A Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease
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|ClinicalTrials.gov Identifier: NCT01839526|
Recruitment Status : Terminated (Enrollment period not extended and screening stopped for slow recruitment and in accordance with provisions of the protocol. Not linked to any safety concern)
First Posted : April 25, 2013
Last Update Posted : October 6, 2016
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.
|Condition or disease||Intervention/treatment||Phase|
|Fabry Disease||Drug: Iohexol||Not Applicable|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||39 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Primary Purpose:||Basic Science|
|Official Title:||A Cross-sectional Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease|
|Study Start Date :||May 2013|
|Actual Primary Completion Date :||September 2016|
|Actual Study Completion Date :||September 2016|
U.S. FDA Resources
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.
- 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 ]
- Cardiovascular Function [ Time Frame: Day 1 to Week 8 ]Assessed by electrocardiogram (ECG) and echocardiography
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01839526
Show 22 Study Locations
|Study Director:||Medical Monitor||Genzyme, a Sanofi Company|