Fabry Disease — Trials, FDA Actions & Pivotal Research

20 active Fabry Disease trials currently recruiting in the US, sourced from ClinicalTrials.gov.

Synced daily from openFDA and ClinicalTrials.gov. Last sync: June 12, 2026.

Recruiting trials by city

Cities where Fabry Disease trials in our archive had recruiting sites. Each link lists every currently recruiting paid clinical trial at that location.

Birmingham, AL Irvine, CA

Active Fabry Disease trials

Currently recruiting US trials matching Fabry Disease, sourced live from ClinicalTrials.gov. We’ll add per-event articles here as those trials post results or hit primary completion.

Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE)

for Periodontal Disease

Sponsor: Stefan Lujinschi

Sex: All·Age: 18+

Code: NCT07575347·Not Yet Recruiting

Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF)

for Fabry Disease

Sponsor: Vastra Gotaland Region

Sex: All·Age: 18+

Code: NCT07560956·Not Yet Recruiting

Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting

for Fabry Disease

Sponsor: Chiesi Italia

Sex: All·Age: 18+

Code: NCT07109375·Not Yet Recruiting

Care Pathway for Patients With Fabry's Disease (Fabry-PATH)

for Fabry Disease

Sponsor: University Hospital, Toulouse

Sex: All·Age: 18+

Code: NCT07485660·Not Yet Recruiting

A Study of Patients With Fabry Disease (US Specific)

for Fabry Disease

8 locations incl. Birmingham

Sponsor: Amicus Therapeutics

Sex: All·Age: 18+

Code: NCT06906367·Recruiting

Apply to this trial

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

for Fabry Disease

6 locations incl. Atlanta

Sponsor: Amicus Therapeutics

Sex: All·Age: 2 - 11

Code: NCT06904261·Phase 3·Recruiting

Apply to this trial

Novel Diagnostic and Prognostic Predictors in Fabry Cardiomyopathy: Proof of Concept in a Rare Disease

for Fabry Disease

Sponsor: Núcleo de Apoio à Investigação Clínica - FMUP

Sex: All·Age: 18+

Code: NCT07351136·Not Yet Recruiting

Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study

for Fabry Disease

Sponsor: Yonsei University

Sex: All·Age: 15 - 75

Code: NCT07235709·Recruiting

Apply to this trial

A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease

for Fabry Disease

Sponsor: Takeda

Sex: All·Age: 7+

Code: NCT07187440·Recruiting

Apply to this trial

Anderson-Fabry Disease Fitness Improvement Training: A-FAD-FIT

for Fabry Disease

Sponsor: General University Hospital, Prague

Sex: All·Age: 18+

Code: NCT07495410·Enrolling By Invitation

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease

for Fabry Disease

6 locations incl. Phoenix

Sponsor: Chiesi Farmaceutici S.p.A.

Sex: All·Age: 2 - 17

Code: NCT06328608·Phase 2, Phase 3·Recruiting

Apply to this trial

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

for Fabry Disease

1 location in Washington D.C.

Sponsor: Chiesi Farmaceutici S.p.A.

Sex: Female·Age: All ages

Code: NCT06941025·Recruiting

Apply to this trial

Aortic Dimensions in Patients With Fabry Disease

for Fabry Disease

Sponsor: University Hospital, Caen

Sex: All·Age: All ages

Code: NCT06956573·Not Yet Recruiting

the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease

for Fabry Disease

Sponsor: Caroline Michaela Kistorp

Sex: All·Age: 18+·Accepts healthy volunteers

Code: NCT06776419·Recruiting

Apply to this trial

Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease

for Fabry Disease

Sponsor: NPO Petrovax

Sex: All·Age: 8 - 65

Code: NCT06880250·Enrolling By Invitation

A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

for Fabry Disease

1 location in Philadelphia

Sponsor: Amicus Therapeutics

Sex: Female·Age: All ages

Code: NCT04252066·Recruiting

Apply to this trial

The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease

for Fabry Disease

Sponsor: Guangzhou Jiayin Biotech Ltd

Sex: All·Age: 18+

Code: NCT06819514·Phase 1, Phase 2·Not Yet Recruiting

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

for Fabry Disease

6 locations incl. Birmingham

Sponsor: Chiesi Farmaceutici S.p.A.

Sex: All·Age: 18+

Code: NCT06663358·Recruiting

Apply to this trial

Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease

for Fabry Disease

Sponsor: The Children's Hospital of Zhejiang University School of Medicine

Sex: All·Age: 7+

Code: NCT06539624·Recruiting

Apply to this trial

Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment

for Fabry Disease

Sponsor: Wladimir MAUHIN, Dr

Sex: All·Age: 65+

Code: NCT07277361·Recruiting

Apply to this trial