ESPN 53rd Annual Meeting

ESPN 2021


 
ePHex: A phase 3 double-blind, placebo-controlled, randomised study to evaluate long-term efficacy and safety of Oxalobacter formigenes in patients with Primary hyperoxaluria
GEMA ARICETA 1 GESA SCHALK 2 LAURE COLLARD 3 SAOUSSEN ABROUG 4 SHABBIR MOOCHHALA 5 EDWARD GOULD 6 ABIR BOUSSETTA 7 MOHAMED BEN HMIDA 8 FAICAL JARRAYA 8 SUDARSANA DEE 9 TRACY E. HUNLEY 10 ANA BANOS 11 ELISABETH LINDNER 11 BASTIAN DEHMEL 11

1- HOSPITAL VALL D’HEBRON, BARCELONA, SPAIN
2- KINDERNIEREN-ZENTRUM, BONN, GERMANY
3- CHU, LIEGE, BELGIUM
4- HôPITAL UNIVERSITAIRE SAHLOUL, SOUSSE, TUNISIA
5- ROYAL FREE HOSPITAL, LONDON, UK
6- VANDERBILT UNIVERSITY MEDICAL CENTER, NASHVILLE, USA
7- HôPITAL CHARLES NICOLLE, TUNIS, TUNISIA
8- HôPITAL HEDI CHAKER, SFAX, TUNISIA
9- QUEEN’S MEDICAL CENTRE, NOTTINGHAM, UK
10- VANDERBILT UNIVERSITY MEDICAL CENTER, NASHVILLE, USA
11- OXTHERA IP AB, STOCKHOLM, SWEDEN
 
Introduction:

Primary hyperoxaluria (PH) is a rare genetic condition characterized by recurrent kidney stones and Chronic Kidney Disease. Oxabact® (O.formigenes) is a commensal, gram-negative, anaerobic gut bacteria with the ability to metabolise and remove excess oxalate from the body.The main objectives of the ePHex study were to evaluate efficacy and safety of Oxabact® in patients with PH, an estimated glomerular filtration rate (eGFR) < 90 ml/min/1.73 m2 and total plasma oxalate (Pox) concentration ≥ 10 μmol/L.

Material and methods:

This international 52-week study evaluated the treatment effect of Oxabact® versus placebo on Pox as the primary endpoint, and kidney function and kidney stone events as key secondary endpoints.Other endpoints included 24-h urinary oxalate (Uox) excretion, stool count for O. formigenes, nephrocalcinosis grade, safety labs and adverse events in patients (≥ 2 years of age) with any type of PH.Subjects receiving vitamin B6 were required to receive a stable dose prior to screening and were not allowed to change the dose during the study.Pre-study kidney function and kidney stone events were collected.Subjects were randomised in a 1:1 ratio to either twice daily Oxabact® or placebo and stratified based on PH type and 24-h Uox excretion.During the study, Pox, eGFR, kidney stone events and safety labs were assessed every 8-weeks.Cardiac function was determined with echocardiography (traditional and Speckle Tracking) and kidney stones with ultrasound. Stool samples and 24-h urine were collected at regular intervals, and adverse events monitored throughout the study.

Results:

Twenty-five subjects were randomized in the study.The results from the study will become available early/mid June 2021 and will be presented for the first time during the ESPN 2021 conference.

Conclusions:

The ePHex study represents the longest placebo-controlled phase 3 study with the objective to investigate efficacy and safety of Oxabact® in the treatment of patients with PH.