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Clinical Trials in Taiwan / NCT06684314
Active, not recruiting Observational

A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan

NCT06684314 · tracked via the Priya Life Science Taiwan tracker
Sponsor
Sanofi
Phase
Observational
Started
2024-11-11
Last updated
2026-09-25

Condition(s) studied

Hemophilia A

Investigational drug(s) / intervention(s)

Efanesoctocog alfa →

Efanesoctocog alfa: This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practices.

Study summary

This is a national, multicenter, retrospective/prospective, observational study in Taiwan designed to assess effectiveness, safety, and usage of efanesoctocog alfa prophylaxis treatment in hemophilia A participants. The data related to efanesoctocog alfa effectiveness, safety and usage will be recorded prospectively during routine visits for up to 5 years following enrollment initiation and the retrospective data will be collected at least 12 months and up to 24 months prior to efanesoctocog alfa initiation. Joint imaging data will be collected in centers performing Joint U/S and/or MRI (≥6 years old). At least 12 months of retrospective data will also be collected from medical records, as available. Prospectively collected data will be recorded at routine clinical visits during a five-year follow-up period. The end of study is defined as the last participant's last visit. No intervention will be administered, and no study related visits are required.

Eligibility

Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Change from baseline in total scores of the Hemophilia Joint Health Score (HJHS) for all joints — At 1, 2, 3, 4 and 5 years
    The HJHS summarizes joint health by providing a clinical score in the domain of body structure and function (i.e., impairment), of the joints most commonly affected by bleeding in hemophilia: (left ankle, right ankle, left elbow, right elbow, left knee, right knee). The HJHS consists of assessments of swelling, duration of swelling, muscle atrophy, crepitus on motion, flexion loss, extension loss, joint pain and strength for elbows, knees and ankles and a global gait score. A higher score indicates worse joint health.
  • Change from baseline in annualized joint bleeding rate (AjBR) for treated and untreated bleeds — At 1, 2, 3, 4 and 5 years
    The AjBR is defined as the number of joint bleeding episodes occurring during the treatment period divided by the duration of the treatment period in days multiplied by 365.25. All types of joint bleeding episodes (spontaneous, traumatic, and type unknown) will be included in determining the annualized number.
  • Number of target joint development, resolution and/or recurrence — At 1, 2 ,3, 4 and 5 years

Trial sites (10)

FacilityCityRegionStatus
Investigational Site Number : 1580008 Changhua Taiwan
Investigational Site Number : 1580009 Kaohsiung City Taiwan
Investigational Site Number : 1580010 Kaohsiung City Taiwan
Investigational Site Number : 1580003 New Taipei City Taiwan
Investigational Site Number : 1580005 Taichung Taiwan
Investigational Site Number : 1580006 Taichung Taiwan
Investigational Site Number : 1580007 Taichung Taiwan
Investigational Site Number : 1580001 Taipei Taiwan
Investigational Site Number : 1580002 Taipei Taiwan
Investigational Site Number : 1580004 Taoyuan City Taiwan

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Other trials for the same condition

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults Active, not recruiting
A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A Active, not recruiting
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B Recruiting
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively Recruiting
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06684314 on ClinicalTrials.gov ↗ ← All trials in Taiwan