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

Sanofi

Status

Enrolling

Conditions

Hemophilia A

Treatments

Drug: Efanesoctocog alfa

Study type

Observational

Funder types

Industry

Identifiers

NCT06684314

OBS18067

U1111-1308-9835 (Registry Identifier)

Details and patient eligibility

About

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.

Enrollment

100 estimated patients

Sex

All

Volunteers

No Healthy Volunteers

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.