US-based biotechnology firm La Jolla Pharmaceutical has started a Phase IIb clinical trial of its galectin-3 inhibitor, GCS-100, to treat advanced chronic kidney disease (CKD) caused by diabetes.
Around 375 diabetic patients with Stage 3b or 4 CKD will be enrolled in the double-blind, multi-centre, placebo-controlled, randomised Phase IIb trial of GCS-100.
During the trial, patients will be randomised 1:1:1:1 to receive fixed doses of GCS-100 (1, 3 or 9mg) or placebo through IV injection once a week for eight weeks and then once every other week for an additional 16 weeks.
CKD is characterised by a gradual loss of kidney function over time that may eventually lead to kidney failure, requiring dialysis or a kidney transplant to maintain life.
The trial's primary endpoint is to compare the change in kidney function, as measured by estimated glomerular filtration rate (eGFR), from baseline to week 26, which is two weeks after the last injection, between patients receiving GCS-100 or placebo.
Secondary efficacy endpoints of the trial include a responder analysis based on pre-specified percentage changes in eGFR and an analysis on progression to renal replacement therapy, while other secondary endpoints are focused on the long-term safety and tolerability of GCS-100, including assessment of the incidence of major cardiac events.
University of Texas Health Science Center clinical associate professor of Medicine and the trial principal investigator Dr Pablo Pergola said: "Chronic kidney disease is an enormous and growing medical problem worldwide.
"A disease-modifying agent that could slow and potentially reverse the tissue fibrosis that is a hallmark of this disease would be a welcome advance in the field and could have a major impact on patients' lives.
"Our experience with GCS-100 in previous trials was very encouraging, and my patients tolerated the therapy well."
GCS-100 is a complex polysaccharide derived from pectin that binds to, and blocks the activity of, the pro-fibrotic mediator galectin-3.
The company had recently completed a multicentre, randomised, placebo-controlled, Phase II trial in advanced CKD patients, in which treatment with GCS-100 resulted in a statistically significant improvement in kidney function compared to placebo.
La Jolla president and chief executive officer George Tidmarsh said: "This Phase IIb trial will build on the knowledge we gained from our recently completed Phase II trial, in which treatment with GCS-100 resulted in a statistically significant improvement in kidney function."