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

Sponsor: Chiesi Farmaceutici S.p.A.

NCT ID: NCT06663358

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Health checks and monitoring — no treatment given
Type of study
Observational (no treatment given)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Pegunigalsidase-alfa (drug)
How long the study runs
Study runs about 60 months (dates as stated)
About the drug or intervention
Pegunigalsidase-alfa — drug: Administered via intravenous (IV) infusion under conditions of routine clinical care
Patient visit burden
Not specified by the sponsor

In plain English

This study looks at how safe and effective Elfabrio (pegunigalsidase alfa) is in adults with Fabry disease, a rare inherited condition. It is observational, meaning researchers collect information without assigning treatments. It is running in several countries and is paid for by Chiesi Farmaceutici S.p.A.

Who can take part

  • Adults over 18, male or female, with a genetic diagnosis of Fabry disease
  • Already taking or planning to take pegunigalsidase alfa (Elfabrio) for Fabry disease
  • Able to have heart scans (cardiac magnetic resonance imaging) with gadolinium dye if in the heart group
  • Kidney filtering level (eGFR) above 45, checked in the last 6 months
  • Must sign a consent form and agree to complete electronic questionnaires
  • Extra rules apply for three groups: people with Fabry-related heart disease, people new to Fabry treatment, and people who took part in a previous Elfabrio study

Who may not be able to

  • Cannot have MRI scans, or a bad reaction to gadolinium dye that cannot be managed with pre-medication
  • Pregnant at enrolment
  • Any medical or mental health condition that doctors think would stop full participation
  • Currently taking part in another interventional Fabry disease study
  • Taking a dose other than the approved 1mg/kg every two weeks at enrolment
  • Previously took part in a pegunigalsidase alfa trial using 2mg/kg every 4 weeks
  • Heart group only: past heart attack, severe heart failure (pumping below 35%), stroke in the last 6 months, chronic liver scarring (cirrhosis), heart disease not related to Fabry, investigational Fabry drugs in the last 6 months, or past gene therapy, or heavy heart scarring on scans
  • New-treatment group only: any previous Fabry treatment (Replagal, Fabrazyme, or Galafold)

What taking part involves

  • • Participants take pegunigalsidase alfa (Elfabrio) as their Fabry treatment, at the approved dose of 1mg/kg every two weeks
  • • Researchers collect information about the medicine's safety and how well it works, including heart scans (cMRI)

Time commitment: Not stated — ask the trial team about how long the study lasts, how many visits are needed, and what taking part involves.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Observing health over time
Ages
18 Years and over
Who
All
Number of participants
100
Started
2024-11-06
Last checked
2024-11

Plain English Summary

What is this study?

  • • Testing a new treatment for fabry disease
  • • Clinical study - 100 participants
  • • 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

Who can take part?

  • • Ages 18 Years and over
  • • Diagnosed with fabry disease

Where?

  • • Edgbaston - University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital
  • • Salford - Salford Royal
  • • London - The Royal Free Hospital

This is a simplified summary. Always discuss with your doctor before making any decisions.

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.

Fabry Disease

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 18 Years and over
  • Who can join: All genders

Biomarkers mentioned

eGFR

What the study is looking for

  • ✓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 heart magnetic resonance imaging (cMRI)
  • ✓heart Cohort:

Who cannot take part

  • ✗safety concern to magnetic resonance imaging (MRI) including known history of hypersensitivity to gadolinium...
  • ✗Pregnant at the time of enrolment.
  • ✗Presence of any medical, emotional, behavioural, or psychological condition that, in the judgment of the physician,...
  • ✗Active participation in any interventional study for Fabry disease
  • ✗Prior participation in a pegunigalsidase alfa trial using a dose of 2 mg/kg administered every 4 weeks.
See the full 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) * Informed consent form (ICF) signed and dated indicating the individual has been informed of and agreed to all pertinent aspects of the study and is willing to comply with all study requirements, including completion of electronic patient reported outcomes (ePROs). * Cardiac Cohort: * Evidence of Fabry disease (FD)-related heart disease including one or more of the following: * Left ventricular hypertrophy (LVH) measured by left ventricular mass index (LVMI) (g/m2) elevation above age/sex specific reference ranges. * Posterior septum wall thickness (e.g., \>=13mm) not explained by other factors (e.g., hypertension) * Low native T1 mapping on cMRI. * Typical Fabry-like scar on cMRI * Participants can receive cardiac magnetic resonance imaging (cMRI) with gadolinium enhancement as part of their SoC. * Estimated glomerular filtration rate (eGFR) \>45 mL/min/1.73 m2, assessed within the prior 6 months. * Naïve Cohort: * Most recent eGFR\>45 mL/min/1.73 m2, assessed within prior 6 months. * Male participants should have abnormal elevation in plasma lysoGb3 as assessed within 6 months prior to enrolment. * Long-Term Cohort: * Participants previously enrolled in the open label study CLI-06657AA1-04 (Previously PB-102-F60) (using pegunigalsidase alfa at a dose of 1mg/kg every 2 weeks) who have initiated or plan to initiate commercial pegunigalsidase alfa (Elfabrio®). Exclusion Criteria: * Contraindication to magnetic resonance imaging (MRI) including known history of hypersensitivity to gadolinium contrast agent that is not managed by the use of premedication. * Pregnant at the time of enrolment. * Presence of any medical, emotional, behavioural, or psychological condition that, in the judgment of the physician, could interfere with the ability to participate in the study. * Active participation in any interventional study for Fabry disease * Treatment regimen at the time of enrolment in the study is different from the approved 1mg/kg every two weeks (note if regimen subsequently changes during the prospective part of the study, the participants can remain in the study) * Prior participation in a pegunigalsidase alfa trial using a dose of 2 mg/kg administered every 4 weeks. * Cardiac Cohort: * History of acute myocardial infarction or congestive heart failure with reduced left ventricular (LV) ejection fraction of less than 35%. * Cerebral vascular accident (CVA) in the prior 6 months. * Chronic liver cirrhosis. * FD-unrelated heart disease (e.g., scarring due to myocardial infarction, symptomatic occlusive coronary artery disease, moderate valvular heart disease not thought to be Fabry related). * The participant is or has been treated with any investigational drug for Fabry disease within 6 months of study start or investigational gene therapy for Fabry disease at any time point in the past. * Severe cardiac fibrosis defined as more than 3 segments that each have \>50% fibrosis upon late gadolinium enhancement cMRI at any prior cMRI. * Naïve Cohort: * Prior exposure to a FD therapy (Replagal®, Fabrazyme®, and Galafold®) at any time point. * Severe cardiac fibrosis defined as more than 3 segments that each have \>50% fibrosis upon late gadolinium enhancement cMRI on any prior cMRI

Where Is This Study? (3 UK sites)

University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital

Edgbaston B152TH, United Kingdom

Recruiting
Site contact (verified)

Salford Royal

Salford M6 8HD, United Kingdom

Recruiting
Site contact (verified)

The Royal Free Hospital

London NW3 2QG, United Kingdom

Recruiting
Site contact (verified)

How to Get in Touch

Medical Information Chiesi

Sponsor contact

CONTACT

(888) 661-9260 us.medical@chiesi.com
Data sourced from ClinicalTrials.gov · Last verified: 2024-11