Actively Recruiting

All Genders
ID06684314

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

Led by Sanofi · Updated on 2025-10-02

100

Participants Needed

10

Research Sites

N/A

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are conducting a national, multicenter, observational study in Taiwan to assess the effectiveness, safety, and use of efanesoctocog alfa as a preventive treatment for people with hemophilia A. This study gathers data both retrospectively from 12 to 24 months before starting efanesoctocog alfa and prospectively for up to five years during routine clinical visits. It includes joint imaging data for participants aged six years and older where available. The study observes participants who are already receiving efanesoctocog alfa prophylaxis as part of their usual care, without administering any treatment or requiring study-specific visits. Data collection includes joint ultrasound or MRI scans, clinical assessments, and treatment usage, with no interventions added by the study team. The study follows participants for five years after enrollment, with retrospective data collected for at least one year prior to treatment initiation. Participants contribute data through their regular clinical visits, where researchers record changes in joint health scores, bleeding rates, treatment adherence, and joint imaging results over time. The study also monitors hospitalizations, surgeries, and adverse events related to hemophilia A and its treatment. The total participation duration includes retrospective data and a five-year prospective follow-up, ending with the last participant's final visit.

CONDITIONS

Brief Title

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

Who Can Participate

All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Participants of all ages diagnosed with moderate to severe hemophilia A without current or at least three years of undetectable inhibitor (<0.6 BU)
  • Hemophilia A defined by factor VIII level ≤ 5%
  • Starting efanesoctocog alfa prophylaxis treatment no more than three months before enrollment
  • Participants aged 6 years and older able to undergo MRI examinations if needed
  • Participants able to undergo joint examinations
  • Physician's decision to treat with efanesoctocog alfa made before and independently of study participation
  • Signed informed consent provided by participant or legal representative; assent for pediatric participants as per local rules
Not Eligible

You will not qualify if you...

  • Participants with coagulation disorders other than hemophilia A
  • Participants diagnosed with other known bleeding disorders
  • Participants currently receiving factor therapy with signs of decreased response to factor VIII therapy
  • Participants with a baseline Radiological Pettersson score greater than 6 for each ankle
  • Enrollment in another clinical interventional study or use of an investigational medicinal product within 3 months before inclusion
  • Pregnant female participants

AI-Screening

AI-Powered Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Long-term Monitoring

Duration - Up to 5 years

Participants who are receiving treatment with efanesoctocog alfa as prescribed in real-world clinical practice are observed over time to assess joint health, bleeding rates, treatment adherence, and other health outcomes.

Regular visits approximately every 6 months and annually for assessments

Trial Site Locations

Total: 10 locations

1

Investigational Site Number : 1580008

Changhua, Taiwan, 500

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2

Investigational Site Number : 1580009

Kaohsiung City, Taiwan, 807

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3

Investigational Site Number : 1580010

Kaohsiung City, Taiwan, 83301

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4

Investigational Site Number : 1580005

Taichung, Taiwan, 40201

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5

Investigational Site Number : 1580006

Taichung, Taiwan, 40447

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6

Investigational Site Number : 1580007

Taichung, Taiwan, 40705

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7

Investigational Site Number : 1580001

Taipei, Taiwan, 100

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8

Investigational Site Number : 1580003

Taipei, Taiwan, 110

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9

Investigational Site Number : 1580002

Taipei, Taiwan, 114

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10

Investigational Site Number : 1580004

Taoyuan City, Taiwan, 33305

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Research Team

T

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

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

1

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