The Fabrazyme and Arbs and ACE Inhibitor Treatment(FAACET) Study

• Research type

  Research Study

• Full title

  Multi-center, Open-Label Study of the Safety and Efficacy of Control of Proteinuria with ACE Inhibitors and ARBS in Patients with Fabry Disease Who are Receiving Fabrazyme: The Fabrazyme + ARBs + ACE Inhibitors Treatments (FAACET) Study: The FAACET Study

• Contact name

  Stephen Waldek

• Sponsor organisation

  Salford Royal NHS Foundation Trust

• Eudract number

  2007-007482-21

• Clinicaltrials.gov Identifier

  NCT00446862

• Research summary

  Multi-centre, Open-label (treatment name is known) Study of the Safety and Efficacy (effectiveness)of Control of Proteinuria (protein in urine)with ACE Inhibitors and ARBS(anti high blood pressure medication) in Patients with Fabry Disease Who Are Receiving Fabrazyme©:(enzyme treatment for fabry disease) The use of ACEI and ARBS (anti high blood pressure medication) is well established in nearly all forms of proteinuric renal diseases (protein in urine from kidney disease) especially diebetes and glomerulonephritis (kidney disease). The anti-proteinuria effects of ACEI/ARB therapy has not been systematically researched in patients with fabry disease as they do not usually have high blood pressure so would not routinely receive this medication. Preliminary pilot (Initial) studies at University of Alabama Birmingham USA with a small number of (06) Fabry disease patients with proteinuria and initial eGFRMDRD <_60 ml/min/1.73 m2 (balance of craetinine and protein in the urine) who were treated with Fabrazyme© (enzyme replacement used to treat patients with Fabry disease) and a combination of ACEI and ARBS (anti high blood pressure tablets) showed markedly improved outcome in the slowing down of the progression of kidney disease. If the single centre experience at University of Alabama Birmingham USA can be replicated (reproduced) in the multi-centre, multinational FAACET Study, then the benefits of treating Fabry patients with Fabrazyme© and ACE1 and ARB's medication even with severe kidney involvement and proteinuria will be confirmed and will clearly benefit these patients by slowing the rate at which their kidney function declines more than has been observed with Fabrazyme© treatment alone.

• REC name

  South Central - Hampshire B Research Ethics Committee

• REC reference

  09/H0504/103

• Date of REC Opinion

  9 Nov 2009

• REC opinion

  Further Information Favourable Opinion