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

Fabry Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01839526
• Other study ID #: AGAL19110
• Secondary ID: 2012-001966-14MS
• Status: Terminated
• Phase: Phase 1
• Start date: May 2013
• Est. completion date: September 2016

Study information

• Verified date: October 2016
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 39
• Est. completion date: September 2016
• Est. primary completion date: September 2016
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years to 25 Years

Eligibility

Inclusion Criteria:

• The patient and/or their parent/legal guardian is willing and able to provide signed informed consent. If the patient is below the age of consent per local guidelines, he is willing to provide assent, if deemed able to do so.
• The patient must have a confirmed diagnosis of Fabry disease as documented by leukocyte a-galactosidase A (aGAL) of <4 nmol/hr/mg leukocyte (preferred assay; results from a central laboratory). If the leukocyte aGAL activity assay is difficult to obtain, the patient may be enrolled based on documented plasma aGAL <1.5 nmol/hr/mL (results from a central laboratory), with the agreement of the Genzyme Medical Monitor.

Exclusion Criteria:

• Patient has received prior treatment with enzyme replacement therapy (ERT) or oral pharmacological chaperone therapy for Fabry disease.
• Patient has received an investigational drug within 30 days of the screening visit.
• Patient is receiving any of the following medications and is clinically unable or unwilling to temporarily discontinue treatment with these medications for the indicated washout period prior to the renal function assessments until completion of

these assessments:

• Angiotensin converting enzyme inhibitors or angiotensin receptor blockers (6 week washout);
• Non-steroidal anti-inflammatory drugs (3 day washout).
• NOTE: Patients who are on chronic dialysis or have had a kidney transplant will not be required to discontinue the above medications because renal function assessments will not be performed in these patients.
• Patient has any contraindication mentioned in the labeling of iohexol. NOTE: patients with an eGFR <30 mL/min/1.73m^2 and patients who are on chronic dialysis or have had a kidney transplant may be enrolled irrespective of any contraindication to iohexol because iGFR will not be measured in these patients.
• Patient has any medical condition or extenuating circumstance which, in the opinion of the Investigator, could interfere with the patient's ability to complete all study procedures, or with the interpretation of study results (e.g., diabetes mellitus).
• The patient and/or their parent or legal guardian, in the opinion of the Investigator, is unable to adhere to the requirements of the study.

Related Conditions & MeSH terms

• Fabry Disease

Intervention

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.

Locations

Country	Name	City	State
Argentina	Investigational Site Number 032001	Capital Federal
Austria	Investigational Site Number 040001	Wien
Belgium	Investigational Site Number 056001	Liège
Brazil	Investigational Site Number 076001	Porto Alegre
Canada	Investigational Site Number 124001	Montreal
Canada	Investigational Site Number 124004	Toronto
Finland	Investigational Site Number 246002	Vaasa
France	Investigational Site Number 250002	Bron Cedex
France	Investigational Site Number 250001	Garches
Hungary	Investigational Site Number 348001	Pécs
Norway	Investigational Site Number 578001	Bergen
Poland	Investigational Site Number 616001	Warszawa
Spain	Investigational Site Number 724001	Badalona
Spain	Investigational Site Number 724002	Girona
Taiwan	Investigational Site Number 158001	Taipai
United Kingdom	Investigational Site Number 826002	London
United Kingdom	Investigational Site Number 826004	London
United States	Investigational Site Number 840003	Cincinnati	Ohio
United States	Investigational Site Number 840006	Decatur	Georgia
United States	Investigational Site Number 840002	Fairfax	Virginia
United States	Investigational Site Number 840010	La Jolla	California
United States	Investigational Site Number 840009	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

United States,  Argentina,  Austria,  Belgium,  Brazil,  Canada,  Finland,  France,  Hungary,  Norway,  Poland,  Spain,  Taiwan,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Glomerular Filtration Rate Estimated from Serum Creatinine (eGFR)	Tests are performed only for patients who are not on chronic dialysis and/or have not received a kidney transplant.	Day 1 to Week 8
Primary	Glomerular Filtration Rate by Plasma Iohexol Clearance (iGFR)	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).	Day 1 to Week 8
Primary	Protein Excretion Assessed from Three First-Morning Urine Voids		Day 1 to Week 12
Secondary	Cardiovascular Function	Assessed by electrocardiogram (ECG) and echocardiography	Day 1 to Week 8