Chiesi Farmaceutici S.p.A.: Fabry Disease trials

The registry lists 10 Fabry Disease studies with Chiesi Farmaceutici S.p.A. as lead sponsor. 5 are open to participants now and 1 have results posted.

Phase 2 2Phase 3 4No phase 6(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world SettingRecruitingNCT07109375
• 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 BreastfeedingRecruitingNCT06941025
• A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry PatientsRecruitingNCT06663358
• A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry DiseaseRecruitingNCT06328608Phase 2/3
• Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry DiseaseRecruitingNCT05710692Phase 2/3
• Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry DiseaseCompletedNCT03566017Phase 3Results posted
• Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly InfusionCompletedNCT05186324
• Global Burden and Treatment Trajectiories in Italian Patients With Fabry DiseaseCompletedNCT04916977
• Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease PatientsApproved for marketingNCT04552691
• Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease PatientsCompletedNCT03614234Phase 3