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RECRUITINGOBSERVATIONAL

German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

Pegunigalsidase-alfa may represent an advance in ERT for FD, based on its unique pharmacokinetics and apparent low immunogenicity. The objective of the study is to document long term data on treatment with pegunigalsidase-alfa under "real world" conditions. 60 patients with FD (therapy-naïve or pretreated with agalsidase-alfa or agalsidase-beta) will be recruited in 8 German Fabry centers. The treatment duration/patient will be 2 years. All patients will be followed-up by the above listed Fabry expert centers.

Who May Be Eligible (Plain English)

Who May Qualify: - Males and females, ≥18 years, diagnosed with Fabry disease. - ERT naïve (patients with signs of organ involvement (kidney, heart and/or CNS signs) to be considered for ERT following the European Consensus Guidelines on ERT (Biegstraaten et al. 2015) or patients with neuropathic pain not controlled with pain medication or patients with GI symptoms not relieved with standard medication or ERT switch patients (under ERT for ≥12 months). - Subjects taking ACE inhibitors, ARBs, or renin inhibitors on a stable dose for at least 4 weeks before screening. - Subjects taking analgesics/antidepressants on a stable dose for at least 4 weeks before screening. - Female patients must have a negative pregnancy test and use a medically accepted form of contraception throughout the study. Who Should NOT Join This Trial: - Patient is unwilling to give willing to sign a consent form. - Patient is unable to comply with the clinical protocol. - Patients on dialysis. - Patient has a clinically significant organ disease (e.g., cancer in the past 5 years) that in the opinion of the investigator would preclude participation in the trial. - Patients with a history of organ transplantation. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: * Males and females, ≥18 years, diagnosed with Fabry disease. * ERT naïve (patients with signs of organ involvement (kidney, heart and/or CNS signs) to be considered for ERT following the European Consensus Guidelines on ERT (Biegstraaten et al. 2015) or patients with neuropathic pain not controlled with pain medication or patients with GI symptoms not relieved with standard medication or ERT switch patients (under ERT for ≥12 months). * Subjects taking ACE inhibitors, ARBs, or renin inhibitors on a stable dose for at least 4 weeks before screening. * Subjects taking analgesics/antidepressants on a stable dose for at least 4 weeks before screening. * Female patients must have a negative pregnancy test and use a medically accepted form of contraception throughout the study. Exclusion Criteria: * Patient is unwilling to give informed consent. * Patient is unable to comply with the clinical protocol. * Patients on dialysis. * Patient has a clinically significant organ disease (e.g., cancer in the past 5 years) that in the opinion of the investigator would preclude participation in the trial. * Patients with a history of organ transplantation.

Treatments Being Tested

DRUG

Pegunigalsidase-alfa

Standard of care

Locations (8)

Fabry disease center Berlin - Charité - Universitätsmedizin Berlin
Berlin, Germany
Fabry disease center Cologne, Universitätsklinikum Köln
Cologne, Germany
Fabry disease center Hamburg, Universitätsklinikum Hamburg
Hamburg, Germany
Fabry disease center Hannover, Universitätsklinikum Hannover
Hanover, Germany
Fabry disease center Mainz, Universitätsmedizin Mainz
Mainz, Germany
Fachinternistische Gemeinschaftspraxis, Müllheim
Müllheim, Germany
Fabry disease center Münster, Universitätsklinikum Münster
Münster, Germany
Fabry disease center Würzburg, Universitätsklinikum Würzburg
Würzburg, Germany