TAKUMI study (TAKUMI study)

Recruiting

• Conditions: Congenital hemophilia A

• Registration Number: jRCT1030240076
• Lead Sponsor: Chugai Pharmaceutical Co., Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-05-10
• Study Start: 2024-07-08
• Last Update Posted: 2025-03-28

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

Subjects will be included in the study if they meet all of the following items.

1. Patients with congenital haemophilia A (any age, male or female) who are undergoing regular bleeding prophylaxis including emicizumab or who are scheduled to start by the start of observation (Week 0 visit)
2. Patients who have received adequate explanation of the content of this study, and who have given written consent based on their own free will from the subjects of the study or their legally acceptable representative, and who are able to comply with the procedures of the study protocol.

Exclusion Criteria

Study subjects who fall under any of the following items will be excluded from the study:

1. Patients with concomitant systemic diseases (e.g., rheumatoid arthritis) with joint involvement
2. Patients with other bleeding disorders other than congenital hemophilia A
3. Patients who are judged by the research director or research co-workers at the participating medical organization to have a significant range of motion restriction or prosthesis in 4 or more of the 6 joints of the left and right foot, knee, and elbow.
4. Subjects whose MRI is judged to be difficult to perform by the study director or study co-workers
5. Patients with difficulties in using electronic patient-reported outcomes (electronic patient-reported outcome:ePRO) (patients or caregivers)
6. Patients who are participating in clinical research involving clinical trials or interventions (including planned participation)
7. Patients who are judged to be inappropriately participating in the study by the research director or research subinvestigator at the participating medical institution.

Study & Design

• Study Type: Observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Assessment of fluid collection		Assessment of fluid collection (joint fluid/blood) based on the central assessment of US images (foot, knee, and elbow).
Assessment of synovial thickening		Assessment of synovial thickening based on the central assessment of US images (foot, knee, and elbow).
Assessment of cartilaginous degeneration		Assessment of cartilaginous degeneration based on the central assessment of US images (foot, knee, and elbow).
Assessment of bone degeneration		Assessment of bone degeneration based on the central assessment of US images (foot, knee, and elbow).

Secondary Outcome Measures

Name	Time	Method
fluid retention		Assessment of fluid retention based on the central assessment of MRI images (foot, knee, and elbow).
synovial thickening		Assessment of synovial thickening based on the central assessment of MRI images (foot, knee, and elbow).
cartilaginous degeneration		Assessment of cartilaginous degeneration based on the central assessment of MRI images (foot, knee, and elbow).
bone degeneration		Assessment of bone degeneration based on the central assessment of MRI images (foot, knee, and elbow).