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

NCT06663358 RECRUITING

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.

Details

Lead sponsor	Chiesi Farmaceutici S.p.A.
Status	RECRUITING
Enrolment	100
Start date	2024-11-06
Completion	2029-11

Conditions

• Fabry Disease

Interventions

• Pegunigalsidase-alfa

Primary outcomes

• Estimated Glomerular Filtration Rate (eGFR) — 4 years
  eGFR, calculated using the Chronic Kidney Disease - Epidemiology Collaboration (eGFRCKD-EPI) equation from study baseline and from pegunigalsidase alfa treatment start to end of follow-up and to pre-specified time points (annually).
• Plasma Globotriaosylsphingosine (LysoGb3) Concentration — 4 years
  Levels of the Fabry disease biomarker plasma globotriaosylsphingosine (lyso-Gb3) over time, and change from study baseline and from pegunigalsidase alfa treatment start to end of follow-up.
• Left Ventricular Mass Index (LVMI; g/m2) — 4 years
  Change in LVMI over time as assessed by cardiac MRI. Based on LVM indexed by height and/or body surface area.
• High Sensitivity Troponin (hs-cTnT) — 4 years
  Change in levels of hs-cTnT over time
• Safety Assessments — 4 years
  The endpoints will include occurrence of SAEs, infusion-related reactions (IRRs), drug-related adverse events, and proportion of participants with ADAs over time.

Countries

United States, Slovenia, United Kingdom