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

Phase 4

Withdrawn

• Conditions: inherited enzyme deficiency; metabolic disorder; 10083624; 10021605

• Registration Number: NL-OMON39057
• Lead Sponsor: Sanofi-aventis

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 29 April 2024

Recruitment & Eligibility

• Status: Withdrawn
• Sex: Not specified
• Target Recruitment: 3

Inclusion Criteria

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 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.
3. The patient must be male and >=5 and <=25 years of age at screening.

Exclusion Criteria

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.
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: Observational invasive
• Study Design: Not specified