The phase 3 trial was stopped following a formal interim assessment that met pre specified criteria for positive efficacy in patients with chronic kidney disease.
On a recommendation from the Independent Data Monitoring Committee, the EMPA-KIDNEY trial evaluating the effect of empagliflozin (Jardiance) in adults with chronic kidney disease (CKD) will stop early due to clear positive efficacy.
The announcement on March 16 followed a formal interim assessment that met pre-specified criteria for positive efficacy, according to a news release from the Medical Research Council (MRC) Population Health Research Unit at the University of Oxford, Boehringer Ingelheim and Eli Lilly and Company.
"People with kidney disease are at risk for both worsening kidney function and heart problems, making the availability of more treatment options and relevant education critical," said Mohamed Eid, MD, MPH, vice president, Clinical Development & Medical Affairs, Cardio-Metabolism & Respiratory Medicine, Boehringer Ingelheim Pharmaceuticals, Inc. "Building on our Fast Track Designation in the US, we are very much encouraged that EMPA-KIDNEY has already demonstrated efficacy for Jardiance at this early stage, as we work to advance treatment for adults living with cardio-renal-metabolic conditions."
The phase 3 trial is the largest in CKD to date, evaluating the efficacy and safety of empagliflozin in adults with CKD who have been underrepresented in previous SGLT2 inhibitor trials. The statement noted that kidney disease is a global public health issue, affecting nearly 37 million people in the US, with links to other metabolic and cardiovascular diseases.
More than 6,000 adults with CKD were randomized in the trial conducted by the MRC Population Health Research Unit at the University of Oxford. This population included patients with mildly to severely reduced eGFR, normal and increased levels of albumin, with and without diabetes, and with CKD attributed to a range of underlying causes.
The trial's primary endpoints were a composite of kidney disease progression or cardiovascular death, with key secondary outcomes including cardiovacular death or hospitalization for heart failure, all-cause hospitalization, and all-cause mortality.
"EMPA-KIDNEY included a range of adults with kidney disease who have been excluded from, or under-represented in, previous trials focusing on the use of SGLT2 inhibitors to slow kidney disease progression," said Jeff Emmick, MD, PhD, vice president, Product Development, Lilly. "The early stop of the trial is a tremendous step toward our goal of improving the lives of adults living with kidney disease."
The FDA granted Fast Track designation to the clinical investigation of empagliflozin to reduce the risk of kidney disease progression and cardiovascular death in adults with CKD in March 2020. Full results from the EMPA-KIDNEY trial are expected to be presented at an upcoming medical congress later this year.
"We are thrilled that the trial has shown that empagliflozin is beneficial among the patients studied in EMPA-KIDNEY," said professor Richard Haynes, co-principal investigator. "We are very grateful to all of the participants who have made this trial possible and look forward to sharing detailed trial results later this year."