A Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease
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.
|Study Design:||Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Basic Science
|Official Title:||A Cross-sectional Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease|
- Glomerular Filtration Rate Estimated from Serum Creatinine (eGFR) [ Time Frame: Day 1 to Week 8 ] [ Designated as safety issue: No ]Tests are performed only for patients who are not on chronic dialysis and/or have not received a kidney transplant.
- Glomerular Filtration Rate by Plasma Iohexol Clearance (iGFR) [ Time Frame: Day 1 to Week 8 ] [ Designated as safety issue: No ]Assessment of iGFR will NOT be performed if a patient is on chronic dialysis, has had a kidney transplant, or has a screening eGFR <30 mL/min/1.73 m^2 or presents any contraindication mentioned in the labeling of iohexol (Omnipaque™ 300).
- Protein Excretion Assessed from Three First-Morning Urine Voids [ Time Frame: Day 1 to Week 12 ] [ Designated as safety issue: No ]
- Cardiovascular Function [ Time Frame: Day 1 to Week 8 ] [ Designated as safety issue: No ]Assessed by electrocardiogram (ECG) and echocardiography
|Study Start Date:||May 2013|
|Estimated Study Completion Date:||August 2016|
|Estimated Primary Completion Date:||August 2016 (Final data collection date for primary outcome measure)|
Glomerular Filtration Rate by Plasma Iohexol Clearance (iGFR)
Evaluations of renal and cardiac function
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.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01839526
|Contact: For site information, send an email with site number to||Contact-Us@sanofi.com|
|Contact: Medical Informationemail@example.com|
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|Study Director:||Medical Monitor||Genzyme, a Sanofi Company|