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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
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
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
Where Is This Study? (3 UK sites)
University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital
Edgbaston B152TH, United Kingdom
Salford Royal
Salford M6 8HD, United Kingdom
The Royal Free Hospital
London NW3 2QG, United Kingdom
How to Get in Touch
Medical Information Chiesi
Sponsor contactCONTACT
