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

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

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

Brief 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 Summary:

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.


Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome:

  • Glomerular Filtration Rate Estimated from Serum Creatinine (eGFR) [ Time Frame: Day 1 to Week 8 ]
    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 ]
    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 ]


Original Primary Outcome: Same as current

Current Secondary Outcome: Cardiovascular Function [ Time Frame: Day 1 to Week 8 ]

Assessed by electrocardiogram (ECG) and echocardiography


Original Secondary Outcome: Same as current

Information By: Sanofi

Dates:
Date Received: April 11, 2013
Date Started: May 2013
Date Completion:
Last Updated: October 5, 2016
Last Verified: October 2016