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

Official title

PROphylactic Efanesoctocog Alfa Therapy Evaluated for Critical Joint Health in Hemophilia A Treatment: The PROTECT-ALT Study

Overview

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.

Interventions

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

Primary outcome measures

• Change from baseline in total scores of the Hemophilia Joint Health Score (HJHS) for all joints [Time frame: At 1, 2, 3, 4 and 5 years]
• Change from baseline in annualized joint bleeding rate (AjBR) for treated and untreated bleeds [Time frame: At 1, 2, 3, 4 and 5 years]
• Number of target joint development, resolution and/or recurrence [Time frame: At 1, 2 ,3, 4 and 5 years]

Secondary outcome measures (12)

• Annualized bleeding rate (ABR) by type and location for treated and all (treated and untreated) bleeds [Time frame: At 1, 2, 3, 4 and 5 years]
• ABR for all bleeding episodes, including untreated bleeding episodes [Time frame: At 1, 2, 3, 4 and 5 years]
• Percentage of participants with zero joint bleeds [Time frame: At 1, 2, 3, 4 and 5 years]
• Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa [Time frame: At 1, 2, 3, 4 and 5 years]
• Number of injections and doses of efanesoctocog alfa to treat a bleeding episode [Time frame: At 1, 2, 3, 4 and 5 years]
• Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period [Time frame: At 1, 2, 3, 4 and 5 years]
• Annualized injection frequency per participant (assessed by prescription) during the followup period [Time frame: At 1, 2, 3, 4 and 5 years]
• Treatment adherence (%) as judged by the physician during the follow-up period [Time frame: At 1, 2, 3, 4 and 5 years]
• Occurrence of a change in treatment regimen at baseline and follow-up [Time frame: 5 years]
• Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores in all joints [Time frame: Every 6 months for 5 years]
• Change from baseline in joint status on functional HJHS [Time frame: At 1, 2, 3, 4 and 5 years]
• Number and percentage of joints with no prior joint damage at baseline, without joint damage on joint ultrasound [Time frame: At 5 years]

Eligibility 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

Taiwan · 10 centers

• Investigational Site Number : 1580008 — Changhua
• Investigational Site Number : 1580009 — Kaohsiung City
• Investigational Site Number : 1580010 — Kaohsiung City
• Investigational Site Number : 1580005 — Taichung
• Investigational Site Number : 1580006 — Taichung
• Investigational Site Number : 1580007 — Taichung
• Investigational Site Number : 1580001 — Taipei
• Investigational Site Number : 1580003 — Taipei
• … and 2 more centers

Identifiers

NCT: NCT06684314 · OBS18067 · U1111-1308-9835

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗