A study to assess the renal function in young male patients with Fabry disease who have never received any specific treatment for this disease.
- Conditions
- Fabry diseaseMedDRA version: 15.0Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2012-001966-14-ES
- Lead Sponsor
- Genzyme, a Sanofi Company
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 39
A patient must meet all of the following criteria to be eligible for this study.
1. 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.
2. The patient must have a confirmed diagnosis of Fabry disease as documented by leukocyte ?-galactosidase A (?GAL) of <4 mol/hr/mg leukocyte (preferred assay; results from a central
laboratory). If the leukocyte ?GAL activity assay is difficult to obtain, the patient may be enrolled based on documented plasma ?GAL <1.5 nmol/hr/mL (results from a central laboratory), with the
agreement of the Genzyme Medical Monitor.
3. The patient must be male and ?5 and ?25 years of age at screening.
Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 15
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
A patient who meets any of the following criteria will be excluded from this study.
1. Patient has received prior treatment with enzyme replacement therapy (ERT) or oral pharmacological chaperone therapy for Fabry disease.
2. Patient has received an investigational drug within 30 days of the screening visit.
3. 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.
4. Patient has any contraindication mentioned in the labeling of iohexol. NOTE: patients with an eGFR <30 mL/min/1.73m2 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.
5. 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).
6. 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method