollment. (3) Patients for whom emicizumab treatment is selected as the most appropriate form of care (patients scheduled to receive emicizumab treatment in physician-patient treatment selection) and who are scheduled to undergo treatment per the latest package insert.

Exclusion criteria

Exclusion criteria: (1) Inherited or acquired bleeding disorder other than congenital hemophilia A. (2) Undergoing immune tolerance induction (ITI) at the time of enrollment. (3) Previous use of emicizumab. (4) Any other reason that, in the judgment of the investigator, would render the patient unsuitable for study participation.

Design outcomes

Primary

Measure	Time frame
1.Endpoints Related to Exercise Status and Bleeds:Patient-reported exercise status. Patient-reported status of bleeds attributable to exercise. Data of physical activity during exercise from wearable activity tracker. 2.Endpoints Related to Quality and Nature of Daily Life:Patient Health Related Quality of Life (HRQoL) determined with SF-36 scores or J-KIDSCREEN-52 scores. Work/study status determined with WPAI+CIQ:HS. Survey of physical activity with IPAQ. Questionnaire-based survey on daily life of patients and caregivers Data of non-exercise physical activity from wearable activity tracker. 3.Endpoints Related to Safety:Overall evaluation of the following measures on safety during emicizumab prophylactic treatment. -Adverse events, Presence of FVIII inhibitors	—

Measure

Time frame

1.Endpoints Related to Exercise Status and Bleeds:Patient-reported exercise status. Patient-reported status of bleeds attributable to exercise. Data of physical activity during exercise from wearable activity tracker. 2.Endpoints Related to Quality and Nature of Daily Life:Patient Health Related Quality of Life (HRQoL) determined with SF-36 scores or J-KIDSCREEN-52 scores. Work/study status determined with WPAI+CIQ:HS. Survey of physical activity with IPAQ. Questionnaire-based survey on daily life of patients and caregivers Data of non-exercise physical activity from wearable activity tracker. 3.Endpoints Related to Safety:Overall evaluation of the following measures on safety during emicizumab prophylactic treatment. -Adverse events, Presence of FVIII inhibitors

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Countries

Japan

Contacts

Public Contactthe TSUBASA Study The secretariat of

EP-CRSU Co.,Ltd. Clinical Research Headquarters

prj-tsubasa@eps.co.jp03-5804-5045

Outcome results