NCT01839526

A Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease

A Cross-sectional Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 39 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: Male
• Age: 5 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

No investigational drug will be administered in this study for the treatment of Fabry disease. This will be a multicenter, multinational, non-treatment, cross-sectional study of young male patients with Fabry disease who have not yet initiated interventional treatment for this disease. The study will consist of a screening visit(s), a clinical investigation visit(s), and a follow-up phone contact. The objectives of the study are: * To document renal function and other Fabry disease manifestations across age in treatment-naïve, young male patients with Fabry disease. * To provide a reference group for comparison with interventional clinical trials of Fabry disease. The duration of each patient's participation in the study, inclusive of the screening visit and follow-up phone contact, will be approximately 12 weeks.

Detailed description

Patients who meet all eligibility criteria based on screening assessments will be scheduled to return to the clinic for assessments of renal function and other disease-related parameters, which may be scheduled over one or more clinical investigation visits. The clinical investigation visit(s) will be scheduled such that renal and cardiac assessments occur after the required medication washout (see exclusion criterion); other procedures may be performed either before or after the medication washout, at the discretion of the investigator. Up to 100 patients will be enrolled in the study, including a minimum of 15 patients in each of the following age groups (based on age at screening): 5 to 11 years, 12 to 17 years, and 18 to 25 years. All patients will be encouraged to enroll in the Fabry Registry (NCT00196742) for continued follow-up after completion of the study.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	Iohexol	Evaluations of renal and cardiac function are standard-of-care in young patients with Fabry disease. This study uses the more precise iGFR measurement of renal function. No investigational drug will be administered in this study for the treatment of Fabry disease.

Timeline

• Start date: 2013-05-01
• Primary completion: 2016-09-01
• Completion: 2016-09-01
• First posted: 2013-04-25
• Last updated: 2020-12-16

Locations

22 sites across 14 countries: United States, Argentina, Austria, Belgium, Brazil, Canada, Finland, France, Hungary, Norway, Poland, Spain, Taiwan, United Kingdom