Evaluating the long-term effects of efanesoctocog alfa on joint health in hemophilia A patients in Taiwan
PROphylactic Efanesoctocog Alfa Therapy Evaluated for Critical Joint Health in Hemophilia A Treatment: The PROTECT-ALT Study
This study is testing how well efanesoctocog alfa helps protect joint health in people with hemophilia A in Taiwan over five years.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 100 (estimated) |
| Sex | All |
| Sponsor | Sanofi Industry-sponsored |
| Locations | 10 sites (Changhua and 9 other locations) |
| Trial ID | NCT06684314 on ClinicalTrials.gov |
What this trial studies
This observational study aims to assess the effectiveness, safety, and usage of efanesoctocog alfa prophylaxis treatment in participants with hemophilia A across multiple centers in Taiwan. The study will collect both retrospective data from medical records and prospective data during routine clinical visits over a five-year period. Joint imaging data will also be gathered for participants aged six and older who can undergo MRI or ultrasound examinations. No interventions will be administered, and participation does not require additional study-related visits.
Who should consider this trial
Good fit: Ideal candidates include individuals of all ages diagnosed with moderate to severe hemophilia A who are starting efanesoctocog alfa prophylaxis treatment.
Not a fit: Patients with mild hemophilia A or those who have had undetectable inhibitors for less than three years may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term joint health outcomes for hemophilia A patients receiving efanesoctocog alfa treatment.
How similar studies have performed: While this study utilizes a well-known treatment, the specific observational approach to long-term joint health outcomes in this population is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Participants with all ages and diagnosis of moderate-severe hemophilia A without current and/or at least three years of un-detectable inhibitor (\<0.6 BU) * Participants with moderate to severe hemophilia A as defined by FVIII level ≤ 5% * Participants starting efanesoctocog alfa prophylaxis treatment as per standard of care no more than three months prior to the enrollment date * Participants aged 6 years and older are able to undergo MRI examinations (sedation given, if necessary, and per investigator discretion) * Participants are able to undergo joint examinations * Physician's decision to treat the participant with efanesoctocog alfa is made prior to and independently of participation in the study * Signed and dated informed consent provided by the participant, or by the participant's legally acceptable representative for participants under the legal age before any study-related activities are undertaken. Assent should be obtained for pediatric participants according to local regulations Exclusion Criteria: * Participants with coagulation disorders other than hemophilia A * Participants diagnosed with other known bleeding disorder * Participants currently receive factor therapy and have signs of decreased response to FVIII therapy * Participants with a baseline Radiological Pettersson score (PS) of greater than 6 for each individual ankle * Enrollment in another concurrent clinical interventional study, or intake of an Investigational Medicinal Product within 3 months prior to inclusion in this study * Pregnant female participants The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Where this trial is running
Changhua and 9 other locations
- Investigational Site Number : 1580008 — Changhua, Taiwan (Recruiting)
- Investigational Site Number : 1580009 — Kaohsiung City, Taiwan (Recruiting)
- Investigational Site Number : 1580010 — Kaohsiung City, Taiwan (Recruiting)
- Investigational Site Number : 1580005 — Taichung, Taiwan (Recruiting)
- Investigational Site Number : 1580006 — Taichung, Taiwan (Recruiting)
- Investigational Site Number : 1580007 — Taichung, Taiwan (Recruiting)
- Investigational Site Number : 1580001 — Taipei, Taiwan (Recruiting)
- Investigational Site Number : 1580003 — Taipei, Taiwan (Recruiting)
- Investigational Site Number : 1580002 — Taipei, Taiwan (Recruiting)
- Investigational Site Number : 1580004 — Taoyuan City, Taiwan (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.