Fabry disease trials in United Kingdom

In United Kingdom 7 Also abroad 48

7 trials · From ClinicalTrials.gov, checked 11 November 2026. Ask the study team whether a trial fits you.

Phase 3Taking people
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
Where
London, Salford
Age
18 years to 60 years
People
16 worldwide
Run by
Idorsia Pharmaceuticals Ltd.
See details and how to joinNCT07778667 · EU 2026-525207-27-00
Phase 3Taking people
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Where
London, Manchester
Age
2 years to 11 years
People
8 worldwide
Run by
Amicus Therapeutics
See details and how to joinNCT06904261
No phaseTaking people
Fabry Disease Registry & Pregnancy Sub-registry
Where
Belfast, Birmingham, Cambridge, London, Manchester, Salford
People
9,000 worldwide
Run by
Genzyme, a Sanofi Company
See details and how to joinNCT00196742
Phase 2/3Taking people
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Where
London
Age
2 years to 17 years
People
22 worldwide
Run by
Chiesi Farmaceutici S.p.A.
See details and how to joinNCT06328608 · EU 2022-503128-29-00
No phaseTaking people
Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Where
London
People
10 worldwide
Run by
Chiesi Farmaceutici S.p.A.
See details and how to joinNCT06941025
No phaseTaking people
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
Where
Edgbaston, London, Salford
Age
18 years and over
People
100 worldwide
Run by
Chiesi Farmaceutici S.p.A.
See details and how to joinNCT06663358
No phaseTaking people
National Registry of Rare Kidney Diseases
Where
Bristol
People
35,000
Run by
UK Kidney Association
See details and how to joinNCT06065852