A Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease
Stopped early · Phase 1
Conditions studied: Fabry Disease
In brief
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.
Key facts
- Study ID
- NCT01839526
- Run by
- Genzyme, a Sanofi Company
- People needed
- 39
- Starts
- 2013-05-01
- Expected to finish
- 2016-09-01
- Last updated by the study team
- 2020-12-16
Who can join
Age: 5 and older, up to 25. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- 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 α-galactosidase A (αGAL) of <4 nmol/hr/mg leukocyte (preferred assay; results from a central laboratory). If the leukocyte αGAL activity assay is difficult to obtain, the patient may be enrolled based on documented plasma αGAL <1.5 nmol/hr/mL (results from a central laboratory), with the agreement of the Genzyme Medical Monitor.
You may not qualify if…
- 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.
Where it is running
- Investigational Site Number 840010 — La Jolla, California, United States
- Investigational Site Number 840006 — Decatur, Georgia, United States
- Investigational Site Number 840003 — Cincinnati, Ohio, United States
- Investigational Site Number 840009 — Salt Lake City, Utah, United States
- Investigational Site Number 840002 — Fairfax, Virginia, United States
- Investigational Site Number 032001 — Capital Federal, Argentina
- Investigational Site Number 040001 — Vienna, Austria
- Investigational Site Number 056001 — Liège, Belgium
- Investigational Site Number 076001 — Porto Alegre, Brazil
- Investigational Site Number 124001 — Montreal, Canada
- Investigational Site Number 124004 — Toronto, Canada
- Investigational Site Number 246002 — Vaasa, Finland
- Investigational Site Number 250002 — Bron, France
- Investigational Site Number 250001 — Garches, France
- Investigational Site Number 348001 — Pécs, Hungary
- Investigational Site Number 578001 — Bergen, Norway
- Investigational Site Number 616001 — Warsaw, Poland
- Investigational Site Number 724001 — Badalona, Spain
- Investigational Site Number 724002 — Girona, Spain
- Investigational Site Number 158001 — Taipai, Taiwan
- Investigational Site Number 826004 — London, United Kingdom
- Investigational Site Number 826002 — London, United Kingdom
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.