NCT07109375

Brief Summary

PEGASO is an observational study designed to collect prospective data on the effectiveness and safety of pegunigalsidase alfa in adult patients with Fabry disease, being treated or planning to start treatment, under real-world setting.

Trial Health

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Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
75

participants targeted

Target at P50-P75 for all trials

Timeline
32mo left

Started Apr 2026

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress4%
Apr 2026Dec 2028

First Submitted

Initial submission to the registry

July 10, 2025

Completed
28 days until next milestone

First Posted

Study publicly available on registry

August 7, 2025

Completed
8 months until next milestone

Study Start

First participant enrolled

April 1, 2026

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2028

Last Updated

February 24, 2026

Status Verified

February 1, 2026

Enrollment Period

2.7 years

First QC Date

July 10, 2025

Last Update Submit

February 23, 2026

Conditions

Fabry Disease

Keywords

Pegunigalsidase alfaFabry diseaseReal-worldAlpha-galactosidase ALysosomal storage disorders

Outcome Measures

Primary Outcomes (3)

  • Assessment of structural abnormalities of the left ventricle

    Changes from baseline in structural abnormalities of the left ventricle assessed by echocardiography and defined by the presence of Left Ventricular Hypertrophy.

    24 months

  • Assessment of left ventricular diastolic function

    Changes from baseline in left ventricular diastolic function by echocardiography. Diastolic dysfunction will be assessed using early to late diastolic trans-mitral flow velocity.

    12 and 24 months

  • Assessment of renal function

    Changes from baseline in renal function by estimated Glomerular Filtration Rate.

    6, 12, 18 and 24 months

Secondary Outcomes (16)

  • Assessment of peak oxygen uptake (pVO2)

    24 months

  • Assessment of carbon dioxide production (VCO2)

    24 months

  • Assessment of NT-pro-BNP and high sensitivity cardiac troponin levels

    6, 12,18 and 24 months

  • Assessment of Cardiovascular Magnetic Resonance (CMR)

    12 and 24 months

  • Assessment of proteinuria and microalbuminuria

    6, 12, 18 and 24 months

  • +11 more secondary outcomes

Study Arms (1)

Fabry patients

Adult patients whit genetically confirmed diagnosis of Fabry disease being treated or planning to start treatment with Pegunigalsidase alfa according to clinical practice. Participants will be required to meet the inclusion and exclusion criteria and sign informed consent to be enrolled in the study.

Drug: pegunigalsidase alfa

Interventions

pegunigalsidase alfaDRUG

Pegunigalsidase alfa is 2 mg/mL concentrate for solution and is administered via intravenous infusion every two weeks.

Also known as: Elfabrio
Fabry patients

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Adults patients who have a genetically confirmed diagnosis of Fabry disease and are being treated or plan to initiate treatment with pegunigalsidase alfa as per clinical practice.

You may qualify if:

  • Male and female adults (≥ 18 years).
  • Patients with a clinical diagnosis of Fabry disease confirmed by α-Gal A assay and detection of mutation in α-Gal A gene.
  • Patients either taking or planning to take pegunigalsidase alfa as treatment for Fabry disease. The treatment decision must be made independently from participation in this study.
  • Written informed consent to participate in the study and for the processing of personal data.

You may not qualify if:

  • History of hypersensitivity reaction to pegunigalsidase alfa.
  • 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.
  • Female subjects who are pregnant or planning to become pregnant during the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

AOU Federico II, Dipartimento di Nefrologia

Naples, Italy

Location

MeSH Terms

Conditions

Fabry DiseaseLysosomal Storage Diseases

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesCerebral Small Vessel DiseasesCerebrovascular DisordersVascular DiseasesCardiovascular DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsLipidosesLipid Metabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Study Officials

  • Antonio Pisani, MD

    AOU Federico II, Dipartimento di Nefrologia, Napoli, Italy

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Chiesi Clinical Trial info

CONTACT

+3905212791clinicaltrials_info@chiesi.com

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 10, 2025

First Posted

August 7, 2025

Study Start

April 1, 2026

Primary Completion (Estimated)

December 1, 2028

Study Completion (Estimated)

December 1, 2028

Last Updated

February 24, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will not share

Locations

IT(1)