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

NCT06684314 | Recruiting FDA Drug

• 2 other identifiers: OBS18067U1111-1308-9835
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 10

Brief 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.

Conditions

Hemophilia A

Keywords

Classic Hemophilia; Factor VIII Deficiency; Haemophilia; Hemophilia; Hemarthrosis; Blood Coagulation Disorders, Inherited; Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn

Outcome Measures

Primary Outcomes (3)

• Change from baseline in total scores of the Hemophilia Joint Health Score (HJHS) for all joints

  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.

  At 1, 2, 3, 4 and 5 years

• Change from baseline in annualized joint bleeding rate (AjBR) for treated and untreated bleeds

  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.

  At 1, 2, 3, 4 and 5 years

• Number of target joint development, resolution and/or recurrence

  At 1, 2 ,3, 4 and 5 years

Secondary Outcomes (37)

• Annualized bleeding rate (ABR) by type and location for treated and all (treated and untreated) bleeds

  At 1, 2, 3, 4 and 5 years

• ABR for all bleeding episodes, including untreated bleeding episodes

  At 1, 2, 3, 4 and 5 years

• Percentage of participants with zero joint bleeds

  At 1, 2, 3, 4 and 5 years

• Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa

  At 1, 2, 3, 4 and 5 years

• Number of injections and doses of efanesoctocog alfa to treat a bleeding episode

  At 1, 2, 3, 4 and 5 years

Study Arms (1)

Efanesoctocog alfa

Participants observed receiving treatment with efanesoctocog alfa for hemophilia A

Drug: Efanesoctocog alfa

Interventions

Efanesoctocog alfa DRUG

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

Also known as: ALTUVIIIO, BIVV001

Efanesoctocog alfa

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants from medical centers in Taiwan will be enrolled in the study after the introduction of efanesoctocog alfa for the treatment of hemophilia A. Eligible participants will include all ages, sexes and moderate to severe hemophilia A severities under efanesoctocog alfa prophylaxis treatment and able to undergo joint and/or MRI examinations during routine clinical practice.

You may qualify if:

• 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

You may not qualify if:

• 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
• Pregnant female participants
• The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (10)

Investigational Site Number : 1580008

Changhua, 500, Taiwan

RECRUITING

Investigational Site Number : 1580009

Kaohsiung City, 807, Taiwan

RECRUITING

Investigational Site Number : 1580010

Kaohsiung City, 83301, Taiwan

RECRUITING

Investigational Site Number : 1580005

Taichung, 40201, Taiwan

RECRUITING

Investigational Site Number : 1580006

Taichung, 40447, Taiwan

RECRUITING

Investigational Site Number : 1580007

Taichung, 40705, Taiwan

RECRUITING

Investigational Site Number : 1580001

Taipei, 100, Taiwan

RECRUITING

Investigational Site Number : 1580003

Taipei, 110, Taiwan

RECRUITING

Investigational Site Number : 1580002

Taipei, 114, Taiwan

RECRUITING

Investigational Site Number : 1580004

Taoyuan, 33305, Taiwan

RECRUITING

MeSH Terms

Conditions

Hemophilia A; Hemarthrosis; Blood Coagulation Disorders, Inherited; Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn

Interventions

BIVV001

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders; Hemic and Lymphatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Joint Diseases; Musculoskeletal Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Trial Transparency email recommended (Toll free for US & Canada)

CONTACT

800-633-1610; Contact-US@sanofi.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 8, 2024
• First Posted: November 12, 2024
• Study Start: November 11, 2024
• Primary Completion (Estimated): August 28, 2031
• Study Completion (Estimated): August 28, 2031
• Last Updated: October 2, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share

Locations

TW (10)