Fabry disease: trials for referral
50 open trials in the registries we read. Each row links to its registry record.
Registry data only. Frontlight does not judge whether a patient can take part: the trial team decides.
Where the open trials are
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50 trials shown
Phase 32 sites
RecruitingPoland
2025-521408-24-00
Low dose corticosteroids adjacent to enzyme replacement therapy or chaperon therapy in patients with cardiac manifestation of Fabry disease
Medical University Of Lodz
Registry updated 4 Nov 2026
Registry record: EU CTISPhase 316 sites
RecruitingCanada, Germany, France, United Kingdom, United States
NCT07778667
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
Idorsia Pharmaceuticals Ltd.
Pre-treatment window Registry sentence: “Treatment-naïve or pseudo-naïve, i.e., without treatment with an approved therapy or any investigational therapy for Fabry disease for at least 6 months prior to screening.”
Registry updated 3 Nov 2026
Registry record: ClinicalTrials.govNo phase8 sites
RecruitingUnited States
NCT06906367
A Study of Patients With Fabry Disease (US Specific)
Amicus Therapeutics
Pre-treatment window Registry sentence: “Untreated patients”
Registry updated 28 Oct 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT04252066
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Amicus Therapeutics
Registry updated 28 Oct 2026
Registry record: ClinicalTrials.govPhase 312 sites
RecruitingBelgium, Germany, Spain, United Kingdom, Japan, United States
NCT06904261
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Amicus Therapeutics
Registry updated 22 Oct 2026
Registry record: ClinicalTrials.govNo phase284 sites
RecruitingArgentina, Australia, Belgium, Bulgaria, Brazil, Canada
NCT00196742
Fabry Disease Registry & Pregnancy Sub-registry
Genzyme, a Sanofi Company
Registry updated 14 Oct 2026
Registry record: ClinicalTrials.govNo phase12 sites
Not yet recruitingFrance
NCT07872852
Therapeutic Implications of CMR Low Native T1 in Non-treated Non-hypertrophic Females With Fabry Disease (FD)
University Hospital, Bordeaux
Registry updated 14 Oct 2026
Registry record: ClinicalTrials.govPhase 1/210 sites
RecruitingUnited States
NCT06270316
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
UniQure Biopharma B.V.
Registry updated 24 Sept 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingCzechia
NCT07495410
Anderson-Fabry Disease Fitness Improvement Training
General University Hospital, Prague
Registry updated 21 Sept 2026
Registry record: ClinicalTrials.govPhase 43 sites
RecruitingIndia
NCT05067868
A Study of Replagal in Children and Adults With Fabry Disease in India
Shire
Registry updated 3 Sept 2026
Registry record: ClinicalTrials.govNo phase1 site
By invitation onlySweden
NCT07560956
Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF)
Vastra Gotaland Region
Registry updated 27 Aug 2026
Registry record: ClinicalTrials.govNo phase2 sites
RecruitingUnited States
NCT01581424
Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study
University of Minnesota
Registry updated 14 Aug 2026
Registry record: ClinicalTrials.govPhase 2/312 sites
RecruitingAustria, Spain, France, United Kingdom, Norway, United States
NCT06328608
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Chiesi Farmaceutici S.p.A.
Registry updated 10 Aug 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT06610019
Cardiovascular Multimodality Imaging Study
Montefiore Medical Center
Registry updated 10 Aug 2026
Registry record: ClinicalTrials.govNo phase9 sites
By invitation onlyUnited States
NCT05368038
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Albert Einstein College of Medicine
Registry updated 28 Jul 2026
Registry record: ClinicalTrials.govNo phase7 sites
RecruitingItaly
NCT07109375
Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
Chiesi Italia
Registry updated 8 Jul 2026
Registry record: ClinicalTrials.govNo phase5 sites
RecruitingItaly
NCT06884358
Functional Capacity in Anderson-Fabry Disease Patients
IRCCS Policlinico S. Donato
Registry updated 2 Jul 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingRomania
NCT07575347
Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE)
Stefan Lujinschi
Registry updated 26 Jun 2026
Registry record: ClinicalTrials.govPhase 33 sites
Not yet recruitingBelgium, Germany, Spain
2025-521244-38-00
An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12-month Treatment with Migalastat in Pediatric Subjects (aged 2 to < 12 years) with Fabry Disease and Amenable GLA Variants
Amicus Therapeutics Inc.
Registry updated 24 Apr 2026
Registry record: EU CTISNo phase1 site
RecruitingCanada
NCT04856059
Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparametric MRI
University Health Network, Toronto
Registry updated 16 Apr 2026
Registry record: ClinicalTrials.govNo phase1 site
Not yet recruitingFrance
NCT07485660
Care Pathway for Patients With Fabry's Disease (Fabry-PATH)
University Hospital, Toulouse
Registry updated 20 Mar 2026
Registry record: ClinicalTrials.govNo phase5 sites
RecruitingGermany, Spain, United Kingdom, Italy, United States
NCT06941025
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
Chiesi Farmaceutici S.p.A.
Registry updated 19 Mar 2026
Registry record: ClinicalTrials.govPhase 2/310 sites
RecruitingJapan
NCT05710692
Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
Chiesi Farmaceutici S.p.A.
Registry updated 18 Mar 2026
Registry record: ClinicalTrials.govNo phase2 sites
RecruitingChina
NCT06539624
Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease
The Children's Hospital of Zhejiang University School of Medicine
Registry updated 27 Feb 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingFrance
NCT04440254
Long Duration Holter ECG in Fabry Disease
Institut National de la Santé Et de la Recherche Médicale, France
Registry updated 18 Feb 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingItaly
NCT07382128
Myocardial Perfusion CMR for Differentiating and Characterizing Hypertrophic Cardiomyopathy Phenotypes
IRCCS Azienda Ospedaliero-Universitaria di Bologna
Registry updated 2 Feb 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingChina
NCT07336394
Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
Chinese Academy of Medical Sciences, Fuwai Hospital
Registry updated 21 Jan 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingTaiwan
NCT05473637
Taiwan Associated Genetic and Nongenetic Small Vessel Disease
National Taiwan University Hospital
Registry updated 20 Jan 2026
Registry record: ClinicalTrials.govNo phase0 sites
Not yet recruiting
NCT07351136
Novel Diagnostic and Prognostic Predictors in Fabry Cardiomyopathy: Proof of Concept in a Rare Disease
Núcleo de Apoio à Investigação Clínica - FMUP
Registry updated 20 Jan 2026
Registry record: ClinicalTrials.govNo phase18 sites
RecruitingChina
NCT07187440
A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
Takeda
Registry updated 2 Jan 2026
Registry record: ClinicalTrials.gov
The same sites as a list
- United States13 open trials
- Italy8 open trials
- United Kingdom7 open trials
- France7 open trials
- Germany7 open trials
- China7 open trials
- Spain5 open trials
- Canada4 open trials
- Taiwan4 open trials
- Japan3 open trials
- Belgium3 open trials
- South Korea3 open trials
- Denmark3 open trials
- India2 open trials
- Sweden2 open trials
- Argentina2 open trials
- Russia2 open trials
- Poland1 open trials
- Philippines1 open trials
- Thailand1 open trials
- Malaysia1 open trials
- Brazil1 open trials
- Chile1 open trials
- Hungary1 open trials
- Netherlands1 open trials
- Estonia1 open trials
- Lithuania1 open trials
- Peru1 open trials
- Czechia1 open trials
- Austria1 open trials
- Norway1 open trials
- Romania1 open trials
- Slovenia1 open trials
- Australia1 open trials
Who treats this
Institutions with trials or publications in this disease (OpenAlex, registries). Institutions only, no people.
Azienda ospedaliera "Bianchi-Melacrino-Morelli"
Reggio Calabria, Italy
0 trials · 6 publications · ROR
Azienda Ospedaliera Universitaria Policlinico "Paolo Giaccone" di Palermo
Palermo, Italy
0 trials · 7 publications · ROR
Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
Brescia, Italy
0 trials · 5 publications · ROR
Cambridge University Hospitals NHS Foundation Trust
Cambridge, United Kingdom
1 trials · 11 publications · ROR
Chinese Academy of Medical Sciences & Peking Union Medical College
Beijing, China
0 trials · 10 publications · ROR
Cincinnati Children's Hospital Medical Center
Cincinnati, United States
5 trials · 16 publications · ROR
Consejo Nacional de Investigaciones Científicas y Técnicas
Buenos Aires, Argentina
0 trials · 7 publications · ROR
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy
2 trials · 9 publications · ROR
Fondazione Istituto Neurologico Nazionale Casimiro Mondino
Pavia, Italy
0 trials · 5 publications · ROR
IRCCS Azienda Ospedliero-Universitaria di Bologna Policlinico di Sant'Orsola
Bologna, Italy
0 trials · 28 publications · ROR
Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, United States
1 trials · 21 publications · ROR
National Hospital for Neurology and Neurosurgery
London, United Kingdom
1 trials · 11 publications · ROR
Ospedale P. Pederzoli – Casa di Cura Privata S.p.a.
Peschiera del Garda, Italy
0 trials · 8 publications · ROR
The Affiliated Taizhou People's Hospital of Nanjing Medical University
Taizhou, China
0 trials · 5 publications · ROR
University Hospitals Birmingham NHS Foundation Trust
Birmingham, United Kingdom
1 trials · 12 publications · ROR
What changed in the last 7 days
Registry facts, one per line, newest first.
Closed for recruitment
NCT05280548 · Phase 3 · Austria, Canada, China, Czechia, Germany, Denmark
Source: ClinicalTrials.govStatus changed: Completed → Running, not recruiting
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
NCT04020055 · Phase 3 · Australia, Spain, France, United Kingdom, Japan, Portugal
Source: ClinicalTrials.govOpened for recruitment
NCT05280548 · Phase 3 · Austria, Canada, China, Czechia, Germany, Denmark
Source: ClinicalTrials.govPhase changed: No phase → Phase 2
Long-Term Follow-up of Subjects Who Were Treated With ST-920
NCT05039866 · Phase 2 · Australia, Canada, Germany, United Kingdom, United States
Source: ClinicalTrials.gov
Results card
Trials that finished or stopped in the last 3 years, with the reason the registry gives and linked publications.
Phase 3Ended 6 Oct 2026
Completed107 participants
NCT03737214
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
No phaseEnded 19 May 2026
Completed500 participants
NCT06935578
RAre, But Not aLone: a Large Italian Network to Empower the Impervious diaGNostic Pathway of Rare cerEbrovascular Diseases (ALIGNED)
No phaseEnded 29 Apr 2026
Phase 3Ended 13 Apr 2026
Completed29 participants
NCT03614234
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
No phaseEnded 16 Dec 2025
No phaseEnded 29 May 2025
No phaseEnded 15 Apr 2025
Completed409 participants
NCT05409846
Screening of Fabry Disease in Portuguese Patients With Idiopathic Cardiomyopathies
Phase 1/2Ended 10 Apr 2025
Completed36 participants
NCT04046224
Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)
Phase 3Ended 31 Mar 2025
Completed20 participants
NCT05843916
Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease
Phase 1/2Ended 19 Mar 2025
Completed8 participants
2024-512695-34-00
A Phase I/II Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects with Fabry Disease.
Phase 3Ended 21 Jan 2025
Completed97 participants
NCT03566017
Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
Phase 3Ended 29 Nov 2024
Completed16 participants
NCT04049760
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
Phase 4Ended 25 Oct 2024
Completed8 participants
NCT06019728
A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
No phaseEnded 30 Sept 2024
No phaseEnded 6 Jun 2024
Phase 1Ended Apr 2024
Completed5 participants
NCT02800070
Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease
No phaseEnded 28 Apr 2024
Completed108 participants
NCT05046379
Studying Lipids as Potential Biomarkers in Patients With Fabry Disease
Phase 3Ended 3 Jan 2024
Completed20 participants
NCT04974749
A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease
No phaseEnded 14 Dec 2023
Completed15 participants
NCT05671770
Study Based on Electronic Health RecOrds to Identify Patients at High-risk of Fabry DiseasE (HOPE Fabry)
Not medical advice. fetched 6 Nov 2026