Elfabrio® for Fabry Disease

A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

Recruiting Observational Study
Fabry Disease
No Placebo Group Every participant receives an active treatment — no one gets a placebo. No Study Drug Researchers observe your health over time — no experimental treatment is given.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
18 and older
Sex
Any
Study type
Observational
Participants needed
100 (estimated)
Sponsor
Chiesi Farmaceutici S.p.A. · Industry
Who this trial is looking for

This trial is looking for adults with Fabry disease who are currently taking or plan to take Elfabrio®. Participants will be observed to assess the safety and effectiveness of the treatment in a clinical setting.

Are You a Good Fit for This Trial?
Must be taking Elfabrio®
Rules you out Heart failureSevere liver disease

You may be able to join if

  • I am over 18 years old.
  • I have been diagnosed with Fabry disease.
  • I am currently taking or plan to take Elfabrio®.
  • I am able to undergo cardiac magnetic resonance imaging (cMRI).
  • I have signed the informed consent form.
  • I have heart disease related to Fabry disease.
  • I have a kidney function measure (eGFR) above 45 mL/min/1.73 m2.
  • I have abnormal lysoGb3 levels if I am male.

You may not be able to join if

  • I am pregnant.
  • I have a history of severe allergies to gadolinium contrast agents.
  • I have a medical condition that would affect my study participation.
  • I participated in another study for Fabry disease recently.
  • I have had a heart attack or heart failure with a low ejection fraction.
  • I have had a stroke in the past 6 months.
  • I have severe liver disease.
  • I have received an investigational drug for Fabry disease recently.

Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.

Think this trial could be right for you?

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About This Trial

A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Male or female aged \> 18 years of age at the time of consent. * Genetically confirmed diagnosis of Fabry disease. * Either taking or planning to take pegunigalsidase alfa as treatment for Fabry disease. * No contraindications for cardiac magnetic resonance imaging (cMRI) * In…
Contacts

Medical Information Chiesi

(888) 661-9260

us.medical@chiesi.com

CONTACT