Examining goal attainment and physical activity in people with hemophilia A
Prospective, Observational Study of the Impact of Efanesoctocog Alfa (ALTUVIIIO®) on Goal Attainment and Physical Activity in People With Moderate or Severe Hemophilia A
This study is testing if a new treatment for hemophilia A helps people aged 12 to 50 become more active and reach their health goals.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 35 (estimated) |
| Ages | 12 Years to 50 Years |
| Sex | All |
| Sponsor | Sanofi Industry-sponsored |
| Locations | 1 site (Peoria, Illinois) |
| Trial ID | NCT06530030 on ClinicalTrials.gov |
What this trial studies
This observational study focuses on individuals aged 12 to 50 years with moderate or severe hemophilia A who are beginning prophylaxis with efanesoctocog alfa. Conducted across multiple centers in the US and Japan, the study aims to enroll 35 patients to assess their goal attainment and physical activity levels. Participants will use a smartphone-compatible activity tracking device to report their outcomes electronically. The study will provide insights into the effectiveness of the new prophylactic treatment in improving physical activity among this patient population.
Who should consider this trial
Good fit: Ideal candidates are individuals aged 12 to 50 years with moderate or severe hemophilia A who are newly starting prophylaxis with efanesoctocog alfa.
Not a fit: Patients with a current diagnosis of FVIII inhibitors or those who have used efanesoctocog alfa for prophylaxis in the past six months may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance understanding of how efanesoctocog alfa impacts physical activity and goal attainment in patients with hemophilia A.
How similar studies have performed: While this study is observational, similar approaches in assessing physical activity in hemophilia patients have shown promise, indicating potential for valuable insights.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * At enrollment, newly starting prophylaxis therapy with efanesoctocog alfa according to usual clinical practice, with efanesoctocog alfa initiated within 6 months after the enrollment visit * Diagnosis of moderate (endogenous FVIII activity between 1% to 5% of normal) or severe (endogenous FVIII activity \<1% of normal) hemophilia A * Aged 12 to 50 years at time of enrollment, inclusive * Access to a smartphone device (Android version 12.0 or higher; or iOS 13 or higher) with Bluetooth 4.0 (minimum) or 5.0 (recommended) capabilities for compatibility with physical activity tracker * Availability of home-based access to internet for electronic patient-reported outcome (ePRO)/diary assessments * Willingness to utilize the activity tracking device Exclusion Criteria: * Current diagnosis of a FVIII inhibitor, defined as inhibitor titer ≥ 0.60 BU/mL * Use of efanesoctocog alfa for prophylaxis in the 6 months prior to enrollment NOTE: Other Inclusion/Exclusion criteria may apply. The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Where this trial is running
Peoria, Illinois
- Bleeding and Clotting Disorders Institute — Peoria, Illinois, United States (Recruiting)
Study contacts
- Study coordinator: Trial Transparency email recommended (Toll free for US & Canada)
- Email: Contact-US@sanofi.com
- Phone: 800-633-1610
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.